Challenge:


A biotech company developing novel gene therapy approaches for in vivo cell trans-differentiation had lead programs in IND-enabling studies for two indications. The client requested assistance from CMC and regulatory experts with experience in gene therapies and working with CDMOs, including oversight.

Solution:


Alacrita provided assistance "as needed" in the below areas:

  • CDMO site audits
  • Routine meetings with CDMO
  • Analytical review of CMC reports and batch record review
  • CMC plan and process development review
  • QA/QC oversight/review
  • IND preparation and submission
  • FDA INTERACT and/or pre-IND meeting preparation and execution
  • Expedited program designation (e.g. orphan status) applications
  • EMA engagement and submission preparations
  • Environmental risk assessments
  • Establishment of a quality management system



Gene Therapy Consulting

Alacrita frequently supports client programs in the gene therapy space, with our expertise spanning a range of therapeutic areas and functional disciplines.

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