Gene Therapy Market by Type of Therapy, Type of Gene Delivery Method Used, Type of Vector Used, Target Therapeutic Areas, Route of Administration, and Key Geographical Regions: Industry Trends and Global Forecasts, 2022-2035

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?

1. PREFACE

1.1. 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