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?
Table of Contents
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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