Gene Therapy Leaders Push for Novel Regulatory Models to Aid Rare Disease Treatments
Gene therapy leaders are advocating for new regulatory models to facilitate the development of personalized treatments for rare diseases. This push comes nearly a year after Baby KJ Muldoon received a groundbreaking CRISPR treatment for CPS1 deficiency, a rare genetic condition. The Innovative Genomics Institute, led by CRISPR pioneer Jennifer Doudna, is preparing to trial a similar treatment for another child with a different genetic disorder. The FDA's plausible mechanism pathway aims to accelerate gene therapies for small patient populations, but some in the biotech industry, like EveryONE Medicines, have found it insufficient for commercial viability. The pathway requires separate applications for each drug, which can be burdensome. Biopharma companies, including Aurora Therapeutics, are working to develop platform technologies to streamline the production of gene therapies for rare mutations. 
