Cambridge Healthtech Institute’s 6th Annual
Gene Therapy Manufacturing
Improving Production, Purification, and Yield
19-20 March 2025
Cambridge Healthtech Institute’s 6th Annual Gene Therapy Manufacturing meeting tackles the practical challenges facing the production, scale-up, and purification of viral and non-viral vector-based gene therapies. Topics include cell line development, upstream processing, high density cell culture, recovery and purification, preparing for commercial manufacturing, and scale-up for clinical and commercial supply. Examples will come from the world of AAV and lentiviral vectors, mRNA and, new for 2025, oligonucleotides and gene-edited therapies.
Coverage will include, but is not limited to:
Commercial Manufacturing Strategies
- Preparing for clinical/commercial manufacture—facility design, scale-up strategies, COGs, platform development
- Developing commercial-ready platforms
- Manufacturing networks, sharing facilities vs. internal vs. outsourcing models, tech transfer
- Role of digitalisation, ML/AI in gene therapy bioproduction
- Scalability—Phase I to Phase III to commercial
- Does adding internal manufacturing capabilities add value?
- The impact of novel capsids on manufacturing
Process Development—Cell Line to Cell Culture
- High cell density approaches—case studies
- Optimizing process development—media development, clone selection, cell line development increasing yield, robustness, speed of scalability, stability
- Producer cell lines; new vector, emerging cell lines
- Process intensification strategies—dealing with increased cell mass, yields
- Transfection agents, engineering vectors, metabolic engineering
Downstream Processing
- Advances in purifying viral vectors
- Overcoming challenges in the development of chromatographic separation of empty, partial, and full AAV capsids for gene therapy applications
- Particles—mixed mode, cation exchange
- What level of full/empty is required?
- Viral safety considerations using viral vectors
Formulation, Fill and Finish
- Successful formulation strategies
- Advances in formulation, stability
- Pre-formulation strategies for gene therapies, forced degradation
- Particle analysis, impact on stability, lyophilization
- How are we administrating to patients
- Improvements in delivery, buffer choice, thaw, freeze, logistics
- Fill and finish—working with CMOs, scaling-up from the lab, issues
- Supply chain issues—outsource vs. internal formulation
Manufacturing of RNA-Based Therapies
- Case studies on non-viral DNA technical development—lipid nanoparticles, RNA
- Unique manufacturing and delivery challenges
The deadline for priority consideration is Friday 13 September
All proposals are subject to review by session chairpersons and/or the Scientific Advisory Committee to ensure the overall quality of the conference program. Additionally, as per Cambridge Healthtech Institute policy, a select number of vendors and consultants who provide products and services will be offered opportunities for podium presentation slots based on a variety of Corporate Sponsorships.
Opportunities for Participation: