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



For sponsorship information, please contact:


Companies A-K

Phillip Zakim-Yacouby

Senior Business Development Manager

Cambridge Healthtech Institute

Phone: (1+) 781-247-1815



Companies L-Z

Aimee Croke

Business Development Manager

Cambridge Healthtech Institute

Phone: (1+) 781-292-0777