When it comes to gene editing technology, there are few meetings as valuable for us and our collaborators as the annual CRISPR meeting. Academic in focus and highly technical in nature, the 2022 edition of this event came to Boston, packing more than 40 lectures and 120 posters into a rapid-fire, three-day learning experience.
Historically speaking, drug master files (DMF) have been used in filing Investigational New Drug applications (IND). However, they’ve never been used for CRISPR because that’s a relatively new therapy. With new draft FDA guidance relating to cell therapies using CRISPR, we’re moving further ahead into a new frontier of genomic medicine.
In my previous post on new draft FDA guidelines that concern cell therapies, I covered key control considerations for CRISPR reagents and the GMP manufacturing considerations for drug substances affected by that guidance. In this post, I’ll go over a couple approaches to consider when working with gene-modified cell therapies, and how they could be affected.
Of all the presentations at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, a presentation by renowned physician and geneticist Dr. Francis Collins caught our attention. In his talk, Dr. Collins spotlighted an encouraging path forward in the treatment of rare diseases with cell and gene therapies. While acknowledging that gene therapy is being taken to new heights in many areas, he noted there are still thousands of diseases that have not yet had a focus for treatment development.
