PharmSource - Gene Therapy Market Opportunity for CMOs - 2019 Edition
Summary
This report will explore whether the bio/pharma industry is adequately prepared for future gene therapy developments and whether CDMOs are making appropriate investments in the capacity and technology to support the coming opportunity. This report is a companion to Cell Therapy Market Opportunity for CMOs - 2018 Edition, PharmSource, July 2018.
This report will explore whether the bio/pharma industry is adequately prepared for future gene therapy developments and whether CDMOs are making appropriate investments in the capacity and technology to support the coming opportunity.
Scope
Reasons to buy
Summary
This report will explore whether the bio/pharma industry is adequately prepared for future gene therapy developments and whether CDMOs are making appropriate investments in the capacity and technology to support the coming opportunity. This report is a companion to Cell Therapy Market Opportunity for CMOs - 2018 Edition, PharmSource, July 2018.
This report will explore whether the bio/pharma industry is adequately prepared for future gene therapy developments and whether CDMOs are making appropriate investments in the capacity and technology to support the coming opportunity.
Scope
- This 47-page report gives important, expert insight you won’t find in any other source. 28 tables and figures throughout the report illustrate major points and trends. This report is required reading for:
- CMO executives who must have deep understanding of the gene therapy marketplace to make strategic planning and investment decisions.
- Sourcing and procurement executives who must understand crucial components of the supply base in order to make decisions about supplier selection and management.
- Pharmaceutical and Biotech companies involved in the development of gene therapies or gene therapy technologies.
- Private equity investors that need a deeper understanding of the market to identify and value potential investment targets.
Reasons to buy
- What are the characteristics of the gene therapy pipeline?
- What volumes of viral vector will be required by 2024 for clinical and commercial gene therapy manufacturing, and how many bioreactors will be needed?
- How many gene therapies from the current clinical pipeline are predicted to be approved?
- What gene therapies have been approved by the FDA and EMA to date?
- What is the geographic reach of gene therapy contract manufacturing organizations and what methods of transfection do they use?
- What are the steps in the in vivo and ex vivo gene therapy supply chains?
- What is the contract manufacturing organization opportunity for gene therapies?
- What are the top therapy areas and indications for gene therapy clinical trials, 2013-2019?
- How can the gene therapy manufacturing process be made more efficient?
- What investments have contract manufacturing organizations and bio/pharmaceutical companies made in gene therapy manufacturing facilities and technologies?
Table of Contents
- 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 (Partial List)
A selection of companies mentioned in this report includes, but is not limited to:
- 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
