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Precision BioSciences gets FDA orphan drug designations for DMD treatment

Published 17/07/2025, 17:12
© Reuters.
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Investing.com -- Precision BioSciences (NASDAQ:DTIL) has received FDA Orphan Drug Designations for its nuclease treatments targeting Duchenne muscular dystrophy.

The designations were granted for the company’s adeno-associated virus serotype 9-delivered DMD19-20L.431 and DMD35-36L.457 nucleases, which are being developed for the treatment of Duchenne muscular dystrophy.

Orphan Drug Designation is given to drugs and biologics intended for the treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the United States. This status provides certain benefits to drug developers, including tax credits for qualified clinical trials and potential market exclusivity.

Duchenne muscular dystrophy is a rare genetic disorder characterized by progressive muscle degeneration and weakness due to alterations in a protein called dystrophin that helps keep muscle cells intact.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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