The FDA and National Institutes of Health (NIH) are partnering with 15 private organizations to increase the number of gene therapies for rare diseases. They are creating the Bespoke Gene Therapy Consortium (BGTC), which includes ten private and five nonprofit partners. The NIH direction states that many rare diseases are caused by a problem in just one gene. A “bespoke” customized therapy that can fix this gene could be a potential therapy. Creating gene therapies is a difficult process that takes time and money. There are also no standards in the field of gene therapy and usually, one disease is addressed at a time. BGTC wants to use the experience these organizations have to create a standardized therapeutic development model that can make gene therapy development more efficient. One way BGTC will do this is by improving their understanding of a common gene delivery method, the adeno-associated viral vector (AAV). BGTC will also create a standard set of tests for making AAV vectors so that the process can be faster. BGTC will also fund 4-6 clinical trials that are focused on various rare diseases caused by single-gene mutations that have no gene therapies under development. BGTC hopes that these trials can also reduce the time needed between testing in animal models and human clinical trials. BGTC will also work to streamline the regulatory requirements needed for FDA approval.
What this means for Usher syndrome: Inherited retinal degenerations (IRD), like retinitis pigmentosa (RP), is one such genetic disorder that can potentially benefit from this research. Vision loss in Usher syndrome is caused by RP. Because of this, research and therapies created by BGTC could be useful for the Usher community.
