‘Bespoke Gene Therapy Consortium’ sets out to enable gene therapies for ultra-rare diseases

The NIH and FDA’s newly launched 27-member public–private partnership will spend US76 million addressing the hurdles of AAV-based gene therapies for ultra-rare diseases. The NIH and FDA’s newly launched 27-member public–private partnership will spend US6 million addressing the hurdles of AAV-based gene therapies for ultra-rare...