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.


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.

Contact Us

Reach out today for more information on our gene therapy consulting services.

Related Case Studies | View all