CMC and regulatory affairs gene therapy support

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.