Cambridge Healthtech Institute’s 5th Annual

Gene Therapy Manufacturing

Improving Production, Purification, and Yield

20-21 March 2024

Cambridge Healthtech Institute’s Gene Therapy Manufacturing conference tackles the practical challenges facing the production, scale-up, and purification of viral and non-vector-based gene therapies. Topics include cell line development, upstream processing, 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 other non-viral approaches.

Coverage will include, but is not limited to:

Gene Therapy Process Development—Cell Line to Cell Culture

Process design and development strategy—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
Scaling up—moving from adherent to suspension, challenges; challenges scaling transient-based processes and industry examples
Transfection agents, engineering vectors, metabolic engineering

Downstream Processing and Formulation

Downstream development—innovations in capture, depth filtration, resin selection, chromatography, scalability
Handling increased mass—harvest, clarification strategies
Particles—mixed mode, cation exchange.
What level of full/empty is required?
Process consideration
Viral safety considerations using viral vectors

Commercial-Scale Gene Therapy Manufacturing

Preparing for commercial manufacture: scale-up strategies, COGs; is triple transfection the most suitable manufacturing platform? For/against
Manufacturing networks, sharing facilities vs. internal vs. outsourcing models
Developing commercial-ready platforms
Scalability—Phase I to Phase III to commercial
Does adding internal manufacturing capabilities add value?

Role of AI and Digitalization in Gene Therapy Production

Bioprocessing 4.0, and AI in gene therapy manufacturing
Machine learning in optimising gene therapy production

Process Development for Non-Viral Gene Therapies, mRNA, and Exosomes

Process development for mRNA therapies—what are the bottlenecks?
Non-viral gene therapies—what are the unique considerations?
Case studies to demonstrate quality and purity

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 administering 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

The deadline for priority consideration is Friday 22 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: