Leveraging a Gene Therapy Approach to Maximize Speed and Minimize Costs

RARECast

While gene therapies hold great promise for patients with rare genetic diseases, one obstacle for ultra-rare conditions is that drug developer may view patient populations as being too small to make the development of a gene therapy economically viable. Taysha Gene Therapies, through its partnership with UT Southwestern, is rapidly developing a robust pipeline of gene therapies that leverage the same vector, manufacturing, and course of administration to enable the company to pursue indication that might not otherwise be feasible. The approach shows how rare disease patient advocates, academic researchers, and biotechnology companies can collaborate to enable treatments that would not otherwise be developed. We spoke to R.A. Session II, founder and CEO of Taysha Gene Therapies, about the origins of the company, its unique relationship with gene therapy innovator Steve Gray and UT Southwestern, and the critical role patient organization have played in the process.