Over the last two decades, there have been several breakthroughs related to the development of gene therapies. In 2020, Libmeldy™, an ex vivo gene therapy received approval for the treatment of metachromatic leukodystrophy. To provide more context, the treatment regimen of such therapies, encompassing gene replacement and gene-editing modalities, is aimed at correction of the mutated gene in patients using molecular carriers (viral and non-viral vectors). Further, post the onset of the COVID-19 pandemic, there has been a steady increase in the investigational new drug (IND) applications filed for cell and gene therapies. In fact, in 2021, more than 200 gene therapies were being evaluated in phase II and III studies. Moreover, in 2022, six gene therapies are expected to receive the USFDA market approval. Promising results from ongoing clinical research initiatives have encouraged government and private firms to make investments to support therapy product development initiatives in this domain. In 2021 alone, gene therapy developers raised around USD 9.5 billion in capital investments. Taking into consideration the continuous progress in this domain, gene therapies are anticipated to be used for the treatment of 1.1 million patients suffering from a myriad of disease indications, by 2035.
Presently, more than 250 companies are engaged in the development of various early and late-stage gene therapies, worldwide. In recent years, there has been a significant increase in the integration of novel technologies, such as gene modification, gene-editing, genome sequencing and manipulation technologies (molecular switches), in conjugation with gene delivery methods. For instance, the CRISPR-Cas9 based gene-editing ool is one of the remarkable technological advancements, which enables the precise alteration of the transgene. It is worth mentioning that the new generation delivery platforms, including nanoparticles and hybrid vector systems, have been demonstrated to be capable of enabling effective and safe delivery of gene based therapeutics. Further, a variety of consolidation efforts are currently ongoing in this industry. Such initiatives are primarily focused on expanding and strengthening the existing development efforts; this can be validated from the fact that 56% of the total acquisitions reported in the domain were focused on drug class consolidation. Driven by the collective and consistent efforts of developers and the growing demand for a single dose of effective therapeutic, the gene therapy market is anticipated to witness significant growth in the foreseen future.
Scope of the Report
The Gene Therapy Market (5th Edition) by Type of Therapy (Gene Augmentation, Oncolytic Viral Therapy, Immunotherapy and Others), Type of Gene Delivery Method Used (Ex vivo and In vivo), Type of Vector Used (Adeno-associated Virus, Adenovirus, Herpes Simplex Virus, Lentivirus, Non-Viral Vectors, Retrovirus and Others), Target Therapeutic Areas (Cardiovascular Diseases, Dermatological Diseases, Genetic Diseases, Hematological Diseases, Infectious Diseases, Metabolic Diseases, Muscle-related Diseases, Oncological Diseases, Ophthalmic Diseases and Others), Route of Administration (Intraarticular, Intracerebral, Intracoronary, Intradermal, Intralesional, Intramuscular, Intrapleural, Intrathecal, Intratumoral, Intravenous, Intravesical, Intravitreal, Subretinal, Topical and Others), and Key Geographical Regions (US, Europe, Asia-Pacific and rest of the world): Industry Trends and Global Forecasts, 2022-2035 report features an extensive study of the current market landscape and the likely future potential associated with the gene therapy market, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, immunotherapies and gene editing therapies. Amongst other elements, the report features:
- A detailed overview of the overall market landscape of gene therapies, including information on their phase of development (marketed, clinical, preclinical and discovery), key therapeutic areas (autoimmune diseases, cardiovascular diseases, dermatological diseases, genetic diseases, hematological diseases, hepatic diseases, immunological diseases, infectious diseases, inflammatory diseases, metabolic diseases, muscle-related diseases, neurological diseases, oncological diseases, ophthalmic diseases and others), target disease indication(s), type of vector used, type of gene/molecule targeted, type of therapy (gene augmentation, immunotherapy, oncolytic viral therapy and others), type of gene delivery method used (ex vivo and in vivo), route of administration and special drug designation(s) awarded (if any).
- A detailed overview of the current market landscape of players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain.
- An elaborate discussion on the various types of viral and non-viral vectors, along with information on design, manufacturing requirements, advantages and limitations of currently available gene delivery vectors.
- A discussion on the regulatory landscape related to gene therapies across various geographies, namely North America (the US and Canada), Europe and Asia-Pacific (Australia, China, Hong Kong, Japan and South Korea), providing details related to the various challenges associated with obtaining reimbursements for gene therapies.
- An elaborate discussion on the various commercialization strategies that have been adopted by drug developers engaged in this domain across different stages of therapy development, including prior to drug launch, at/during drug launch and post-marketing stage.
- Detailed profiles of marketed and late stage (phase II/III and above) gene therapies, along with information on the development timeline of the therapy, current development status, mechanism of action, affiliated technology, patent portfolio strength, dosage and manufacturing details, as well as details related to the developer company.
- A review of the various emerging technologies and therapy development platforms that are being used to manufacture gene therapies, featuring detailed profiles of technologies that were/are being used for the development of four or more products/product candidates.
- An in-depth analysis of various patents that have been filed/granted related to gene therapies and gene editing therapies, since 2017, based on several relevant parameters, such as type of patent (granted patents, patent applications and others), publication year, regional applicability, CPC symbols, emerging focus areas, leading industry players (in terms of the number of patents filed/granted), and patent valuation.
- A detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2022, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company’ product, target therapeutic area and deal multiples.
- An analysis of the investments made at various stages, such as seed financing, venture capital financing, IPOs, secondary offerings, debt financing, grants and other equity offerings, by companies that are engaged in this domain.
- An analysis of completed, ongoing and planned clinical studies, based on several relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population.
- An analysis of the various factors that are likely to influence the pricing of gene therapies, featuring different models/approaches that may be adopted by manufacturers to decide the prices of these therapies.
- An analysis of the startup companies engaged in this domain (established between 2017-2022) based on year of experience.
- A detailed review of the various gene therapy-based initiatives undertaken by big pharma players, highlighting trend across parameters, such as number of gene therapies under development, funding information, partnership activity and patent portfolio strength.
- An informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength.
- A case study on the prevalent and emerging trends related to vector manufacturing, along with information on companies offering contract services for manufacturing vectors. The study also includes a detailed discussion on the manufacturing processes associated with various types of vectors.
A discussion on the various operating models adopted by gene therapy developers for supply chain management, highlighting the stakeholders involved, factors affecting the supply of therapeutic products and challenges encountered by developers across the different stages of the gene therapy supply chain.
One of the key objectives of the report was to estimate the existing market size and the future opportunity associated with gene therapies, over the next decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the evolution of the market for the period 2022-2035. Our year-wise projections of the current and future opportunity have further been segmented on the basis of [A] type of therapy (gene augmentation, immunotherapy, oncolytic viral therapy and others), [B] type of gene delivery method used (ex vivo and in vivo), [C] type of vector used (adeno-associated virus, adenovirus, herpes simplex virus, lentivirus, non-viral vectors, retrovirus and others), [D] target therapeutic areas (cardiovascular diseases, dermatological diseases, genetic diseases, hematological diseases, infectious diseases, metabolic diseases, muscle-related diseases, oncological diseases, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebral, intracoronary, intradermal, intralesional, intramuscular, intrapleural, intrathecal, intratumoral, intravenous, intravesical, intravitreal, subretinal, topical and others), and [F] key geographical regions (US, Europe, Asia-Pacific and rest of the world). In order to account for future uncertainties and to add robustness to our model, we have provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industry’s growth.
The opinions and insights presented in this study were Influenced by discussions conducted with multiple stakeholders in this domain. The report features detailed transcripts of interviews held with the following individuals:
- Buel Dan Rodgers (Founder and CEO, AAVogen)
- Sue Washer (President and CEO, AGTC)
- Patricia Zilliox (President and CEO, Eyevensys)
- Christopher Reinhard (CEO and Chairman, Gene Biotherapeutics (previously known as Cardium Therapeutics))
- Adam Rogers (CEO, Hemera Biosciences)
- Ryo Kubota (CEO, Chairman and President, Kubota Pharmaceutical Holdings (Acucela))
- Al Hawkins (CEO, Milo Biotechnology)
- Jean-Phillipe Combal (CEO, Vivet Therapeutics)
- Robert Jan Lamers (former CEO, Arthrogen)
- Tom Wilton (former CBO, LogicBio Therapeutics)
- Michael Triplett (former CEO, Myonexus Therapeutics)
- Molly Cameron (former Corporate Communications Manager, Orchard Therapeutics)
- Cedric Szpirer (Executive and Scientific Director, Delphi Genetics)
- Marco Schmeer (Project Manager) and Tatjana Buchholz (former Marketing Manager, PlasmidFactory)
- Jeffrey Hung (CCO, Vigene Biosciences)
All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.
Key Questions Answered
- Who are the key industry players engaged in the development of gene therapies?
- How many gene therapy candidates are present in the current development pipeline? Which key disease indications are targeted by such products?
- Which types of vectors are most commonly used for effective delivery of gene therapies?
- What are the key regulatory requirements for gene therapy approval, across various geographies?
- Which commercialization strategies are most commonly adopted by gene therapy developers, across different stages of development?
- What are the different pricing models and reimbursement strategies currently being adopted for gene therapies?
- What are the various technology platforms that are either available in the market or are being designed for the development of gene therapies?
- Who are the key CMOs/CDMOs engaged in supplying viral/plasmid vectors for gene therapy development?
- What are the key value drivers of the merger and acquisition activity in the gene therapy industry?
- Who are the key stakeholders that have actively made investments in the gene therapy domain?
- Which are the most active trial sites (in terms of number of clinical studies being conducted) in this domain?
- How is the current and future market opportunity likely to be distributed across key market segments?
Please note: This report can be updated on request. Please contact our Customer Experience team using the Ask a Question widget on our website.
Table of Contents
1. PREFACE1.1. Scope of the Report
1.2. Research Methodology
1.3. Key Questions Answered
1.4. Chapter Outlines
2. EXECUTIVE SUMMARY
2.1. Chapter Overview
3. INTRODUCTION
3.1. Context and Background
3.2. Evolution of Gene Therapies
3.3. Classification of Gene Therapies
3.3.1. Somatic and Germline Gene Therapies
3.3.2. Ex Vivo and In Vivo Gene Therapies
3.4. Routes of Administration
3.5. Mechanism of Action
3.6. Overview of Gene Editing
3.6.1. Evolution of Genome Editing
3.6.2. Applications of Genome Editing
3.6.3. Available Genome Editing Techniques
3.7. Advantages and Disadvantages of Gene Therapies
3.7.1 Ethical and Social Concerns Related to Gene Therapies
3.7.2. Constraints and Challenges Related to Gene Therapies
3.7.3. Therapy Development Concerns
3.7.4. Manufacturing Concerns
3.7.5. Commercial Viability Concerns
4. GENE DELIVERY VECTORS
4.1. Chapter Overview
4.2. Viral and Non-Viral Methods of Gene Transfer
4.3. Viral Vectors for Genetically Modified Therapies
4.4. Types of Viral Vectors
4.4.1. Adeno-associated Viral Vectors
4.4.1.1. Overview
4.4.1.2. Design and Manufacturing
4.4.1.3. Advantages Offered
4.4.1.4. Associated Limitations
4.4.2. Adenoviral Vectors
4.4.2.1. Overview
4.4.2.2. Design and Manufacturing
4.4.2.3. Advantages Offered
4.4.2.4. Associated Limitations
4.4.3. Lentiviral Vectors
4.4.3.1. Overview
4.4.3.2. Design and Manufacturing
4.4.3.3. Advantages Offered
4.4.3.4. Associated Limitations
4.4.4. Retroviral Vectors
4.4.4.1. Overview
4.4.4.2. Design and Manufacturing
4.4.4.3. Advantages Offered
4.4.4.4. Associated Limitations
4.4.5. Other Viral Vectors
4.4.5.1. Alphavirus
4.4.5.2. Foamy Virus
4.4.5.3. Herpes Simplex Virus
4.4.5.4. Sendai Virus
4.4.5.5. Simian Virus
4.4.5.6. Vaccinia Virus
4.5. Types of Non-Viral Vectors
4.5.1. Plasmid DNA
4.5.2. Liposomes, Lipoplexes and Polyplexes
4.5.3. Oligonucleotides
4.5.4. Nanoparticles
4.5.5. Hybrid Vector Systems
4.5.6. Other Non-Viral Vectors
4.5.7. Gene Delivery Using Non-Viral Vectors
4.5.7.1. Biolistic Methods
4.5.7.2. Electroporation
4.5.7.3. Receptor Mediated Gene Delivery
4.5.7.4. Gene Activated Matrix (GAM)
5. REGULATORY LANDSCAPE AND REIMBURSEMENT SCENARIO
5.1. Chapter Overview
5.2. Regulatory Guidelines in North America
5.2.1. The US Scenario
5.2.2. Canadian Scenario
5.3. Regulatory Guidelines in Europe
5.3.1. Quality of Gene Therapy Products
5.3.2. Non-Clinical Development
5.3.3. Clinical Development
5.4. Regulatory Guidelines in Asia-Pacific
5.4.1. Chinese Scenario
5.4.1.1. Construction of DNA Expression Cassette and Gene Delivery Systems
5.4.1.2. Generation and Characterization of Cell Banks and Engineered Bacteria Banks
5.4.1.3. Manufacturing of Gene Therapy Products
5.4.1.4. Quality Control
5.4.1.5. Evaluation of Efficacy of Gene Therapy Products
5.4.1.6. Safety Evaluation of Gene Therapy Products
5.4.1.7. Clinical Trial of Gene Therapy Products
5.4.1.8. Ethics Study
5.4.2. Japanese Scenario
5.4.3. South Korean Scenario
5.4.4. Australian Scenario
5.4.5. Hong Kong Scenario
5.5. Reimbursement Scenario
5.5.1. Challenges Related to Reimbursement
5.6. Commonly Offered Payment Models for Gene Therapies
6. MARKET OVERVIEW
6.1. Chapter Overview
6.1.1. Analysis of Gene Therapy Candidates by Stage of Development
6.2. Gene Therapy Market: Clinical and Commercial Pipeline
6.2.1. Analysis by Phase of Development
6.2.2. Analysis by Therapeutic Area
6.2.3. Analysis by Type of Vector Used
6.2.4. Analysis by Type of Gene/Molecule Targeted
6.2.5. Analysis by Type of Therapy
6.2.6. Analysis by Type of Gene Delivery Method Used
6.2.7. Analysis by Route of Administration
6.3. Gene Therapy Market: Early-Stage Pipeline
6.3.1. Analysis by Phase of Development
6.3.2. Analysis by Therapeutic Area
6.3.3. Analysis by Type of Vector Used
6.3.4. Analysis by Type of Therapy
6.3.5. Analysis by Type of Gene Delivery Method Used
6.3.6. Analysis by Route of Administration
6.4. Gene Therapy Market: Special Drug Designations
6.4.1. Analysis by Special Designation(s) Awarded
6.5. Analysis by Phase of Development, Therapeutic Area and Type of Therapy (Grid Representation)
7. COMPETITIVE LANDSCAPE
7.1. Chapter Overview
7.2. Gene Therapy Market: List of Developers
7.2.1. Analysis by Year of Establishment
7.2.2. Analysis by Company Size
7.2.3. Analysis by Location of Headquarters
7.2.3.1. Analysis by Year of Establishment, Company Size and Location of Headquarters
7.2.3.2. Regional Landscape
7.3. Key Players: Analysis by Number of Pipeline Candidates
8. MARKETED GENE THERAPIES
8.1. Chapter Overview
8.2. Gendicine® (Shenzhen Sibiono GeneTech)
8.2.1. Company Overview
8.2.2. Development Timeline
8.2.3. Mechanism of Action and Vector Used
8.2.4. Target Indication(s)
8.2.5. Current Status of Development
8.2.6. Manufacturing, Dosage and Sales
8.3. Oncorine® (Shanghai Sunway Biotech)
8.3.1. Company Overview
8.3.2. Development Timeline
8.3.3. Mechanism of Action and Vector Used
8.3.4. Target Indication(s)
8.3.5. Current Status of Development
8.3.6. Manufacturing, Dosage and Sales
8.4. Rexin-G® (Epeius Biotechnologies)
8.4.1. Company Overview
8.4.2. Development Timeline
8.4.3. Mechanism of Action and Vector Used
8.4.4. Target Indication(s)
8.4.5. Current Status of Development
8.4.6. Manufacturing, Dosage and Sales
8.5. Neovasculgen® (Human Stem Cells Institute)
8.5.1. Company Overview
8.5.2. Development Timeline
8.5.3. Mechanism of Action and Vector Used
8.5.4. Target Indication(s)
8.5.5. Current Status of Development
8.5.6. Manufacturing, Dosage and Sales
8.6. Imlygic® (Amgen)
8.6.1. Company Overview
8.6.2. Development Timeline
8.6.3. Mechanism of Action and Vector Used
8.6.4. Target Indication(s)
8.6.5. Current Status of Development
8.6.6. Manufacturing, Dosage and Sales
8.7. Strimvelis® (Orchard Therapeutics)
8.7.1. Company Overview
8.7.2. Development Timeline
8.7.3. Mechanism of Action and Vector Used
8.7.4. Target Indication(s)
8.7.5. Current Status of Development
8.7.6. Manufacturing, Dosage and Sales
8.8. Luxturna™ (Spark Therapeutics)
8.8.1. Company Overview
8.8.2. Development Timeline
8.8.3. Mechanism of Action and Vector Used
8.8.4. Target Indication(s)
8.8.5. Current Status of Development
8.8.6. Manufacturing, Dosage and Sales
8.9. Zolgensma™ (Novartis)
8.9.1. Company Overview
8.9.2. Development Timeline
8.9.3. Mechanism of Action and Vector Used
8.9.4. Target Indication(s)
8.9.5. Current Status of Development
8.9.6. Manufacturing, Dosage and Sales
8.10. Collategene® (AnGes)
8.10.1. Company Overview
8.10.2. Development Timeline
8.10.3. Mechanism of Action and Vector Used
8.10.4. Target Indication(s)
8.10.5. Current Status of Development
8.10.6. Manufacturing, Dosage and Sales
8.11. Zyntelgo™ (bluebird bio)
8.11.1. Company Overview
8.11.2. Development Timeline
8.11.3. Mechanism of Action and Vector Used
8.11.4. Target Indication(s)
8.11.5. Current Status of Development
8.11.6. Manufacturing, Dosage and Sales
8.12. Libmeldy™ (Orchard Therapeutics)
8.12.1. Company Overview
8.12.2. Development Timeline
8.12.3. Mechanism of Action and Vector Used
8.12.4. Target Indication(s)
8.12.5. Current Status of Development
8.12.6. Manufacturing, Dosage and Sales
9. KEY COMMERCIALIZATION STRATEGIES
9.1. Chapter Overview
9.2. Successful Drug Launch Strategy: ROOTS Framework
9.3. Successful Drug Launch Strategy: Product Differentiation
9.4. Commonly Adopted Commercialization Strategies based on Phase of Development of Product
9.5. List of Currently Approved Gene Therapies
9.6. Key Commercialization Strategies Adopted by Gene Therapy Developers
9.6.1. Strategies Adopted Before Therapy Approval
9.6.1.1. Participation in Global Events
9.6.1.2. Collaboration with Stakeholders and Pharmaceutical Firms
9.6.1.3. Indication Expansion
9.6.2. Strategies Adopted During/Post Therapy Approval
9.6.2.1. Geographical Expansion
9.6.2.2. Participation in Global Events
9.6.2.3. Patience Assistance Programs
9.6.2.4. Awareness through Product Websites
9.6.2.5. Collaboration with Stakeholders and Pharmaceutical Firms
9.7. Concluding Remarks
10. LATE STAGE (PHASE II/III AND ABOVE) GENE THERAPIES
10.1. Chapter Overview
10.2. Lumevoq® (GS010): Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.3. OTL-103: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.4. PTC-AADC: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.5. BMN 270: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.6. rAd-IFN/Syn3: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.7. beti-cel: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.8. eli-cel: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.9. lovo-cel: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.10. SRP-9001: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.11. EB-101: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.12. ProstAtak®: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.13. D-Fi: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.14. CG0070: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.15. Vigil™-EWS: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.16. Engensis®: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.17. VGX-3100: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.18. Invossa™ (TG-C): Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.19. VYJUVEKT™: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.20. PF-06939926: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.21. PF-06838435: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.22. PF-07055480: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.23. SPK-8011: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.24. AMT-061: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.25. VB-111: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.26. Generx®: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.27. ADXS-HPV: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.28. AGTC 501: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.29. LYS-SAF302: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.30. NFS-01: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.31. AG0302-COVID19: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.32. RGX-314: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.33. Hologene 5: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
11. EMERGING TECHNOLOGIES
11.1. Chapter Overview
11.2. Gene Editing Technologies
11.2.1. Overview
11.2.2. Applications
11.3. Emerging Gene Editing Platforms
11.3.1. CRISPR/Cas9 System
11.3.1.1. Key Components and Functions
11.3.1.2. Mechanism of Action
11.3.1.3. Targeting Efficiency and Challenges
11.3.1.4. Next-GEN CRISPR Technology
11.3.1.5. Technology Providers
11.3.2. TALENs
11.3.2.1. Structural Features
11.3.2.2. Mechanism of Action
11.3.2.3. Advantages and Challenges
11.3.3. megaTAL
11.3.3.1. Technology Providers
11.3.4. Zinc Finger Nuclease
11.3.4.1. Technology Providers
11.4. Gene Expression Regulation Technologies
11.4.1. Technology Providers
11.5. Technology Platforms for Developing/Delivering Gene Therapies
12. KEY THERAPEUTICS AREAS
12.1. Chapter Overview
12.2. Analysis by Therapeutic Area and Special Designation(s) Awarded
12.3. Oncological Diseases
12.3.1. Analysis by Target Indication
12.3.2. Analysis by Type of Vector Used
12.4. Neurological Diseases
12.4.1. Analysis by Target Indication
12.4.2. Analysis by Type of Vector Used
12.5. Ophthalmic Diseases
12.5.1. Analysis by Target Indication
12.5.2. Analysis by Type of Vector Used
12.6. Metabolic Diseases
12.6.1. Analysis by Target Indication
12.6.2. Analysis by Type of Vector Used
12.7. Genetic Diseases
12.7.1. Analysis by Target Indication
12.7.2. Analysis by Type of Vector Used
13. PATENT ANALYSIS
13.1. Chapter Overview
13.2. Gene Therapy Market: Patent Analysis
13.2.1. Scope and Methodology
13.2.1.1. Analysis by Publication Year
13.2.1.2. Analysis by Publication Year and Type of Patent
13.2.1.3. Analysis by Geography
13.2.1.4. Analysis by CPC Symbols
13.2.1.5. Analysis by Emerging Focus Areas
13.2.1.6. Leading Players: Analysis by Number of Patents
13.2.1.7. Patent Benchmark Analysis
13.2.1.8. Patent Valuation Analysis
13.3. Gene Editing Market: Patent Analysis
13.3.1. Scope and Methodology
13.3.1.1. Analysis by Publication Year
13.3.1.2. Analysis by Publication Year and Type of Patent
13.3.1.3. Analysis by Geography
13.3.1.4. Analysis by CPC Symbols
13.3.1.5. Analysis by Emerging Focus Areas
13.3.1.6. Leading Players: Analysis by Number of Patents
13.3.1.7. Patent Benchmark Analysis
13.3.1.8. Patent Valuation Analysis
13.4. Overall Intellectual Property Portfolio: Analysis by Type of Organization
14. MERGERS AND ACQUISITIONS
14.1. Chapter Overview
14.2. Merger and Acquisition Models
14.3. Gene Therapy Market: Mergers and Acquisitions
14.3.1. Analysis by Year of Merger/Acquisition
14.3.2. Analysis by Type of Agreement
14.3.3. Analysis by Geography
14.3.3.1. Continent-wise Distribution
14.3.3.2. Intercontinental and Intracontinental Deals
14.3.3.3. Local and International Deals
14.3.4. Analysis by Key Value Drivers
14.3.4.1. Analysis by Key Value Drivers and Year of Acquisition
14.3.5. Analysis by Phase of Development of the Acquired Company’s Product
14.3.6. Analysis by Therapeutic Area
14.3.7. Analysis by Deal Multiples
15. FUNDING AND INVESTMENT ANALYSIS
15.1. Chapter Overview
15.2. Types of Funding
15.3. Gene Therapy Market: Funding and Investment Analysis
15.3.1. Analysis of Funding Instances by Year
15.3.2. Analysis of Amount Invested by Year
15.3.3. Analysis of Funding Instances and Amount Invested by Type of Funding
15.3.4. Analysis of Funding Instances and Amount Invested by Year and Type of Funding
15.3.5. Analysis of Funding Instances and Amount Invested by Type of Therapy
15.3.6. Analysis of Funding Instances and Amount Invested by Geography
15.3.7. Analysis of Funding Instances and Amount Invested by Highest Phase of Development
15.3.8. Analysis of Funding Instances by Therapeutic Area
15.3.9. Most Active Players: Analysis by Number of Funding Instances and Amount Invested
15.3.10. Key Investors: Analysis by Number of Funding Instances
15.4. Concluding Remarks
16. CLINICAL TRIAL ANALYSIS
16.1. Chapter Overview
16.2. Scope and Methodology
16.3. Gene Therapy Market: Clinical Trial Analysis
16.3.1. Analysis by Trial Registration Year
16.3.2. Analysis by Trial Status
16.3.3. Analysis by Trial Phase
16.3.4. Analysis by Therapeutic Area
16.3.5. Analysis by Geography
16.3.6. Analysis by Trial Registration Year and Geography
16.3.7. Analysis by Trial Status and Geography
16.3.8. Analysis by Trial Status, Trial Phase and Geography
16.3.9. Analysis by Therapeutic Area and Geography
16.4. Analysis by Type of Sponsor
16.5. Analysis by Prominent Treatment Sites
16.6. Gene Therapy Market: Analysis of Enrolled Patient Population
16.6.1. Analysis by Trial Registration Year
16.6.2. Analysis by Trial Status
16.6.3. Analysis by Trial Phase
16.6.4. Analysis by Therapeutic Area
16.6.5. Analysis by Location of Trial Site
16.6.6. Analysis by Trial Status and Location of Trial Site
16.6.7. Analysis by Trial Status, Trial Phase and Location of Trial Site
16.6.8. Analysis by Therapeutic Area and Location of Trial Site
16.7. Concluding Remarks
16.7.1. Emerging Molecules to Watch
16.7.2. Most Important Trials to Watch
17. COST PRICE ANALYSIS
17.1. Chapter Overview
17.2. Gene Therapy Market: Factors Contributing to the Price of Gene Therapies
17.3. Gene Therapy Market: Pricing Models
17.3.1. Based on Associated Product/Component Costs
17.3.2. Based on Competition
17.3.3. Based on Patient Segment
17.3.4. Based on Opinions of Industry Experts
18. START-UP VALUATION
18.1. Chapter Overview
18.2. Valuation by Year of Experience
18.2.1. Methodology
18.2.2. Results and Interpretation
19. BIG PHARMA PLAYERS: ANALYSIS OF GENE THERAPY RELATED INITIATIVES
19.1. Chapter Overview
19.2. Gene Therapy Market: List of Most Prominent Big Pharmaceutical Players
19.2.1. Analysis by Therapeutic Area
19.2.2. Analysis by Type of Vector Used
19.2.3. Analysis by Type of Therapy
19.2.4. Analysis by Type of Gene Delivery Method
19.3. Benchmark Analysis of Key Parameters
19.3.1. Spider Web Analysis: Pipeline Strength
19.3.2. Spider Web Analysis: Merger/Acquisitions
19.3.3. Spider Web Analysis: Funding and Investments
19.3.4. Spider Web Analysis: Clinical Trials
19.3.5. Spider Web Analysis: Technologies
19.3.6. Spider Web Analysis: Patents
19.4. Benchmark Analysis of Big Pharmaceutical Players
20. DEMAND ANALYSIS
20.1. Chapter Overview
20.2. Methodology
20.3. Global Demand for Gene Therapies, 2022-2035
20.3.1. Analysis by Type of Therapy
20.3.2. Analysis by Therapeutic Area
20.3.3. Analysis by Geography
21. MARKET FORECAST AND OPPORTUNITY ANALYSIS
21.1. Chapter Overview
21.2. Scope and Limitations
21.3. Key Assumptions and Forecast Methodology
21.4. Global Gene Therapy Market, 2022-2035
21.4.1. Gene Therapy Market: Analysis by Therapeutic Area
21.4.2. Gene Therapy Market: Analysis by Type of Vector Used
21.4.3. Gene Therapy Market: Analysis by Type of Therapy
21.4.4. Gene Therapy Market: Analysis by Gene Delivery Method Used
21.4.5. Gene Therapy Market: Analysis by Route of Administration
21.4.6. Gene Therapy Market: Analysis by Geography
21.5. Gene Therapy Market: Value Creation Analysis
21.6. Gene Therapy Market: Product-wise Sales Forecasts
21.6.1. Gendicine®
21.6.1.1. Target Patient Population
21.6.1.2. Sales Forecast
21.6.1.3. Net Present Value
21.6.1.4. Value Creation Analysis
21.6.2. Oncorine®
21.6.2.1. Target Patient Population
21.6.2.2. Sales Forecast
21.6.2.3. Net Present Value
21.6.2.4. Value Creation Analysis
21.6.3. Rexin-G®
21.6.3.1. Target Patient Population
21.6.3.2. Sales Forecast
21.6.3.3. Net Present Value
21.6.3.4. Value Creation Analysis
21.6.4. Neovasculgen®
21.6.4.1. Target Patient Population
21.6.4.2. Sales Forecast
21.6.4.3. Net Present Value
21.6.4.4. Value Creation Analysis
21.6.5. Strimvelis®
21.6.5.1. Target Patient Population
21.6.5.2. Sales Forecast
21.6.5.3. Net Present Value
21.6.5.4. Value Creation Analysis
21.6.6. Imlygic®
21.6.6.1. Target Patient Population
21.6.6.2. Sales Forecast
21.6.6.3. Net Present Value
21.6.6.4. Value Creation Analysis
21.6.7. Luxturna™
21.6.7.1. Target Patient Population
21.6.7.2. Sales Forecast
21.6.7.3. Net Present Value
21.6.7.4. Value Creation Analysis
21.6.8. Zolgensma™
21.6.8.1. Target Patient Population
21.6.8.2. Sales Forecast
21.6.8.3. Net Present Value
21.6.8.4. Value Creation Analysis
21.6.9. Collategene®
21.6.9.1. Target Patient Population
21.6.9.2. Sales Forecast
21.6.9.3. Net Present Value
21.6.9.4. Value Creation Analysis
21.6.10. Libmeldy™
21.6.10.1. Target Patient Population
21.6.10.2. Sales Forecast
21.6.10.3. Net Present Value
21.6.10.4. Value Creation Analysis
21.6.11. Lumevoq® (GS010)
21.6.11.1. Target Patient Population
21.6.11.2. Sales Forecast
21.6.11.3. Net Present Value
21.6.11.4. Value Creation Analysis
21.6.12. OTL-103
21.6.12.1. Target Patient Population
21.6.12.2. Sales Forecast
21.6.12.3. Net Present Value
21.6.12.4. Value Creation Analysis
21.6.13. PTC-AADC
21.6.13.1. Target Patient Population
21.6.13.2. Sales Forecast
21.6.13.3. Net Present Value
21.6.13.4. Value Creation Analysis
21.6.14. BMN 270
21.6.14.1. Target Patient Population
21.6.14.2. Sales Forecast
21.6.14.3. Net Present Value
21.6.14.4. Value Creation Analysis
21.6.15. rAd-IFN/Syn3
21.6.15.1. Target Patient Population
21.6.15.2. Sales Forecast
21.6.15.3. Net Present Value
21.6.15.4. Value Creation Analysis
21.6.16. beti-cel
21.6.16.1. Target Patient Population
21.6.16.2. Sales Forecast
21.6.16.3. Net Present Value
21.6.16.4. Value Creation Analysis
21.6.17. eli-cel
21.6.17.1. Target Patient Population
21.6.17.2. Sales Forecast
21.6.17.3. Net Present Value
21.6.17.4. Value Creation Analysis
21.6.18. lovo-cel
21.6.18.1. Target Patient Population
21.6.18.2. Sales Forecast
21.6.18.3. Net Present Value
21.6.18.4. Value Creation Analysis
21.6.19. SRP-9001
21.6.19.1. Target Patient Population
21.6.19.2. Sales Forecast
21.6.19.3. Net Present Value
21.6.19.4. Value Creation Analysis
21.6.20. EB-101
21.6.20.1. Target Patient Population
21.6.20.2. Sales Forecast
21.6.20.3. Net Present Value
21.6.20.4. Value Creation Analysis
21.6.21. ProstAtak®
21.6.21.1. Target Patient Population
21.6.21.2. Sales Forecast
21.6.21.3. Net Present Value
21.6.21.4. Value Creation Analysis
21.6.22. D-Fi
21.6.22.1. Target Patient Population
21.6.22.2. Sales Forecast
21.6.22.3. Net Present Value
21.6.22.4. Value Creation Analysis
21.6.23. CG0070
21.6.23.1. Target Patient Population
21.6.23.2. Sales Forecast
21.6.23.3. Net Present Value
21.6.23.4. Value Creation Analysis
21.6.24. Vigil™-EWS
21.6.24.1. Target Patient Population
21.6.24.2. Sales Forecast
21.6.24.3. Net Present Value
21.6.24.4. Value Creation Analysis
21.6.25. Engensis®
21.6.25.1. Target Patient Population
21.6.25.2. Sales Forecast
21.6.25.3. Net Present Value
21.6.25.4. Value Creation Analysis
21.6.26. VGX-3100
21.6.26.1. Target Patient Population
21.6.26.2. Sales Forecast
21.6. 26.3. Net Present Value
21.6.26.4. Value Creation Analysis
21.6.27. Invossa™ (TG-C)
21.6.27.1. Target Patient Population
21.6.27.2. Sales Forecast
21.6.27.3. Net Present Value
21.6.27.4. Value Creation Analysis
21.6.28. VYJUVEKT™
21.6.28.1. Target Patient Population
21.6.28.2. Sales Forecast
21.6.28.3. Net Present Value
21.6.28.4. Value Creation Analysis
21.6.29. PF-06939926
21.6.29.1. Target Patient Population
21.6.29.2. Sales Forecast
21.6.29.3. Net Present Value
21.6.29.4. Value Creation Analysis
21.6.30. PF-06838435
21.6.30.1. Target Patient Population
21.6.30.2. Sales Forecast
21.6.30.3. Net Present Value
21.6.30.4. Value Creation Analysis
21.6.31. PF-07055480
21.6.31.1. Target Patient Population
21.6.31.2. Sales Forecast
21.6.31.3. Net Present Value
21.6.31.4. Value Creation Analysis
21.6.32. SPK-8011
21.6.32.1. Target Patient Population
21.6.32.2. Sales Forecast
21.6.32.3. Net Present Value
21.6.32.4. Value Creation Analysis
21.6.33. AMT-061
21.6.33.1. Target Patient Population
21.6.33.2. Sales Forecast
21.6.33.3. Net Present Value
21.6.33.4. Value Creation Analysis
21.6.34. VB-111
21.6.34.1. Target Patient Population
21.6.34.2. Sales Forecast
21.6.34.3. Net Present Value
21.6.34.4. Value Creation Analysis
21.6.35. Generx®
21.6.35.1. Target Patient Population
21.6.35.2. Sales Forecast
21.6.35.3. Net Present Value
21.6.35.4. Value Creation Analysis
21.6.36. AMG001
21.6.36.1. Target Patient Population
21.6.36.2. Sales Forecast
21.6.36.3. Net Present Value
21.6.36.4. Value Creation Analysis
21.6.37. OAV-101
21.6.37.1. Target Patient Population
21.6.37.2. Sales Forecast
21.6.37.3. Net Present Value
21.6.37.4. Value Creation Analysis
21.6.38. ADXS-HPV
21.6.38.1. Target Patient Population
21.6.38.2. Sales Forecast
21.6.38.3. Net Present Value
21.6.38.4. Value Creation Analysis
21.6.39. AGTC 501
21.6.39.1. Target Patient Population
21.6.39.2. Sales Forecast
21.6.39.3. Net Present Value
21.6.39.4. Value Creation Analysis
21.6.40. LYS-SAF302
21.6.40.1. Target Patient Population
21.6.40.2. Sales Forecast
21.6.40.3. Net Present Value
21.6.40.4. Value Creation Analysis
21.6.41. NFS-01
21.6.41.1. Target Patient Population
21.6.41.2. Sales Forecast
21.6.41.3. Net Present Value
21.6.41.4. Value Creation Analysis
21.6.42. AG0302-COVID19
21.6.42.1. Target Patient Population
21.6.42.2. Sales Forecast
21.6.42.3. Net Present Value
21.6.42.4. Value Creation Analysis
21.6.43. RGX-314
21.6.43.1. Target Patient Population
21.6.43.2. Sales Forecast
21.6.43.3. Net Present Value
21.6.43.4. Value Creation Analysis
21.6.44. Hologene 5
21.6.44.1. Target Patient Population
21.6.44.2. Sales Forecast
21.6.44.3. Net Present Value
21.6.44.4. Value Creation Analysis
22. VECTOR MANUFACTURING
22.1. Chapter Overview
22.2. Overview of Viral Vector Manufacturing
22.3. Viral Vector Manufacturing Process
22.3.1. Mode of Vector Production
22.3.2. Adherent and Suspension Cultures
22.3.3. Unit Processes and Multiple Parallel Processes
22.3.4. Cell Culture Systems for Production of Viral Vectors
22.3.4.1. Small Scale/Laboratory Scale Cell Culture Systems
22.3.4.2. Large Scale Cell Culture Systems
22.3.5. Culture Media Specifications
22.4. Bioprocessing of Viral Vectors
22.4.1. AAV Vector Production
22.4.2. Adenoviral Vector Production
22.4.3. Lentiviral Vector Production
22.4.4. y-Retroviral Vector Production
22.5. Challenges Associated with Vector Manufacturing
22.6. Contract Service Providers for Viral and Plasmid Vectors
23. CASE STUDY: GENE THERAPY SUPPLY CHAIN
23.1. Chapter Overview
23.2. Overview of Gene Therapy Supply Chain
23.3. Implementation of Supply Chain Models
23.4. Logistics in Gene Therapies
23.4.1. Logistic Processes for Autologous and Allogeneic Therapies
23.5. Regulatory Supply Chain Across the Globe
23.6. Challenges Associated with Gene Therapy Supply Chain
23.7. Optimizing Cell and Advanced Therapies Supply Chain Management
23.7.1. Enterprise Manufacturing System
23.7.2. Laboratory Information Management System
23.7.3. Inventory Management System
23.7.4. Quality Management System
23.7.5. Logistics Management System
23.7.6. Patient Management System
23.7.7. Electronic Clinical Outcome Assessments System
23.7.8. Supply Chain Orchestration Platform
23.8. Recent Developments and Upcoming Trends
24. CONCLUSION
24.1. Chapter Overview
25. INTERVIEW TRANSCRIPTS
25.1. Chapter Overview
25.2. Buel Dan Rodgers (Founder and CEO, AAVogen)
25.3. Sue Washer (President and CEO, AGTC)
25.4. Patricia Zilliox (President and CEO, Eyevensys)
25.5. Christopher Reinhard (CEO and Chairman, Gene Biotherapeutics (previously known as Cardium Therapeutics))
25.6. Adam Rogers (CEO, Hemera Biosciences)
25.7. Ryo Kubota (CEO, Chairman and President, Kubota Pharmaceutical Holdings (Acucela))
25.8. Al Hawkins (CEO, Milo Biotechnology)
25.9. Jean-Phillipe Combal (CEO, Vivet Therapeutics)
25.10. Robert Jan Lamers (former CEO, Arthrogen)
25.11. Tom Wilton (former CBO, LogicBio Therapeutics)
25.12. Michael Tripletti (former CEO, Myonexus Therapeutics)
25.13. Molly Cameron (former Corporate Communications Manager, Orchard Therapeutics)
25.14. Cedric Szpirer (former Executive and Scientific Director, Delphi Genetics)
25.15. Marco Schmeer (Project Manager) and Tatjana Buchholz, (former Marketing Manager, PlasmidFactory)
25.16. Jeffrey Hung (CCO, Vigene Biosciences)
26. APPENDIX 1: TABULATED DATA
27. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS
List Of Figures
Figure 2.1 Executive Summary: Overall Market Landscape of Gene Therapies
Figure 2.2 Executive Summary: Competitive Landscape
Figure 2.3 Executive Summary: Key Therapeutic Areas
Figure 2.4 Executive Summary: Patent Analysis
Figure 2.5 Executive Summary: Mergers and Acquisitions
Figure 2.6 Executive Summary: Funding and Investment Analysis
Figure 2.7 Executive Summary: Clinical Trial Analysis
Figure 2.8 Executive Summary: Demand Analysis
Figure 2.9 Executive Summary: Market Forecast and Opportunity Analysis
Figure 3.1 Evolution of Gene Therapies
Figure 3.2 Ex Vivo Gene Therapies
Figure 3.3 In Vivo Gene Therapies
Figure 3.4 Mechanism of Action of Gene Therapies
Figure 3.5 Genome Editing: Historical Milestones
Figure 3.6 Genome Editing Techniques
Figure 3.7 Advantages and Disadvantages of Gene Therapies
Figure 4.1 Gene Transfer: Viral and Non-Viral Methods
Figure 5.1 Centralized Regulatory Pathway for Market Approval in the US
Figure 5.2 Centralized Regulatory Pathway for Market Approval in Europe
Figure 5.3 Manufacturing Requirements for Gene Therapy Products in China
Figure 5.4 Key Elements of Quality Control in China
Figure 5.5 Centralized Regulatory Pathway for Market Approval in Japan
Figure 6.1 Gene Therapies: Distribution by Stage of Development
Figure 6.2 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Phase of Development
Figure 6.3 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Therapeutic Area
Figure 6.4 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Phase of Development and Therapeutic Area
Figure 6.5 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Vector Used
Figure 6.6 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Gene/Molecule Targeted
Figure 6.7 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Therapy
Figure 6.8 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Vector Used and Type of Therapy
Figure 6.9 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Gene Delivery Method Used
Figure 6.10 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Route of Administration
Figure 6.11 Early-Stage Pipeline of Gene Therapies: Distribution by Phase of Development
Figure 6.12 Early-Stage Pipeline of Gene Therapies: Distribution by Therapeutic Area
Figure 6.13 Early-Stage Pipeline of Gene Therapies: Distribution by Phase of Development and Therapeutic Area
Figure 6.14 Early-Stage Pipeline of Gene Therapies: Distribution by Type of Vector Used
Figure 6.15 Early-Stage Pipeline of Gene Therapies: Distribution by Type of Therapy
Figure 6.16 Early-Stage Pipeline of Gene Therapies: Distribution by Type of Vector Used and Type of Therapy
Figure 6.17 Early-Stage Pipeline of Gene Therapies: Distribution by Type of Gene Delivery Method Used
Figure 6.18 Early-Stage Pipeline of Gene Therapies: Distribution by Route of Administration
Figure 6.19 Gene Therapies: Distribution by Special Designation(s) Awarded
Figure 6.20 Gene Therapies: Distribution by Phase of Development, Therapeutic Area and Type of Therapy (Grid Representation)
Figure 7.1 Gene Therapy Developers: Distribution by Year of Establishment
Figure 7.2 Gene Therapy Developers: Distribution by Company Size
Figure 7.3 Gene Therapy Developers: Distribution by Location of Headquarters
Figure 7.4 Gene Therapy Developers: Distribution by Year of Establishment, Company Size and Location of Headquarters
Figure 7.5 Gene Therapy Developers: Distribution of Players based in North America
Figure 7.6 Gene Therapy Developers: Distribution of Players based in Europe
Figure 7.7 Gene Therapy Developers: Distribution of Players based in Asia-Pacific
Figure 7.8 Gene Therapy Developers: Distribution of Players based in Middle East and North Africa
Figure 7.9 Key Players: Distribution by Number of Gene Therapies
Figure 7.10 Key Players: Distribution by Number of Gene Therapies and Phase of Development
Figure 8.1 Gendicine®: Development Timeline
Figure 8.2 Oncorine®: Development Timeline
Figure 8.3 Oncorine®: Mechanism of Action
Figure 8.4 Rexin-G®: Development Timeline
Figure 8.5 Neovasculgen®: Development Timeline
Figure 8.6 Imlygic®: Development Timeline
Figure 8.7 Imlygic®: Mechanism of Action
Figure 8.8 Strimvelis®: Development Timeline
Figure 8.9 Luxturna™: Development Timeline
Figure 8.10 Zolgensma™: Development Timeline
Figure 8.11 Collategene®: Development Timeline
Figure 8.12 Zyntelgo™: Development Timeline
Figure 8.13 Libmeldy™: Development Timeline
Figure 9.1 Successful Drug Launch Strategy: ROOTS Framework
Figure 9.2 Successful Drug Launch Strategy: Product Differentiation
Figure 9.3 Commonly Adopted Commercialization Strategies based on Phase of Development of Product
Figure 9.4 Harvey Ball Analysis: Key Commercialization Strategies Adopted by Gene Therapy Developers
Figure 9.5 Approved Gene Therapies: Historical Timeline of Geographical Expansion
Figure 9.6 Approved Gene Therapies: Snapshot of Promotional Activities on Product Websites
Figure 9.7 Key Commercialization Strategies: Harvey Ball Analysis by Ease of Implementation, Value Addition and Current Adoption
Figure 11.1 Gene Editing: Key Application Areas
Figure 11.2 Gene Editing: Emerging Technology Platforms
Figure 12.1 Gene Therapies: Distribution by Therapeutic Area and Special Designation(s) Awarded
Figure 12.2 Gene Therapies for Oncological Diseases: Distribution by Target Indication and Phase of Development
Figure 12.3 Gene Therapies for Oncological Diseases: Distribution by Type of Vector Used
Figure 12.4 Gene Therapies for Neurological Diseases: Distribution by Target Indication and Phase of Development
Figure 12.5 Gene Therapies for Neurological Diseases: Distribution by Type of Vector Used
Figure 12.6 Gene Therapies for Ophthalmic Diseases: Distribution by Target Indication and Phase of Development
Figure 12.7 Gene Therapies for Ophthalmic Diseases: Distribution by Type of Vector Used
Figure 12.8 Gene Therapies for Metabolic Diseases: Distribution by Target Indication and Phase of Development
Figure 12.9 Gene Therapies for Metabolic Diseases: Distribution by Type of Vector Used
Figure 12.10 Gene Therapies for Genetic Diseases: Distribution by Target Indication and Phase of Development
Figure 12.11 Gene Therapies for Genetic Diseases: Distribution by Type of Vector Used
Figure 13.1 Gene Therapy Patent Portfolio: Distribution by Type of Patent
Figure 13.2 Gene Therapy Patent Portfolio: Cumulative Distribution by Publication Year, 2017-2022
Figure 13.3 Gene Therapy Patent Portfolio: Distribution by Type of Patent and Publication Year
Figure 13.4 Gene Therapy Patent Portfolio: Distribution by Geography
Figure 13.5 Gene Therapy Patent Portfolio: North America Scenario
Figure 13.6 Gene Therapy Patent Portfolio: Europe Scenario
Figure 13.7 Gene Therapy Patent Portfolio: Asia-Pacific Scenario
Figure 13.8 Gene Therapy Patent Portfolio: Distribution by CPC Symbols
Figure 13.9 Gene Therapy Patent Portfolio: Emerging Focus Areas
Figure 13.10 Leading Industry Players (Gene Therapies): Distribution by Number of Patents
Figure 13.11 Gene Therapy Patent Portfolio (Sanofi and Genethon): Benchmarking by Patent Characteristics
Figure 13.12 Gene Therapy Patent Portfolio (Other Leading Industry Players): Benchmarking by Patent Characteristics
Figure 13.13 Gene Therapy Patent Portfolio: Distribution by Patent Age (2017-2022)
Figure 13.14 Gene Therapy Patent Portfolio: Valuation Analysis
Figure 13.15 Gene Editing Patent Portfolio: Distribution by Type of Patent
Figure 13.16 Gene Editing Patent Portfolio: Cumulative Distribution by Publication Year, 2017-2022
Figure 13.17 Gene Editing Patent Portfolio: Distribution by Type of Patent and Publication Year
Figure 13.18 Gene Editing Patent Portfolio: Distribution by Geography
Figure 13.19 Gene Editing Patent Portfolio: North America Scenario
Figure 13.20 Gene Editing Patent Portfolio: Europe Scenario
Figure 13.21 Gene Editing Patent Portfolio: Asia-Pacific Scenario
Figure 13.22 Gene Editing Patent Portfolio: Distribution by CPC Symbols
Figure 13.23 Gene Editing Patent Portfolio: Emerging Focus Areas
Figure 13.24 Leading Industry Players (Gene Editing): Distribution by Number of Patents
Figure 13.25 Gene Editing Patent Portfolio (Intellia Therapeutics and CRISPR Therapeutics): Benchmarking by Patent Characteristics
Figure 13.26 Gene Editing Patent Portfolio (Other Leading Industry Players): Benchmarking by Patent Characteristics
Figure 13.27 Gene Editing Patent Portfolio: Distribution by Patent Age (2017-2022)
Figure 13.28 Gene Editing Patent Portfolio: Valuation Analysis
Figure 13.29 Gene Therapy Patent Portfolio: Cumulative Distribution by Type of Organization
Figure 13.30 Gene Editing Patent Portfolio: Cumulative Distribution by Type of Organization
Figure 14.1 Mergers and Acquisitions: Cumulative Year-Wise Trend, 2015-2022
Figure 14.2 Mergers and Acquisitions: Distribution by Type of Agreement
Figure 14.3 Mergers and Acquisitions: Distribution by Year and Type of Agreement
Figure 14.4 Mergers and Acquisitions: Continent-wise Distribution
Figure 14.5 Mergers and Acquisitions: Intercontinental and Intracontinental Deals
Figure 14.6 Mergers and Acquisitions: Local and International Deals
Figure 14.7 Acquisitions: Distribution by Key Value Drivers
Figure 14.8 Acquisitions: Distribution by Year of Acquisition and Key Value Drivers
Figure 14.9 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company’s Product
Figure 14.10 Mergers and Acquisitions: Distribution by Year of Acquisition and Phase of Development of the Acquired Company’s Product
Figure 14.11 Mergers and Acquisitions: Distribution of Deal Amount by Phase of Development of the Acquired Company’s Product
Figure 14.12 Mergers and Acquisitions: Distribution by Therapeutic Area
Figure 15.1 Funding and Investments: Cumulative Distribution of Instances by Year, 2015-2022
Figure 15.2 Funding and Investments: Cumulative Distribution of Amount Invested by Year, 2015-2022 (USD Million)
Figure 15.3 Funding and Investments: Distribution of Instances by Type of Funding, 2015-2022
Figure 15.4 Funding and Investments: Distribution of Total Amount Invested by Type of Funding, 2015-2022 (USD Million)
Figure 15.5 Funding and Investments: Distribution of Instances by Year and Type of Funding, 2015-2022
Figure 15.6 Funding and Investments: Distribution of Amount Invested by Year and Type of Funding, 2015-2022 (USD Million)
Figure 15.7 Funding and Investments: Summary of Investments, 2015-2022 (USD Million)
Figure 15.8 Funding and Investments: Distribution of Instances and Amount Invested by Type of Therapy
Figure 15.9 Funding and Investments: Distribution of Instances and Amount Invested by Geography
Figure 15.10 Funding and Investments: Regional Distribution of Funding Instances
Figure 15.11 Funding and Investments: Distribution of Instances and Amount Invested by Highest Phase of Development of the Company’s Product
Figure 15.12 Funding and Investments: Distribution of Instances by Therapeutic Area
Figure 15.13 Most Active Players: Distribution by Number of Funding Instances, 2015-2022
Figure 15.14 Most Active Players: Distribution by Amount Invested, 2015-2022 (USD Million)
Figure 15.15 Funding and Investment Analysis: Distribution of Instances by Type of Investors
Figure 15.16 Leading Investors: Distribution by Number of Instances
Figure 15.17 Funding and Investment Summary, 2015-2022 (USD Million)
Figure 16.1 Clinical Trial Analysis: Distribution by Trial Registration Year, 2017-2022
Figure 16.2 Clinical Trial Analysis: Distribution by Trial Status
Figure 16.3 Clinical Trial Analysis: Distribution by Trial Phase
Figure 16.4 Clinical Trial Analysis: Distribution by Therapeutic Area
Figure 16.5 Clinical Trial Analysis: Distribution by Geography
Figure 16.6 Clinical Trial Analysis: Distribution by Trial Registration Year and Geography
Figure 16.7 Clinical Trial Analysis: Distribution by Trial Status and Geography
Figure 16.8 Clinical Trial Analysis: Distribution by Trial Status, Trial Phase and Geography
Figure 16.9 Clinical Trial Analysis: Distribution by Therapeutic Area and Geography
Figure 16.10 Clinical Trial Analysis: Distribution by Type of Sponsor
Figure 16.11 Clinical Trial Analysis: Distribution by Prominent Treatment Sites
Figure 16.12 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Registration Year, 2017-2022
Figure 16.13 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status
Figure 16.14 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Phase
Figure 16.15 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Therapeutic Area
Figure 16.16 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Location of Trial Site
Figure 16.17 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status and Location of Trial Site
Figure 16.18 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status, Trial Phase and Location of Trial Site
Figure 16.19 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Therapeutic Area and Location of Trial Site
Figure 17.1 Gene Therapy: Pricing Model Based on Patient Segment
Figure 18.1 Start-up Valuation based on Experience of the Gene Therapy Developer
Figure 19.1 Big Pharma Players: Top Pharmaceutical Companies (Heat Map Representation)
Figure 19.2 Big Pharma Players: Analysis by Therapeutic Area
Figure 19.3 Big Pharma Players: Analysis by Type of Vector Used
Figure 19.4 Big Pharma Players: Analysis by Type of Therapy
Figure 19.5 Big Pharma Players: Analysis by Type of Gene Delivery Method Used
Figure 20.1 Global Demand for Gene Therapies, 2022-2035
Figure 20.2 Demand Analysis: Distribution by Type of Therapy, 2022, 2030 and 2035
Figure 20.3 Demand Analysis: Distribution by Therapeutic Area, 2022, 2030 and 2035
Figure 20.4 Demand Analysis: Distribution by Geography, 2022, 2030 and 2035
Figure 20.5 Demand Analysis: Country-wise Distribution (Asia-Pacific), 2022, 2030 and 2035
Figure 21.1 Global Gene Therapy Market, 2022-2035 (USD Billion)
Figure 21.2 Gene Therapy Market: Distribution by Therapeutic Area, 2022, 2030 and 2035
Figure 21.3 Gene Therapy Market: Distribution by Type of Vector Used, 2022, 2030 and 2035
Figure 21.4 Gene Therapy Market: Distribution by Type of Therapy, 2022, 2030 and 2035
Figure 21.5 Gene Therapy Market: Distribution by Type of Gene Delivery Method Used, 2022, 2030 and 2035
Figure 21.6 Gene Therapy Market: Distribution by Route of Administration, 2022, 2030 and 2035
Figure 21.7 Gene Therapy Market: Distribution by Geography, 2022, 2030 and 2035
Figure 21.8 Gene Therapy Market: Country-wise Distribution (Asia-Pacific), 2022, 2030 and 2035
Figure 21. 9 Gendicine® Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.10 Oncorine® Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.11 Rexin-G® Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.12 Neovasculgen® Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.13 Strimvelis® Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.14 Imlygic® Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.15 Luxturna™ Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.16 Zolgensma™ Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.17 Collategene® Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.18 Libmeldy™ Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.19 Lumevaq® (GS010) Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.20 OTL-103 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.21 PTC-AADC Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.22 BMN 270 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.23 rAd-IFN/Syn3 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.24 beti-cel Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.25 eli-cel Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.26 lovo-cel Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.27 SRP-9001 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.28 EB-101 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.29 ProstAtak® Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.30 D-Fi Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.31 CG0070 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.32 Vigil™-EWS Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.33 Engensis® Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.34 VGX-3100 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.35 Invossa™ (TG-C) Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.36 VYJUVEKT™ Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.37 PF-06939926 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.38 PF-06838435 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.39 PF-07055480 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.40 SPK-8011 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.41 AMT-061 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.42 VB-111 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.43 Generx® Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.44 AMG001 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.45 OAV-101 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.46 ADXS-HPV Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.47 AGTC 501 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.48 LYS-SAF302 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.49 NFS-01 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.50 AG0302-COVID19 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.51 RGX-314 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.52 Hologene 5 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 22.1 Viral Vectors: Key Manufacturing Steps
Figure 23.1 Steps Involved in the Development of Gene Therapies
Figure 23.2 Overall Development Process of Cell-based Gene Therapies
Figure 23.3 Key Responsibilities of Stakeholders Involved in Advanced Therapies Supply Chain
Figure 23.4 Key Steps Involved in the Implementation of a Supply Chain Strategy
Figure 23.5 Keys Steps Involved in the Logistics Process of Autologous Therapies
Figure 23.6 Comparison of Logistics Processes for Autologous and Allogeneic Therapies
Figure 23.7 Global Regulatory Authorities for Gene Therapy Supply Chain
Figure 23.8 Overview of Supply Chain Orchestration Platform
Figure 23.9 Functions of Key Technological Solutions for Cell and Advanced Therapies Supply Chain Management
Figure 24.1 Concluding Remarks: Overall Market Overview
Figure 24.2 Concluding Remarks: Patent Analysis
Figure 24.3 Concluding Remarks: Mergers and Acquisitions
Figure 24.4 Concluding Remarks: Funding and Investments
Figure 24.5 Concluding Remarks: Clinical Trials Analysis
Figure 24.6 Concluding Remarks: Big Pharmaceutical Gene Therapy Related Initiatives
Figure 24.7 Concluding Remarks: Market Forecast and Opportunity Analysis
Figure 24.8 Concluding Remarks: Market Forecast and Opportunity Analysis
Table 3.1 Comparison of Ex Vivo and In Vivo Techniques
Table 3.2 Vectors Used for Targeted Gene Delivery to Tissues/Organs
Table 3.3 Routes of Administration of Gene Therapies: Advantages and Disadvantages
Table 4.1 Viral Vectors: Key Features
Table 5.1 Approved Advanced Therapies Medicinal Products (ATMPs) and their Reimbursement Status in Europe
Table 5.2 Payment Options Available for Gene Therapies
Table 6.1 Gene Therapy Market: Clinical and Commercial Pipeline
Table 6.2 Gene Therapy Market: Early-Stage Pipeline
Table 6.3 Gene Therapy Market: Special Designation(s) Awarded
Table 7.1 Gene Therapy Market: List of Developers
Table 8.1 Gene Therapies: Marketed and Approved Products
Table 8.2 Shenzhen Sibiono GeneTech: Company Overview
Table 8.3 Gendicine®: Patent Portfolio
Table 8.4 Gendicine®: Status of Development
Table 8.5 Shanghai Sunway Biotech: Company Overview
Table 8.6 Oncorine®: Status of Development
Table 8.7 Epeius Biotechnologies: Company Overview
Table 8.8 Rexin-G®: Status of Development
Table 8.9 Human Stem Cell Institute: Company Overview
Table 8.10 Neovasculgen®: Status of Development
Table 8.11 Amgen: Company Overview
Table 8.12 Imlygic®: Status of Development
Table 8.13 Imlygic®: Recommended Dose and Schedule
Table 8.14 Imlygic®: Determination of Injection Volume based upon Lesion Size
Table 8.15 Orchard Therapeutics: Company Overview
Table 8.16 Strimvelis®: Status of Development
Table 8.17 Spark Therapeutics: Company Overview
Table 8.18 Luxturna™: Status of Development
Table 8.19 Novartis Gene Therapies: Company Overview
Table 8.20 Zolgensma™: Status of Development
Table 8.21 AnGes: Company Overview
Table 8.22 Collategene®: Status of Development
Table 8.23 bluebird bio: Company Overview
Table 8.24 Zyntelgo™: Status of Development
Table 8.25 Libmeldy™: Status of Development
Table 9.1 Libmeldy™: Presentations in Conferences Before Product Approval
Table 9.2 Luxturna™: Presentations in Conferences Before Product Approval
Table 9.3 Zolgensma™: Presentations in Conferences Before Product Approval
Table 9.4 Zyntelgo™: Presentations in Conferences Before Product Approval
Table 9.5 Imlygic®: Presentations in Conferences After Product Approval
Table 9.6 Libmeldy™: Presentations in Conferences After Product Approval
Table 9.7 Luxturna™: Presentations in Conferences After Product Approval
Table 9.8 Strimvelis™: Presentations in Conferences After Product Approval
Table 9.9 Zolgensma™: Presentations in Conferences After Product Approval
Table 9.10 Zyntelgo™: Presentations in Conferences After Product Approval
Table 9.11 Approved Gene Therapies: Patient Support Services Available on Product Websites (Comparative Analysis)
Table 10.1 Gene Therapies: Late Stage (Phase II/III and Above) Drug Candidates
Table 10.2 Lumevaq® (GS010): Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.3 OTL-103: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.4 PTC-AADC: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.5 BMN 270: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.6 rAd-IFN/Syn3: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.7 beti-cel: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.8 eli-cel: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.9 lovo-cel: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.10 SRP-9001: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.11 EB-101: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.12 ProstAtak®: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.13 D-Fi: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.14 CG0070: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.15 Vigil™-EWS: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.16 Engensis®: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.17 VGX-3100: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.18 Invossa™ (TG-C): Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.19 VYJUVEKT™: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.20 PF-06939926: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.21 PF-06838435: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.22 PF-07055480: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.23 SPK-8011: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.24 AMT-061: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.25 VB-111: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.26 Generx®: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.27 ADXS-HPV: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.28 AGTC 501: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.29 LYS-SAF302: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.30 NFS-01: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.31 AG0302-COVID19: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.32 RGX-314: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 10.32 Hologene 5: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 11.1 Gene Editing Technology Platforms
Table 11.2 ASC Therapeutics: Collaborations
Table 11.3 CRISPR Therapeutics: Funding Instances
Table 11.4 CRISPR Therapeutics: Collaborations
Table 11.5 Editas Medicine: Funding Instances
Table 11.6 Editas Medicine: Collaborations
Table 11.7 Emendo Biotherapeutics: Collaborations
Table 11.8 Excision Biotherapeutics: Funding Instances
Table 11.9 Intellia Therapeutics: Funding Instances
Table 11.10 Intellia Therapeutics: Collaborations
Table 11.11 Sarepta Therapeutics: Funding Instances
Table 11.12 Sarepta Therapeutics: Collaborations
Table 11.13 ToolGen: Collaborations
Table 11.14 bluebird bio: Funding Instances
Table 11.15 bluebird bio: Collaborations
Table 11.16 Sangamo Therapeutics: Funding Instances
Table 11.17 Sangamo Therapeutics: Collaborations
Table 11.18 Gene Switch Technology Platforms
Table 11.19 Precigen: Funding Instances
Table 11.20 Precigen: Collaborations
Table 11.21 MeiraGTx: Funding Instances
Table 11.22 MeiraGTx: Collaborations
Table 11.23 Gene Therapy: Technology Platforms
Table 12.1 Oncological Diseases: Information on Highest Phase of Development of Gene Therapy and Type of Vector Used
Table 12.2 Neurological Diseases: Information on Highest Phase of Development of Gene Therapy and Type of Vector Used
Table 12.3 Ophthalmic Diseases: Information on Highest Phase of Development of Gene Therapy and Type of Vector Used
Table 12.4 Metabolic Diseases: Information on Highest Phase of Development of Gene Therapy and Type of Vector Used
Table 12.5 Genetic Diseases: Information on Highest Phase of Development of Gene Therapy and Type of Vector Used
Table 13.1 Gene Therapy Patent Portfolio: List of Top CPC Symbols
Table 13.2 Gene Therapy Patent Portfolio: Summary of Benchmarking Analysis
Table 13.3 Gene Therapy Patent Portfolio: Categorizations based on Weighted Valuation Scores
Table 13.4 Gene Therapy Patent Portfolio: List of Leading Patents (in terms of Highest Relative Valuation)
Table 13.5 Gene Editing Patent Portfolio: List of Top CPC Symbols
Table 13.6 Gene Editing Patent Portfolio: Summary of Benchmarking Analysis
Table 13.7 Gene Editing Patent Portfolio: Categorizations based on Weighted Valuation Scores
Table 13.8 Gene Editing Patent Portfolio: List of Leading Patents (in terms of Highest Relative Valuation)
Table 14.1 Gene Therapy: List of Mergers and Acquisitions, 2015-2022
Table 14.2 Acquisitions: Key Value Drivers
Table 14.3 Mergers and Acquisitions: Information on Phase of Development and Therapeutic Area of the Acquired Company’s Gene Therapy Product
Table 14.4 Mergers and Acquisitions: Information on Deal Multiple based on Acquisition Amount
Table 15.1 Gene Therapy Market: Funding and Investments, 2015-2022
Table 15.2 Funding and Investments: Summary of Investments
Table 15.3 Funding and Investments: Summary of Venture Capital Funding
Table 16.1 Clinical Trial Analysis: Emerging Molecules to Watch
Table 16.2 Clinical Trial Analysis: Most Important Trials to Watch
Table 17.1 Pricing Model: Price of Marketed Gene/Cell Therapies
Table 17.2 Pricing Model: Price of Marketed Targeted Drugs (Antibody Drug Conjugates and Monoclonal Antibodies)
Table 17.3 Pricing Model: Opinions of Experts/Other Analysts
Table 18.1 Start-up Valuation: Estimates based on Overall Experience
Table 21.1 Gene Therapies: Expected Launch Years of Advanced Stage Drug Candidates
Table 21.2 Gendicine®: Target Patient Population
Table 21.3 Gendicine®: Net Present Value (USD Million)
Table 21.4 Gendicine®: Value Creation Analysis (USD Million)
Table 21.5 Oncorine®: Target Patient Population
Table 21.6 Oncorine®: Net Present Value (USD Million)
Table 21.7 Oncorine®: Value Creation Analysis (USD Million)
Table 21.8 Rexin-G®: Target Patient Population
Table 21.9 Rexin-G®: Net Present Value (USD Million)
Table 21.10 Rexin-G®: Value Creation Analysis (USD Million)
Table 21.11 Neovasculgen®: Target Patient Population
Table 21.12 Neovasculgen®: Net Present Value (USD Million)
Table 21.13 Neovasculgen®: Value Creation Analysis (USD Million)
Table 21.14 Strimvelis®: Target Patient Population
Table 21.15 Strimvelis®: Net Present Value (USD Million)
Table 21.16 Strimvelis®: Value Creation Analysis (USD Million)
Table 21.17 Imlygic®: Target Patient Population
Table 21.18 Imlygic®: Net Present Value (USD Million)
Table 21.19 Imlygic®: Value Creation Analysis (USD Million)
Table 21.20 Luxturna™: Target Patient Population
Table 21.21 Luxturna™: Net Present Value (USD Million)
Table 21.22 Luxturna™: Value Creation Analysis (USD Million)
Table 21.23 Zolgensma™: Target Patient Population
Table 21.24 Zolgensma™: Net Present Value (USD Million)
Table 21.25 Zolgensma™: Value Creation Analysis (USD Million)
Table 21.26 Collategene®: Target Patient Population
Table 21.27 Collategene®: Net Present Value (USD Million)
Table 21.28 Collategene®: Value Creation Analysis (USD Million)
Table 21.29 Libmedly™: Target Patient Population
Table 21.30 Libmedly™: Net Present Value (USD Million)
Table 21.31 Libmedly™: Value Creation Analysis (USD Million)
Table 21.32 Lumevaq® (GS010): Target Patient Population
Table 21.33 Lumevaq® (GS010): Net Present Value (USD Million)
Table 21.34 Lumevaq® (GS010): Value Creation Analysis (USD Million)
Table 21.35 OTL-103: Target Patient Population
Table 21.36 OTL-103: Net Present Value (USD Million)
Table 21.37 OTL-103: Value Creation Analysis (USD Million
Table 21.38 PTC-AADC: Target Patient Population
Table 21.39 PTC-AADC: Net Present Value (USD Million)
Table 21.40 PTC-AADC: Value Creation Analysis (USD Million)
Table 21.41 BMN 270: Target Patient Population
Table 21.42 BMN 270: Net Present Value (USD Million)
Table 21.43 BMN 270: Value Creation Analysis (USD Million)
Table 21.44 rAd-IFN/Syn3: Target Patient Population
Table 21.45 rAd-IFN/Syn3: Net Present Value (USD Million)
Table 21.46 rAd-IFN/Syn3: Value Creation Analysis (USD Million)
Table 21.47 beti-cel: Target Patient Population
Table 21.48 beti-cel: Net Present Value (USD Million)
Table 21.49 beti-cel: Value Creation Analysis (USD Million)
Table 21.50 eli-cel: Target Patient Population
Table 21.51 eli-cel: Net Present Value (USD Million)
Table 21.52 eli-cel: Value Creation Analysis (USD Million)
Table 21.53 lovo-cel: Target Patient Population
Table 21.54 lovo-cel: Net Present Value (USD Million)
Table 21.55 lovo-cel: Value Creation Analysis (USD Million)
Table 21.56 SRP-9001: Target Patient Population
Table 21.57 SRP-9001: Net Present Value (USD Million)
Table 21.58 SRP-9001: Value Creation Analysis (USD Million)
Table 21.59 EB-101: Target Patient Population
Table 21.60 EB-101: Net Present Value (USD Million)
Table 21.61 EB-101: Value Creation Analysis (USD Million)
Table 21.62 ProstAtak®: Target Patient Population
Table 21.63 ProstAtak®: Net Present Value (USD Million)
Table 21.64 ProstAtak®: Value Creation Analysis (USD Million)
Table 21.65 D-Fi: Target Patient Population
Table 21.66 D-Fi: Net Present Value (USD Million)
Table 21.67 D-Fi: Value Creation Analysis (USD Million)
Table 21.68 CG0070: Target Patient Population
Table 21.69 CG0070: Net Present Value (USD Million)
Table 21.70 CG0070: Value Creation Analysis (USD Million)
Table 21.71 Vigil™-EWS: Target Patient Population
Table 21.72 Vigil™-EWS: Net Present Value (USD Million)
Table 21.73 Vigil™-EWS: Value Creation Analysis (USD Million)
Table 21.74 Engensis®: Target Patient Population
Table 21.75 Engensis®: Net Present Value (USD Million)
Table 21.76 Engensis®: Value Creation Analysis (USD Million)
Table 21.77 VGX-3100: Target Patient Population
Table 21.78 VGX-3100: Net Present Value (USD Million)
Table 21.79 VGX-3100: Value Creation Analysis (USD Million)
Table 21.80 Invossa™ (TG-C): Target Patient Population
Table 21.81 Invossa™ (TG-C): Net Present Value (USD Million)
Table 21.82 Invossa™ (TG-C): Value Creation Analysis (USD Million)
Table 21.83 VYJUVEKT™: Target Patient Population
Table 21.84 VYJUVEKT™: Net Present Value (USD Million)
Table 21.85 VYJUVEKT™: Value Creation Analysis (USD Million)
Table 21.86 PF-06939926: Target Patient Population
Table 21.87 PF-06939926: Net Present Value (USD Million)
Table 21.88 PF-06939926: Value Creation Analysis (USD Million)
Table 21.89 PF-06838435: Target Patient Population
Table 21.90 PF-06838435: Net Present Value (USD Million)
Table 21.91 PF-06838435: Value Creation Analysis (USD Million)
Table 21.92 PF-07055480: Target Patient Population
Table 21.93 PF-07055480: Net Present Value (USD Million)
Table 21.94 PF-07055480: Value Creation Analysis (USD Million)
Table 21.95 SPK-8011: Target Patient Population
Table 21.96 SPK-8011: Net Present Value (USD Million)
Table 21.97 SPK-8011: Value Creation Analysis (USD Million)
Table 21.98 AMT-061: Target Patient Population
Table 21.99 AMT-061: Net Present Value (USD Million)
Table 21.100 AMT-061: Value Creation Analysis (USD Million)
Table 21.101 VB-111: Target Patient Population
Table 21.102 VB-111: Net Present Value (USD Million)
Table 21.103 VB-111: Value Creation Analysis (USD Million)
Table 21.104 Generx®: Target Patient Population
Table 21.105 Generx®: Net Present Value (USD Million)
Table 21.106 Generx®: Value Creation Analysis (USD Million)
Table 21.107 AMG001: Target Patient Population
Table 21.108 AMG001: Net Present Value (USD Million)
Table 21.109 AMG001: Value Creation Analysis (USD Million)
Table 21.110 OAV-101: Target Patient Population
Table 21.111 OAV-101: Net Present Value (USD Million)
Table 21.112 OAV-101: Value Creation Analysis (USD Million)
Table 21.113 ADXS-HPV: Target Patient Population
Table 21.114 ADXS-HPV: Net Present Value (USD Million)
Table 21.115 ADXS-HPV: Value Creation Analysis (USD Million)
Table 21.116 AGTC 501: Target Patient Population
Table 21.117 AGTC 501: Net Present Value (USD Million)
Table 21.118 AGTC 501: Value Creation Analysis (USD Million)
Table 21.119 LYS-SAF302: Target Patient Population
Table 21.120 LYS-SAF302: Net Present Value (USD Million)
Table 21.121 LYS-SAF302: Value Creation Analysis (USD Million)
Table 21.122 NFS-01: Target Patient Population
Table 21.123 NFS-01: Net Present Value (USD Million)
Table 21.124 NFS-01: Value Creation Analysis (USD Million)
Table 21.125 AG0302-COVID19: Target Patient Population
Table 21.126 AG0302-COVID19: Net Present Value (USD Million)
Table 21.127 AG0302-COVID19: Value Creation Analysis (USD Million)
Table 21.128 RGX-314: Target Patient Population
Table 21.129 RGX-314: Net Present Value (USD Million)
Table 21.130 RGX-314: Value Creation Analysis (USD Million)
Table 21.131 Hologene 5: Target Patient Population
Table 21.132 Hologene 5: Net Present Value (USD Million)
Table 21.133 Hologene 5: Value Creation Analysis (USD Million)
Table 22.1 Small-scale Cell Culture Systems
Table 22.2 List of Contract Manufacturing Service Providers for Viral Vectors and Plasmid DNA
Table 26.1 Gene Therapies: Distribution by Stage of Development
Table 26.2 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Phase of Development
Table 26.3 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Therapeutic Area
Table 26.4 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Phase of Development and Therapeutic Area
Table 26.5 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Vector Used
Table 26.6 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Gene/Molecule Targeted
Table 26.7 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Therapy
Table 26.8 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Vector Used and Type of Therapy
Table 26.9 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Gene Delivery Method Used
Table 26.10 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Route of Administration
Table 26.11 Early-Stage Pipeline of Gene Therapies: Distribution by Phase of Development
Table 26.12 Early-Stage Pipeline of Gene Therapies: Distribution by Therapeutic Area
Table 26.13 Early-Stage Pipeline of Gene Therapies: Distribution by Phase of Development and Therapeutic Area
Table 26.14 Early-Stage Pipeline of Gene Therapies: Distribution by Type of Vector Used
Table 26.15 Early-Stage Pipeline of Gene Therapies: Distribution by Type of Therapy
Table 26.16 Early-Stage Pipeline of Gene Therapies: Distribution by Type of Vector Used and Type of Therapy
Table 26.17 Early-Stage Pipeline of Gene Therapies: Distribution by Type of Gene Delivery Method Used
Table 26.18 Early-Stage Pipeline of Gene Therapies: Distribution by Route of Administration
Table 26.19 Gene Therapies: Distribution by Special Designation(s) Awarded
Table 26.20 Gene Therapy Developers: Distribution by Year of Establishment
Table 26.21 Gene Therapy Developers: Distribution by Company Size
Table 26.22 Gene Therapy Developers: Distribution by Location of Headquarters
Table 26.23 Gene Therapy Developers: Distribution by Year of Establishment, Company Size and Location of Headquarters
Table 26.24 Most Active Players: Distribution by Number of Gene Therapies
Table 26.25 Most Active Players: Distribution by Number of Gene Therapies and Phase of Development
Table 26.26 Gene Therapies: Distribution by Therapeutic Area and Special Designation(s) Awarded
Table 26.27 Gene Therapies for Oncological Diseases: Distribution by Target Indication and Phase of Development
Table 26.28 Gene Therapies for Oncological Diseases: Distribution by Type of Vector Used
Table 26.29 Gene Therapies for Neurological Diseases: Distribution by Target Indication and Phase of Development
Table 26.30 Gene Therapies for Neurological Diseases: Distribution by Type of Vector Used
Table 26.31 Gene Therapies for Ophthalmic Diseases: Distribution by Target Indication and Phase of Development
Table 26.32 Gene Therapies for Ophthalmic Diseases: Distribution by Type of Vector Used
Table 26.33 Gene Therapies for Metabolic Diseases: Distribution by Target Indication and Phase of Development
Table 26.34 Gene Therapies for Metabolic Diseases: Distribution by Type of Vector Used
Table 26.35 Gene Therapies for Genetic Diseases: Distribution by Target Indication and Phase of Development
Table 26.36 Gene Therapies for Genetic Diseases: Distribution by Type of Vector Used
Table 26.37 Gene Therapy Patent Portfolio: Distribution by Type of Patent
Table 26.38 Gene Therapy Patent Portfolio: Cumulative Distribution by Publication Year, 2017-2022
Table 26.39 Gene Therapy Patent Portfolio: Distribution by Type of Patent and Publication Year
Table 26.40 Gene Therapy Patent Portfolio: Distribution by Geography
Table 26.41 Gene Therapy Patent Portfolio: North America Scenario
Table 26.42 Gene Therapy Patent Portfolio: Europe Scenario
Table 26.43 Gene Therapy Patent Portfolio: Asia-Pacific Scenario
Table 26.44 Gene Therapy Patent Portfolio: Distribution by CPC Symbols
Table 26.45 Gene Therapy Patent Portfolio: Leading Industry Players: Distribution by Number of Patents
Table 26.46 Gene Therapy Patent Portfolio (Sanofi and Genethon): Benchmarking by Patent Characteristics
Table 26.47 Gene Editing Patent Portfolio: Distribution by Type of Patent
Table 26.48 Gene Editing Patent Portfolio: Cumulative Distribution by Publication Year, 2017-2022
Table 26.49 Gene Editing Patent Portfolio: Distribution by Type of Patent and Publication Year
Table 26.50 Gene Editing Patent Portfolio: Distribution by Geography
Table 26.51 Gene Editing Patent Portfolio: North America Scenario
Table 26.52 Gene Editing Patent Portfolio: Europe Scenario
Table 26.53 Gene Editing Patent Portfolio: Asia-Pacific Scenario
Table 26.54 Gene Editing Patent Portfolio: Distribution by CPC Symbols
Table 26.55 Gene Editing Patent Portfolio: Leading Industry Players: Distribution by Number of Patents
Table 26.56 Gene Editing Patent Portfolio (Intellia Therapeutics and CRISPR Therapeutics): Benchmarking by Patent Characteristics
Table 26.57 Mergers and Acquisitions: Cumulative Year-Wise Trend, 2015-2022
Table 26.58 Mergers and Acquisitions: Distribution by Type of Agreement
Table 26.59 Mergers and Acquisitions: Distribution by Year and Type of Agreement
Table 26.60 Mergers and Acquisitions: Intercontinental and Intracontinental Deals
Table 26.61 Mergers and Acquisitions: Local and International Deals
Table 26.62 Acquisitions: Distribution by Key Value Drivers
Table 26.63 Acquisitions: Distribution by Year of Acquisition and Key Value Drivers
Table 26.64 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company’s Product
Table 26.65 Mergers and Acquisitions: Distribution by Year of Acquisition and Phase of Development of the Acquired Company’s Product
Table 26.66 Mergers and Acquisitions: Distribution by Therapeutic Area
Table 26.67 Funding and Investments: Cumulative Distribution of Instances by Year, 2015-2022
Table 26.68 Funding and Investments: Cumulative Distribution of Amount Invested by Year, 2015-2022 (USD Million)
Table 26.69 Funding and Investments: Distribution of Instances by Type of Funding, 2015-2022
Table 26.70 Funding and Investments: Distribution of Total Amount Invested by Type of Funding, 2015-2022 (USD Million)
Table 26.71 Funding and Investments: Summary of Investments, 2015-2022 (USD Million)
Table 26.72 Funding and Investments: Distribution of Instances and Amount Invested by Type of Therapy
Table 26.73 Funding and Investments: Distribution of Instances and Amount Invested by Geography
Table 26.74 Funding and Investments: Regional Distribution of Funding Instances
Table 26.75 Funding and Investments: Distribution of Instances and Amount Invested by Highest Phase of Development of the Company’s Product
Table 26.76 Funding and Investments: Distribution of Instances by Therapeutic Area
Table 26.77 Most Active Players: Distribution by Number of Funding Instances, 2015-2022
Table 26.78 Most Active Players: Distribution by Amount Invested, 2015-2022 (USD Million)
Table 26.79 Funding and Investments: Distribution of Instances by Type of Investors
Table 26.80 Leading Investors: Distribution by Number of Instances
Table 26.81 Funding and Investment Summary, 2015-2022 (USD Million)
Table 26.82 Clinical Trial Analysis: Distribution by Trial Registration Year, 2017-2022
Table 26.83 Clinical Trial Analysis: Distribution by Trial Status
Table 26.84 Clinical Trial Analysis: Distribution by Trial Phase
Table 26.85 Clinical Trial Analysis: Distribution by Therapeutic Area
Table 26.86 Clinical Trial Analysis: Distribution by Geography
Table 26.87 Clinical Trial Analysis: Distribution by Trial Registration Year and Geography
Table 26.88 Clinical Trial Analysis: Distribution by Trial Status and Geography
Table 26.89 Clinical Trial Analysis: Distribution by Type of Sponsor
Table 26.90 Clinical Trial Analysis: Distribution by Prominent Treatment Sites
Table 26.91 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Registration Year, 2017-2022
Table 26.92 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status
Table 26.93 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Phase
Table 26.94 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Therapeutic Area
Table 26.95 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Location of Trial Site
Table 26.96 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status and Location of Trial Site
Table 26.97 Demand Analysis: Overall Demand for Gene Therapies
Table 26.98 Demand Analysis: Distribution by Type of Therapy, 2022, 2030 and 2035
Table 26.99 Demand Analysis: Distribution by Therapeutic Area, 2022, 2030 and 2035
Table 26.100 Demand Analysis: Distribution by Geography, 2022, 2030 and 2035
Table 26.101 Demand Analysis: Country-wise Distribution (Asia-Pacific), 2022, 2030 and 2035
Table 26.102 Global Gene Therapy Market, 2022-2035 (USD Billion)
Table 26.103 Gene Therapy Market: Distribution by Therapeutic Area, 2022, 2030 and 2035
Table 26.104 Gene Therapy Market: Distribution by Type of Vector Used, 2022, 2030 and 2035
Table 26.105 Gene Therapy Market: Distribution by Type of Therapy, 2022, 2030 and 2035
Table 26.106 Gene Therapy Market: Distribution by Type of Gene Delivery Method Used, 2022, 2030 and 2035
Table 26.107 Gene Therapy Market: Distribution by Route of Administration, 2022, 2030 and 2035
Table 26.108 Gene Therapy Market: Distribution by Geography, 2022, 2030 and 2035
Table 26.109 Gene Therapy Market: Country-wise Distribution (Asia-Pacific), 2022, 2030 and 2035
Table 26.110 Gendicine® Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.111 Oncorine® Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.112 Rexin-G® Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.113 Neovasculgen® Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.114 Strimvelis® Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.115 Imlygic® Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.116 Luxturna™ Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.117 Zolgensma™ Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.118 Collategene® Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.119 Libmeldy™ Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.120 Lumevaq® (GS010) Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.121 OTL-103 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.122 PTC-AADC Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.123 BMN 270 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.124 rAd-IFN/Syn3 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.125 beti-cel Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.126 eli-cel Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.127 lovo-cel Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.128 SRP-9001 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.129 EB-101 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.130 ProstAtak® Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.131 D-Fi Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.132 CG0070 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.133 Vigil™-EWS Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.134 Engensis® Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.135 VGX-3100 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.136 Invossa™ (TG-C) Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.137 VYJUVEKT™ Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.138 PF-06939926 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.139 PF-06838435 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.140 PF-07055480 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.141 SPK-8011 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.142 AMT-061 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.143 VB-111 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.144 Generx® Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.145 AMG001 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.146 OAV-101 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.147 ADXS-HPV Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.148 AGTC 501 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.149 LYS-SAF302 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.150 NFS-01 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.151 AG0302-COVID19 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.152 RGX-314 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 26.153 Hologene 5 Sales Forecast (Till 2035): Base Scenario (USD Million)
Companies Mentioned
- 3SBio
- 4basebio
- 4BIO Capital
- 4D Molecular Therapeutics
- 5AM Ventures
- 683 Capital Management
- AAVantgarde Bio
- AavantiBio
- aaVective
- AaviGen
- AAVogen
- AbbVie
- Abby Grace Foundation
- Abeona Therapeutics
- Abingworth
- Access Biotechnology
- Adage Capital Management
- Adrenas Therapeutics
- Advaxis
- Adverum Biotechnologies
- Aevitas Therapeutics
- Affinia Therapeutics
- Agathos Biologics
- Agent Capital
- AGTC
- Aisling Capital
- AJU IB Investment
- Akouos
- Alaska Permanent Fund
- Alexandria Venture Investments
- Alia Therapeutics
- Allergan
- AllStripes
- Ally Bridge Group
- Almirall
- Alpha Cognition
- Alpha Holdings
- AlphaVax
- Altheia Science
- Altitude Life Science Ventures
- Alumni Ventures
- Amarna Therapeutics
- Ambulero
- American Gene Technologies™
- Amgen
- Amicus Therapeutics
- Amplo
- Anaeropharma Science
- Andelyn Biosciences
- Andreessen Horowitz
- AnGes
- Angionetics
- Anjarium Biosciences
- Anlong Biopharmaceutical
- Apellis Pharmaceuticals
- ApolloBio
- Apple Tree Partners
- Arbor Biotechnologies
- ARCH Overage Fund
- ARCH Venture Partners
- Arctic Light Venture Capital
- Arix Bioscience
- Arkin Bio Ventures
- ArrowMark Partners
- Arthrogen
- ARTIS Ventures
- Aruvant Sciences
- Asabys Partners
- ASC Therapeutics
- Ascidian Therapeutics
- Asia Alpha
- Asklepios BioPharmaceutical
- Aspa Therapeutics
- Aspire Capital Fund
- Astellas Gene Therapies
- Astellas Pharma
- AstraZeneca
- Atamyo Therapeutics
- Atlas Venture
- Atsena Therapeutics
- Auburn University
- Audition Therapeutics
- Auriga Partners
- Australian Taxation Office
- Avellino Lab USA
- AviadoBio
- Avidity Partners
- Avrion Therapeutics
- AVROBIO
- Axovia Therapeutics
- Baillie Gifford
- Bain Capital Life Sciences
- Banca Esperia
- Bayer
- Beam Therapeutics
- Belief BioMed
- Berkeley Catalyst Fund
- Bessemer Trust
- Bezos Expeditions
- Bill & Melinda Gates Foundation
- Biogen
- Biointaxis
- BioInvent International
- Biolocity
- BioMarin Pharmaceutical
- Bionic Sight
- Biotechnology Value Fund
- BioVec Pharma
- Blue Bear Ventures
- bluebird bio
- BlueWater Angels
- Boehringer Ingelheim
- Bonus BioGroup
- Borea Therapeutics
- Boxer Capital
- Bpifrance
- Brace Pharma Capital
- Breton Capital
- BridgeBio
- BrightGene Bio-Medical Technology
- Bristol Myers Squibb
- Brookside Capital
- BVF Partners
- C4 Holding
- California Institute for Regenerative Medicine (CIRM)
- CAM Capital
- Cambridge Enterprise
- Cambridge Gene Therapy
- Cambridge Innovation Capital
- Camford Capital
- Canada Pension Plan Investment Board
- CANbridge Pharmaceuticals
- Cancer Research Institute
- Cancer Research UK
- Candel Therapeutics
- CanSino Biologics
- CapDecisif Management
- Capsida Biotherapeutics
- Carmine Therapeutics
- Casdin Capital
- Case Western Reserve University
- Castle Creek Biosciences
- Catalyst Biosciences
- CavoGene LifeSciences
- CCRM Enterprises
- CDG Capital
- Celgene
- CellGenTech
- Celsion
- Cenova Capital
- CG Oncology
- CGP Investment
- Charcot-Marie-Tooth Association
- Chende Capital
- Chiba University
- Chiesi Ventures
- Children’s Medical Research Foundation
- Children’s Oncology Group
- CICC
- Cima Universidad de Navarra
- CincyTech
- CITIC Group
- CITIC Medical Fund
- Clarus Ventures
- Clearside Biomedical
- CLINO
- Clough Capital Partners
- CMG-SDIC Capital
- Coalition for Epidemic Preparedness Innovations
- Coave Therapeutics
- CODA Biotherapeutics
- Code Biotherapeutics
- Codexis
- Columbia University
- Columbus Venture Partners
- CombiGene
- Conectus
- Consort Medical
- Copernicus Therapeutics
- Cormorant Asset Management
- Cowen
- Cowen Healthcare Investments
- Cowen Private Investments
- CRISPR Therapeutics
- CSL Behring
- CureDuchenne Ventures
- Cynvec
- Cyprium Therapeutics
- Cystic Fibrosis Foundation
- D1 Capital Partners
- Daiichi Sankyo
- DARPA
- Decheng Capital
- Decibel Therapeutics
- Deerfield Management
- Delphi Genetics
- Dexcel Pharma
- DH-LT-Investments
- DiNAQOR
- DNAlite Therapeutics
- DNAtrix
- Driehaus Capital Management
- Duke University
- Dyno Therapeutics
- EB Medical Research Foundation
- EB Research Partnership (EBRP)
- EcoR1 Capital
- EDBI
- Editas Medicine
- Edmond de Rothschild Investment Partners
- Eight Roads Ventures
- Eikonoklastes Therapeutics
- Elaia
- ElevateBio
- Eli Lilly
- Emendo Biotherapeutics
- Emory University
- Encoded Therapeutics
- Endsulin
- enGene
- Ennovation Ventures
- Enochian BioSciences
- Ensoma
- Entos Pharmaceuticals
- Epeius Biotechnologies
- EPIQ Capital Group
- EPS Holdings
- Esco Ventures
- Esteve
- Emerging Technology Partners (ETP)
- Eureka Eurostars
- EUROFANCOLEN
- European Innovation Council Accelerator
- European Investment Bank
- Eventide Asset Management
- Evox Therapeutics
- EVX Ventures
- Excision BioTherapeutics
- Exegenesis Bio
- Exen Capital
- Expression Therapeutics
- ExSight Capital
- Eyevensys
- F2 Ventures
- Farallon Capital Management
- FAUN Foundation
- FEDER
- FerGene
- Fidelity Biosciences
- Fidelity Management & Research Company
- Fidim
- Fin Posillipo
- Flagship Pioneering
- Flash Therapeutics
- Flerie Invest
- Flexion Therapeutics
- Fondation Sanfilippo Suisse
- Forbion
- Foresite Capital
- Forge Biologics
- Fosun Capital
- Foundation Fighting Blindness
- Fountain Healthcare Partners
- FoxKiser
- F-Prime Capital
- Franklin Templeton Investments
- Frazier Healthcare Partners
- Freeline Therapeutics
- Friedreich’s Ataxia Research Alliance
- Fund+
- Fundación Sanfilippo B
- G&P Biosciences
- Galia Gestion
- Gene Biotherapeutics
- Gene Therapy Research Institution
- GeneCure Biotechnologies
- Genelux
- GeneMedicine
- Genenta Science
- GeneOne Life Science
- GeneQuine Biotherapeutics
- Generation Bio
- Genespire
- Genethon
- Genexine
- Genflow Biosciences
- Genprex
- GenSight Biologics
- Genzyme
- Georgia Research Alliance
- Ghost Tree Capital Group
- Gimv
- GO CAPITAL
- Go Capital Amorçage
- GOFAR
- GOG Foundation
- Google Ventures
- GordonMD Global Investments
- Gradalis
- Graphite Bio
- GreatPoint Ventures
- Green Cross Holdings
- Guofang Capital
- Gyroscope Therapeutics
- Harvard University
- Hatteras Venture Partners
- Hayfin Capital Management
- HBM Healthcare Investments
- HealthCap
- Helixmith
- Hemera Biosciences
- Herantis Pharma
- Hillhouse Capital Group
- Hiroshima Venture Capital
- HIV Vaccine Trials Network
- Holostem Terapie Avanzate
- Homology Medicines
- Horizon Technology Finance
- Hovione Scientia
- Huapont Life Sciences
- Huida (Shanghai) Biotechnology
- Human Stem Cells Institute PJSC (HSCI)
- Idinvest Partners
- iECURE
- Ikarian Capital
- Ikarovec
- Illumina Ventures
- Ilya Pharma
- Imperial Innovations
- Imperial Innovations Group
- Imvus
- Inception Capital Management
- New Energy and Industrial Technology Development Organization (NEDO)
- InnoSkel
- Innovate UK
- Innovation Angel Funds
- Innovation Network Corporation of Japan
- Innovation Norway
- InnoVision Capital
- Inovio Pharmaceuticals
- Inserm Transfert
- Institute of Myology
- Intas Pharmaceuticals
- Intellia Therapeutics
- InverVest
- Investitionsbank des Landes Brandenburg
- Invus
- Israel Innovation Authority
- IVERIC bio
- Jaguar Gene Therapy
- Janssen Pharmaceuticals
- Janus Capital Management
- Janus Henderson Investors
- Jasper Therapeutics
- Jeito Capital
- Jennison Associates
- Jeune
- Jinwei
- Johnson & Johnson Development
- Juda Capital
- K2 Venture Capital
- Kate Therapeutics
- Kendall Capital Partners
- Kepler Cheuvreux
- Keytone Ventures
- Kissei Pharmaceutical
- Kiwoom Securities
- Kodikaz Therapeutic Solutions
- Kohlberg Kravis Roberts (KKR)
- Kolon TissueGene
- Korea Investment Partners
- Kriya Therapeutics
- Krystal Biotech
- Kubota Vision
- Kurma Partners
- Kyorin Pharmaceutical
- Lacerta Therapeutics
- Leerink Partners
- Lee's Pharmaceutical
- Legend Capital
- Leiden University Medical Center
- Leland Stanford Junior University
- Lentistem® Biotech
- Centre Léon Bérard
- Lepu Medical Technology
- LEXEO Therapeutics
- Life Sciences Partners (LSP)
- LifeArc
- Lightstone Ventures
- Lilly Asia Ventures
- LIME Asset Management
- Lipigon Pharmaceuticals
- LogicBio Therapeutics
- Lokon Pharma
- Longevity Biotech
- Longitude Capital
- Longmen Capital
- Longwood Fund
- LP Investments
- Ludwig Cancer Research
- Lumira Ventures
- Lundbeckfond Ventures
- LYFE Capital
- Lysogene
- Malin
- Mangrove Partners
- Maryland Technology Development
- Matrix Capital Management
- Matrix Partners China
- Maverick Ventures
- MDimune
- Mediphage Bioceuticals
- Medison Ventures
- MeiraGTx
- Memorial Sloan Kettering Cancer Center
- Mercia Technologies
- Merck
- MERITZ Securities
- Metagenomi
- Michigan Economic Development
- MidCap Financial
- Milo Biotechnology
- Mirae Asset Global Investments
- Mirum Pharmaceuticals
- Mita Securities
- Mitsubishi Tanabe Pharma
- Mitsubishi UFJ Capital
- Moderna
- Moffitt Cancer Center
- Momotaro-Gene
- Monograph Capital
- Morningside
- Movember
- MPM Capital
- MultiVir
- Muscular Dystrophy Association
- Mustang Bio
- Myonexus Therapeutics
- Myosana Therapeutics
- Myrtelle
- NanoCor Therapeutics
- Nanoscope Therapeutics
- Narya Capital
- National Cancer Institute
- National Eye Institute
- National Institute of Allergy and Infectious Diseases
- National Institute of Child Health and Human Development
- National Institute of Neurological Disorders and Stroke
- National Institutes of Health
- National MPS Society
- Nationwide Children’s Hospital
- Navega Therapeutics
- National Education Association (NEA)
- NEC
- Neo Gene Pharm
- NeoMed
- Nerveda
- Netherlands Enterprise Agency
- NeuExcell Therapeutics
- Neuracle Genetics
- Neurocrine Biosciences
- Neurogene
- Neurophth Therapeutics
- Neuvocor
- New Enterprise Associates
- New Leaf Venture Partners
- Newsoara BioPharma
- Ninevah Therapeutics
- Nissei Capital
- Noga Therapeutics
- Nolan Capital
- Bioscience Innovation Grant Program (BIG)
- Northern Light Venture Capital
- Northpond Ventures
- Novartis
- Novo Holdings
- Novo Nordisk
- Nuntius Therapeutics
- Oberland Capital
- Obsidian Therapeutics
- Ocugen
- Odylia Therapeutics
- Oisin Biotechnologies
- Omega Funds
- Omnes Capital
- Oncolys BioPharma
- Oncorus
- OncoSec Immunotherapies
- OncoSenX
- Opus Genetics
- OrbiMed
- ORCA Therapeutics
- Orchard Therapeutics
- ORI Capital
- Orphinic Scientific
- Osage University Partners
- Osaka University
- OSI Investment Management
- Otonomy
- Oxford BioMedica
- Oxford Finance
- Oxular
- Pacira BioSciences
- Panmure Gordon
- Pappas Capital
- Paragon Biosciences
- Parker Institute for Cancer Immunotherapy
- Parkwalk
- Partner Fund Management
- Partners Innovation Fund
- Partners Investment
- Passage Bio
- Patient Square Capital
- Pattern BioSciences
- Pavilion Capital
- PBM Capital
- Pentwater Capital Management
- Perceptive Advisors
- PeriphaGen
- Perseverance Capital Management
- Pfizer
- PFM Health Sciences
- Pharmakon Advisors
- Pharming Group
- Phoenix Nest
- PhorMed
- Pivotal bioVenture Partners
- PlasmidFactory
- Pontifax
- POSCO Capital
- Poseida Therapeutics
- Precigen
- Precision BioSciences
- Prostate Cancer Foundation
- PsiOxus Therapeutics
- PTC Therapeutics
- Purespring Therapeutics
- Qianhai Funds of Funds
- Qianzhan Investment Management
- Qiming Venture Partners
- QVT Financial
- RA Capital Management
- RBV Capital
- RD Fund
- Red Sanfilippo Foundation
- Redbiotec
- Redmile Group
- Redpin Therapeutics
- Reflection Biotechnologies
- Regeneron Pharmaceuticals
- REGENXBIO
- Rejuvenate Bio
- Remedium Bio
- Renova Therapeutics
- Research Council of Norway
- Rev1 Ventures
- Reyon Pharmaceutical
- Ridgeback Capital Investments
- Ring Therapeutics
- Roche
- Rock Springs Capital Management
- Rocket Pharmaceuticals
- Roswell Park Comprehensive Cancer Center
- Royal Holloway University of London
- Royal Melbourne Hospital
- RTW Investments
- Saifu Pharmaceutical
- SalioGen Therapeutics
- Samsara BioCapital
- Samum Vermögensverwaltungs
- San Rocco Therapeutics
- Sana Biotechnology
- Sanfilippo Children’s Foundation
- Sangamo Therapeutics
- Sanofi Genzyme
- Santen Pharmaceutical
- Sarepta Therapeutics
- SBI Japan-Israel Innovation Fund
- Scancell
- Schroder Adveq
- Scottish Enterprise
- S-Cubed Capital
- SDL Ventures
- Seattle Children’s Research Institute
- Sectoral Asset Management
- Selecta Biosciences
- Seneca Therapeutics
- Sensorion
- Sequoia Capital China
- Seroba Life Sciences
- SAFE Pharmaceutical
- Seven and Eight Biopharmaceuticals
- Seventure Partners
- Sham Innovation Santé
- Shanghai BDgene Technology
- Shanghai Sunway Biotech
- Shape Therapeutics
- Shenzhen Qianhai Taxus
- Sherpa Healthcare Partners
- Shinhan Capital
- Shinsei Corporate Investment
- Sibiono GeneTech
- Sight Again
- Silicon Valley Bank
- SillaJen
- Sio Gene Therapies
- Sirtex Medical
- SiSaf
- Skyline Therapeutics
- SMBC Venture Capital
- Sofinnova Ventures
- Solid Biosciences
- Song Hong Fang
- Sonova
- Sorrento Therapeutics
- SOSV
- Spanish Ministry of Science
- SparingVision
- Spark Therapeutics
- Sphera Global Healthcare Fund
- Spirovant Sciences
- SpliceBio
- SR One
- SR-Tiget
- St. Jude Children’s Research Hospital
- Stockholm University
- Stop Sanfilippo Fundación
- StrideBio
- STRM.BIO
- Surveyor Capital
- Suvretta Capital Management
- SV Health Investors
- SVB Leerink
- SVE Capital
- SwanBio Therapeutics
- Swedbank Robur
- Syncona
- T. Rowe Price Associates
- Takeda Pharmaceutical
- Tamid Bio
- Target ALS Foundation
- Targovax
- Taysha Gene Therapies
- Team Sanfilippo Foundation
- TeamedOn International
- Tech Coast Angels
- TechLife Capital
- Temasek
- Temple University
- Tenaya Therapeutics
- Tetraneuron
- The Column Group (TCG)
- The Invus Group
- Théa Open Innovation
- TheraBiologics
- Theravectys
- Third Rock Ventures
- ToolGen
- TPG Capital
- Transgene
- Transhuman Capital
- Trentino Invest
- Triumph Venture Capital
- Trizell
- Tsinghua Holdings Capital
- Tufts University
- Turenne
- Tyris Therapeutics
- UCB
- UCL Technology Fund
- UK Cystic Fibrosis Gene Therapy Consortium
- UK Innovation & Science Seed Fund
- Ultragenyx Pharmaceutical
- UMass Chan Medical School
- UNC Gene Therapy Center
- uniQure Biopharma
- Universitat Autònoma de Barcelona (UAB)
- University College London
- University Hospitals Cleveland Medical Center
- University of Alabama at Birmingham
- University of California, Berkeley
- University of California San Diego School of Medicine
- University of California San Francisco
- University of Florida
- University of Leeds
- University of Massachusetts
- University of Massachusetts Boston
- University of Minnesota
- University of Modena and Reggio Emilia
- University of North Carolina at Chapel Hill
- University of Padua
- University of Pennsylvania
- University of Pittsburgh
- University of Washington
- University of Washington School of Medicine
- UPMC Enterprises
- Uppsala University
- Urovant Sciences
- Usher 2020 Foundation
- Valencian Innovation Agency
- Valencian Institute of Finance
- Valor Equity Partners
- V-Bio Ventures
- VBL Therapeutics
- VCN Biosciences
- VectorY Therapeutics
- venBio
- Venrock
- Versant Ventures
- Vertex Pharmaceuticals
- Vickers Venture Partners
- Vida Ventures
- Vigene Biosciences
- ViGeneron
- Viking Global Investors
- ViralGEN
- Vivet Therapeutics
- Vivo Capital
- Voyager Therapeutics
- VREX Therapeutics
- Washington Research Foundation
- Washington State University
- Waverly Capital
- Weill Cornell Medicine
- Weizmann Institute of Science
- Wellcome Trust
- Wellington Management
- Westlake Village BioPartners
- Whitesun Healthcare Ventures
- WI Harper
- Wize Pharma
- Woodford Investment Management
- Woodline Partners
- Wu Capital
- Wuhan Binhui Biotechnology
- WuXi Advanced Therapies
- WuXi AppTec
- Wyvern Pharmaceuticals
- Xalud Therapeutics
- XyloCor Therapeutics
- Yangtze River Pharmaceutical
- Ysios Capital
- Yuhan
- Ziopharm Oncology
Methodology
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