A successful gene therapy trialed in a dog model at Michigan State to treat a rare, inherited eye disease has been selected to be advanced to a clinical trial in human patients.
On May 16 at the American Society of Gene and Cell Therapy’s 26th Annual Meeting in Los Angeles, the Foundation for the National Institutes of Health announced that the Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium had selected eight rare diseases for its clinical trial portfolio. Among those selected is retinitis pigmentosa 45.
What is the Bespoke Gene Therapy Consortium?
Creating genetic therapies is incredibly expensive, and development programs can impose major challenges. Because of this, drug companies typically focus on more common diseases for which the cost of creating the therapy can be more easily recouped and profits more quickly made. Ultra-rare conditions affect between one and a few hundred people; these individuals requiring “bespoke” gene therapies are left wanting.
The Bespoke Gene Therapy Consortium is a 27-member, public-private partnership launched by the NIH and FDA to address the challenges of developing genetic therapies for ultra-rare diseases. The five-year, $80M initiative includes NIH institutes, the FDA, 12 industry players (Biogen Inc.; Danaher Corporation; Janssen Research & Development, LLC; Novartis Institutes for BioMedical Research; Ovid Therapeutics Inc.; Pfizer Inc.; REGENXBIO Inc.; Spark Therapeutics; Takeda Pharmaceutical Company Limited; Taysha Gene Therapies; Thermo Fisher Scientific; and Ultragenyx Pharmaceutical), and 10 not-for-profit organizations. It is led by the Foundation for the NIH.