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.