The National Institutes of Health (NIH), the U.S. Food and Drug Administration (FDA), 10 pharmaceutical companies, and five non-profits have partnered as the Bespoke Gene Therapy Consortium (BGTC) to help speed up the development of gene therapies for rare diseases. Many rare diseases are caused by a single gene having a defect, and many such mutations are already known. This could make gene therapy a promising therapeutic approach. However, development of any gene therapy for one specific rare disease is complex, expensive, and time-consuming. BGTC hopes to pool resources to create a standardized, vector-based delivery for gene therapies that can be applied to many rare diseases. By working together, the consortium can create bespoke gene therapies and encourage other companies to target rare diseases. BGTC will try to improve its understanding of this common gene delivery vector, adeno-associated virus (AAV), that could improve the overall therapy. BGTC will also develop standardized analytical tests to use on these vectors to make development of gene therapies more efficient. BGTC will also fund research that will support four to six clinical trials related to treatments of different rare diseases using AAV vectors. Another aim of BGTC is to accelerate the process of research trials and FDA approval of gene therapies for rare diseases.
What this means for Usher syndrome: Because Usher syndrome is a rare disease, this consortium could help accelerate the process of developing new therapies for USH. This consortium focuses on gene delivery technology using AAV which has been used in past USH research. This announcement also seems to show that the NIH and FDA are putting more effort into rare disease research.
