A Study to Evaluate the Safety and Efficacy of EX 151 Single-Cut CRISPR Gene Editing in Males with Duchenne Muscular Dystrophy (DMD), with mutations amenable to exon 51 skipping or reframing
Vertex Non-confidential report
Vertex proposed a novel CRISPR/Cas9 based therapy designed for patients with DMD who have mutations amenable to exon 51 skipping.
The committee felt that this application represents a novel and exciting technology with substantial potential benefit for DMD and broad implications to the entire neuromuscular field, supported by promising pre-clinical data in the DMD mouse and dog models.
TACT provided recommendations and advice on follow-up work for pre-clinical experiments. Advice was also included on the planned clinical trial including powering, dosing, inclusion/exclusion criteria and regulatory assurance for the proposed study.
This was a well-constructed application for TACT advice that resulted in fruitful discussion. The committee appreciated the opportunity to consider this application.
Name of applicant: Alison McVie Wylie Reviewed: November, 2019 in Toronto, ON, Canada
Vertex proposed a novel CRISPR/Cas9 based therapy designed for patients with DMD who have mutations amenable to exon 51 skipping.
The committee felt that this application represents a novel and exciting technology with substantial potential benefit for DMD and broad implications to the entire neuromuscular field, supported by promising pre-clinical data in the DMD mouse and dog models.
TACT provided recommendations and advice on follow-up work for pre-clinical experiments. Advice was also included on the planned clinical trial including powering, dosing, inclusion/exclusion criteria and regulatory assurance for the proposed study.
This was a well-constructed application for TACT advice that resulted in fruitful discussion. The committee appreciated the opportunity to consider this application.
