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# Bespoke Gene Therapy Portfolio has 8 Diseases
- URL: https://www.fdaweb.com/bespoke-gene-therapy-portfolio-has-8-diseases/
- Published: 2023-05-16T12:00:00.000Z
- Updated: 2026-09-14T18:32:18.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5154461

The Accelerating Medicines Partnership Bespoke Gene Therapy Consortium, a public-private partnership between FDA, the National Institutes of Health, biopharmaceutical and life science companies, and nonprofit and other organizations to help speed the development and delivery of customized or “bespoke” gene therapies that could treat the millions of people affected by rare diseases, has selected eight diseases for its clinical trial portfolio. An [announcement](https://fnih.org/news/press-releases/foundation-national-institutes-health-announces-selection-eight-rare-diseases?bbeml=tp-dnwJc27W802R9IacBWspeg.jLBb8BQS5%5FEW%5FQ2SgT4KfVw.r9spSWVR6u06rWIE-jOOolQ.lpC3UICuKskOxaztbzF8J5g&ref=fdaweb.com) from the Foundation for the National Institutes of Health, which manages the partnership, says the portfolio “pioneers a novel approach to gene therapy development for rare diseases by demonstrating that manufacturing and testing standards can provide a streamlined approval pathway for first-in-human clinical trials.

The diseases in the clinical trial portfolio are Charcot-Marie-Tooth disease type 43, congenital hereditary endothelial dystrophy, Morquio A syndrome, multiple sulfatase deficiency, NPHP5 retinal degeneration, propionic acidemia, retinitis pigmentosa 45, and spastic paraplegia 50\. “These selections were made based on rigorous scientific and technical review by a panel of gene therapy experts using the Bespoke Gene Therapy Consortium’s established criteria,” the foundation says. “These criteria include, but are not limited to, the adequacy of the gene for insertion into an adeno-associated virus vector, sufficient proof of concept and natural history data, the existence of an established disease model, a lack of available treatment, and an overall readiness for entering into a clinical trial.”