Executive Summary
Introduction
Commercial Gene Therapy Experience
Gene Therapy Pipeline
The Viral Vector Shortage
Approvals Outlook
Gene Therapy Supply Chain
Demand Models
What It Means
Notes on Methodology
Appendix

List of Tables
Table 1: FDA-Approved, Marketed Gene Therapies
Table 2: EMA-Approved, Marketed Gene Therapies
Table 3: Top 10 Gene Therapy Sponsor Companies, All Development Stages
Table 4: Top 10 Gene Therapy Sponsor Companies, Phases I-III
Table 5: Predicted Gene Therapy Approvals
Table 6: Gene Therapy CMO Sites and their Methods of Transfection
Table 7: Model for Zolgensma and Luxturna Viral Vector Commercial Requirements, 2024
Table 8: Model for Yearly Commercial Gene Therapy Viral Vector Requirements
Table 9: Model for Yearly Clinical Gene Therapy Viral Vector Requirements
Table 10: Model for AAV Bioreactor Capacity
Table 11: Model for Lentiviral Bioreactor Capacity
Table 12: Forecast: Annual Viral Vector Bioreactor Capacity for Clinical and Commercial Gene Therapy
Table 13: Likelihood of Approval “N” values for Gene Therapy

List of Figures
Figure 1: Zolgensma Global Sales Forecast, 2019-2024 ($M)
Figure 2: Luxturna Global Sales Forecast 2019-2024 ($M)
Figure 3: Pipeline Gene Therapy Drugs by Stage
Figure 4: Phase I-III Gene Therapy Trial Starts by Sponsor Type
Figure 5: Phase I Gene Therapy Trial Starts by Sponsor Type
Figure 6: Phase II Gene Therapy Trial Starts by Sponsor Type
Figure 7: Phase III Gene Therapy Trial Starts by Sponsor Type
Figure 8: Top Ten Sponsors of Phase I-III Gene Therapy Trials, 2013-2019
Figure 9: Top Five Therapy Areas in Gene Therapy Trials, 2013-2019
Figure 10: Top Seven Indications in Gene Therapy Trials, 2013-2019
Figure 11: Reasons for Discontinuations of Gene Therapy Trials, 2013-2019
Figure 12: Top Gene Therapy CMOs by Number of Gene Therapy Facilities
Figure 13: Gene Therapy CDMOs by Number of Gene Therapy Facilities
Figure 14: Geographic Spread of Dedicated Contract Gene Therapy Contract Manufacturing Facilities
Figure 15: Geographic Spread of Excess Capacity Gene Therapy Contract Manufacturing Facilities

Companies Mentioned
- Abeona Therapeutics Inc
- Advanced BioScience Laboratories Inc
- Adverum Biotechnologies Inc
- Alliance for Regenerative Medicine
- Amicus Therapeutics Inc
- Applied Genetic Technologies Corp
- AskBio
- Atvio Biotech Ltd
- Audentes Therapeutics Inc
- AveXis Inc
- Axovant Sciences Ltd
- Batavia Biosciences BV
- Benitec Biopharma Ltd
- Biogen Inc
- BioNTech SE
- BioReliance Corp
- Biovian Oy
- bluebird bio
- Brammer Bio LLC
- Candel Therapeutics
- Candel Therapeutics
- Catalent Inc
- Cell & Gene Therapy Catapult
- Charles River Laboratories International Inc
- City of Hope
- Clinical BioManufacturing Facility
- Cobra Biologics Ltd
- Delphi Genetics SA
- EMA
- Emergent BioSolutions Inc
- Evitria SA
- Expression Systems LLC
- FDA
- FinVector Oy
- FKD Therapies Oy
- Fraunhofer-Gesellschaft zur Forderung der Angewandten Forschung eV
- Fujifilm Diosynth Biotechnologies USA Inc
- GE Healthcare
- GenIbet Biopharmaceuticals SA
- Genscript Biotech Corp
- Genzyme Corp
- GlaxoSmithKline Plc
- Helixmith Co Ltd
- Hitachi Chemical Co Ltd
- Huazhong University of Science & Technology
- Human Stem Cells Institute
- iCarTAB BioMed Inc
- ID Pharma Co Ltd
- IDT Biologika GmbH
- Instituto de Biologia Experimental e Tecnologica
- International Society for Cellular Therapy
- Kaneka Eurogentec SA
- King’s College London
- LakePharma Inc
- Lonza Group Ltd
- MassBiologics
- MeiraGTx
- Memorial Sloan Kettering Cancer Center
- Merck & Co Inc
- Merck KGaA
- Meridian Life Science Inc
- Mitsubishi Tanabe Pharma Corp
- MolMed SpA
- MTG Biotherapeutics Inc
- Nationwide Children’s Hospital
- NCI Biopharmaceutical Development Program
- Neurotech Pharmaceuticals Inc
- Nightstar Therapeutics PLC
- Novartis
- Novasep Holding SAS
- OncoSec Medical Inc
- Orchard Therapeutics Plc
- Orgenesis
- Oxford BioMedica Plc
- Oxford Genetics Ltd
- Paragon Bioservices Inc
- Pfizer Inc
- Polyplus
- RegenxBio Inc
- Sangamo Therapeutics Inc
- Sanofi
- Sarepta Therapeutics Inc
- Shenzen SiBiono GeneTech Co Ltd
- Sorrento Therapeutics Inc
- Spark Therapeutics Inc
- Symbiosis Pharmaceutical Services Ltd
- Takara Bio Inc
- Thermo Fisher Scientific
- Tocagen Inc
- UNC Vector Core
- UniQure NV
- Vascular Biogenics Ltd
- Vibalogics GmbH
- ViGene Biosciences Inc
- ViralGen
- Virovek InCorp
- Voyager Therapeutics
- Waisman Biomanufacturing
- WuXi AppTec (Shanghai) Co Ltd
- WuXi Biologics Co Ltd
- Yposkesi SAS
- Zelluna Immunotherapy
- ZIOPHARM Oncology Inc