Taking gene therapies into ultra-rare diseases: BGTC’s next step

The Foundation for the National Institutes of Health and its collaborators have identified a path to advance gene therapy development for indications too rare to support commercial development. Now, they need to find a way to scale it. 

Last week, the Bespoke Gene Therapy Consortium (BGTC), which is run by FNIH through its Accelerating Medicines Partnership, announced the eight rare diseases that will make up its clinical portfolio. For each indication, the plan is to piece together AAV technology and IP, advance the programs through preclinical development, then run Phase I trials. ...