Bespoke Gene Therapy Consortium for Rare Diseases Launches

The FDA has teamed up with the National Institutes of Health (NIH), 10 pharmaceutical companies and five nonprofit organizations to launch the Bespoke Gene Therapy Consortium to accelerate development of gene therapies for rare diseases.

The new consortium, which will operate as part of the NIH Accelerating Medicines Partnership program, will work on streamlining the gene therapy development process and will support four to six clinical trials, each focused on a different rare disease.

“By leveraging on experience with a platform technology and by standardizing processes, gene therapy product development can be accelerated to allow more timely access to promising new therapies for patients who need them most,” said Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research.

A primary aim of the consortium is to improve understanding of the basic biology of the adeno-associated virus (AAV), a common gene-delivery vector. Consortium researchers will examine.