Cambridge Healthtech Institute’s 5th Annual

Gene Therapy CMC and Analytics

Improving the Analysis, Control, and Quality of Gene Therapies

19-20 March 2024

Cambridge Healthtech Institute’s Gene Therapy CMC and Analytics conference uncovers the practical challenges facing the analysis, characterisation, and quality of viral vector-based gene therapies for clinical and commercial development. The conference features dedicated sessions on CMC strategy, regulatory feedback, analytical and process characterisation, development and qualification, product- and process-related impurities, empty/full capsids and their link to quality, bioassays, comparability, stability, formulation, and analytics for non-viral gene therapies.

Coverage will include, but is not limited to:

A: Current Challenges in Gene Therapy CMC Development

Current challenges and future of gene therapy technical development
Lessons learned from commercial/late-stage products
Investors’ perspective of gene therapy development

B: Regulatory Feedback, Quality, and Standards

Regulatory CMC for gene therapies
Current expectations from regulatory authorities
Toxicity concerns
Release testing, changes to Compendium

C: CMC Strategies, Raw Materials

CMC strategies for rare diseases
Qualifying starting/intermediary materials
Adventitious agent testing—update from PDA group
Comparability strategies following process change
Case studies from AAV, lentiviral, non-viral, mRNA, exosomes

D: Analytics—Product and Process Characterisation

Characterisation strategies used for faster, deeper characterisation: mass spec, next-generation sequencing, mass photometry, SEC, DLS
Developing next-generation analytical technologies
Developing faster analytics, DLS for aggregation, stability-indicating assays, digital droplet PCR?
Method development and lifecycle management of analytical methods
Analytics: Quality-by-Design and PAT
Quality-by-Design approaches to gene therapy
Setting Critical Quality Attributes
Case studies from AAV, lentiviral, non-viral, mRNA, exosomes

E: Analytics—Potency Assays

Developing robust and quantitative potency assays: case studies
Validating analytical methods, HTPD analytical strategies
Comparability strategies

F: Product-Related Impurities and Ensuring Quality

Characterisation and removal of process related impurities
Quantitation of empty, full, and other particles
Particle analysis—demonstrating purity, particle vs. mass
Role of metabolomics in process development
Final product testing—release testing strategies
Adventitious agent testing
Use of AI in process characterisation

G: Analytics for Non-Viral Gene Therapies, mRNA, and Exosomes

CMC strategies for non-viral gene therapies—what are the unique considerations?
Analytical considerations of viral vs. non-viral gene therapies
Case studies to demonstrate quality and purity

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: