Source: Epider Molysis Bulls News
(Cleveland, Ohio) — Abeona Therapeutics‘ EB-101, a gene therapy in development for patients with recessive dystrophic epidermolysis bullosa (RDEB), has received the regenerative medicine advanced therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA).
The RMAT designation is attributed to promising “regenerative” therapies — cell therapies, tissue engineering, or cell and tissue products — that are supported by preliminary clinical data showing potential to advance the therapeutic care of unmet medical needs. The new designation is part of the 21st Century Cures Act, and opens the possibility of priority review and accelerated approvals by the FDA.
FDA’s decision was also supported by data from a Phase 1/2 clinical trial (NCT01263379) in which researchers used patients’ own skin cells (keratinocytes) to insert, using a virus, a correct version of the COL7A1 gene, which is defective in patients with epidermolysis bullosa. This led to the production of functional collagen protein.