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:
For more details on the conference, please contact:
Daniel Barry
Senior Conference Director
Cambridge Healthtech Institute
Phone: (+44) 7837 651 303
Email: mailto:dbarry@healthtech.com
For sponsorship information, please contact:
Companies A-K
Phillip Zakim-Yacouby
Senior Business Development Manager
Cambridge Healthtech Institute
Phone: (1+) 781-247-1815
Email: pzakim-yacouby@cambridgeinnovationinstitute.com
Companies L-Z
Aimee Croke
Business Development Manager
Cambridge Healthtech Institute
Phone: (1+) 781-292-0777
Email: acroke@cambridgeinnovationinstitute.com