Executive Summary
Section I:
View from the Top: Cell and Gene Therapy Successes and Promise
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2022 was A Good Year
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2023 Shows Real Promise
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Massive Clinical Trial Landscape
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Strong Growth is Expected to Continue Beyond 2023
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If Commercialization is Truly Possible
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2023: A Pivotal Year for Cell and Gene Therapies
Section II:
Understanding a Rapidly Evolving Sector
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Improving Process Scalability
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Reducing Manufacturing Timelines
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Innovations in Input Materials
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Novel Materials and Reagents for Viral Vector Manufacturing
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Capsid and Vector Engineering
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Novel Viral Vector Purification Processes
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Improved Analytics
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Optimizing Transduction Efficiency with Adjuvants and Enhancers
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More Efficient Cell Cultivation for Cell Therapy Production
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Manufacturing Solutions for Decentralized Cell Therapy Production
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Applying AI in Cell and Gene Therapy R&D
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The Rise of Nonviral Vectors for Cell and Gene Therapies
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Changing Trends in CAR-T Cell Therapy Research
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Commercial Viability: The Final Hurdle
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Conclusions
Section III:
The Cell and Gene Therapy and Outsourced Service Markets
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Current and Projected Sales of Cell and Gene Therapy
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Economic Outlook of cell and gene therapies in clinical trials
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Realizing the Potential Requires Overcoming Real Challenges
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Funding Challenges Drive Resilience Development
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Outsourced Viral Vector and Plasmid DNA Manufacturing Market
Section IV:
Update on Regulatory Guidance for Cell and Gene Therapies
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Regulations Lag Science
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The FDA Responds
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Guidance for Gene-Editing Therapies
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Guidance on Development of CAR-T Cell Therapies
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Guidance on Gene Therapies for Neurodegenerative Diseases
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Umbrella Guidance
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Other FDA Moves
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Need for Global Standardization of Cell and Gene Therapy Regulations
Section V:
Recent Cell and Gene Therapy Clinical Trials Struggles
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More Clinical Trials Leading to More FDA Holds
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Many Possible Impacts
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Biggest Number of Holds in 2021
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A Few Holds in 2022
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2023 was No Exception
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Some Voluntary Stoppages
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Outright Failures
Section VI:
The Next Frontier – Non-Viral Delivery and Gene Editing
Section VII:
AAV Manufacture for Gene Therapy
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Choosing a Cell Line for AAV Manufacture
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Vector Production Strategies
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Traditional AAV Vectors
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Novel AAV Vectors Becoming Commercially Available
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Plasmids required for Triple Transfection of AAV
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Transfection Reagents for AAV Vectors
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Upstream Processing (USP) for AAV Vectors
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Harvesting and Downstream Processing (DSP) for AAV Vectors
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Analytics for AAV Vectors
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Stability, Formulation and Fill/Finish of AAV Vectors
Section VIII:
Lentivirus Manufacture for CAR T-Cell Therapy
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Autologous vs. Allogenic
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Plasmids Required for Lentiviral Vectors
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Transfection Reagents for LV Vectors
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Harvest and DSP for LV Vectors
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Analytics for LV Vectors
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Generation and Expansion of CAR-T Cells
Section IX:
CDMO Pricing for Viral Vector Manufacture
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What Is the Material Needs for a Typical Program?
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FAQs in Viral Vector Manufacturing
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Methodology
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Viral Vector Pricing Matrix
Section X:
Plasmid DNA Manufacture
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Origin of Replication, Antibiotic Resistance and Percent Supercoiled
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Host Strains
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Quality of Plasmid DNA (non-GMP vs GMP)
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Cell Banks for Plasmid DNA
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QC Testing of Plasmid DNA
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Fill/Finish for Plasmid DNA
Section XI:
CDMO Pricing for Plasmid DNA Manufacture
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Methodology
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Plasmid DNA Manufacturing Pricing Matrix
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Comments
Section XII:
Cell and Gene CDMO Company Overview and Profiles
Appendix I:
References
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