AAV Vector Consortia Address Quality Assays, Endpoints
Executive Summary
Two outside groups aim to help CBER on gene therapy questions from ‘N of one’ to markers for hemophilia.
You may also be interested in...
Bespoke Gene Therapy ‘Playbook’ Outlines Platform Approach To AAV-Based Treatments
Supporting ‘n of 1’ gene therapy development, the playbook from a Foundation for the National Institutes of Health-backed consortium aims to help investigators who are not traditional product sponsors prepare for meetings with US FDA and IND submissions.
Regenerative Medicines: Development Slowed By Lack of Standardization, Regulatory And Manufacturing Challenges
A Government Accountability Office report identifies 10 policy options aimed at speeding development and boosting the use of regenerative therapies, but breaks little new ground, highlighting the continuing difficulties in the space.
Califf: Decades Of Follow-Up Necessary For Gene Therapy
FDA Commissioner Califf acknowledges that regulatory pathways for gene therapies are still being developed and that many factors outside of the modality impact agency requirements as he emphasized the need for long-term follow up due to the unknowns of gene modification.