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FLORHAM PARK, N.J. – Celularity Inc. (NASDAQ: CELU), a biotechnology agency specializing in regenerative drugs, has formally requested orphan drug designation from the U.S. Meals and Drug Administration (FDA) for PDA-002, its placental-derived cell remedy meant for the therapy of Facioscapulohumeral Muscular Dystrophy (FSHD). Orphan drug standing is granted to therapies for uncommon ailments affecting fewer than 200,000 people in the US and may present sure advantages, together with tax credit and market exclusivity.
The corporate beforehand secured FDA Investigational New Drug Software clearance for PDA-002 in FSHD and anticipates initiating a section 1/2 research within the latter half of 2024. This medical trial is a vital step in direction of a possible Biologics License Software submission for FSHD therapy, a debilitating situation with no present treatment or disease-modifying therapies. FSHD, one of the vital prevalent types of muscular dystrophy, impacts round 1 in 8,000 folks and results in extreme muscle losing.
Celularity’s CEO, Robert Hariri, M.D., Ph.D., emphasised the corporate’s dedication to addressing the unmet wants of sufferers with difficult ailments by way of their cell remedy platform. The upcoming research is a part of Celularity’s broader technique to develop mobile immunotherapies for neurodegenerative issues.
The corporate’s portfolio features a vary of cell therapies derived from the placenta, concentrating on ailments associated to getting older, most cancers, and immune issues. Along with PDA-002, Celularity is engaged on mesenchymal stem cells (MSCs), T-cells, pure killer (NK) cells, and exosomes for therapeutic functions.
The knowledge on this article is predicated on a press launch assertion from Celularity Inc.
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