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Rocket Pharmaceuticals withdraws US application for blood disorder gene therapy
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Rocket Pharmaceuticals withdraws US application for blood disorder gene therapy
Oct 3, 2025 4:46 AM

Oct 3 (Reuters) - Rocket Pharmaceuticals ( RCKT ) said

on Friday it has withdrawn its application for U.S. approval of

its experimental gene therapy for a rare inherited blood

disorder.

The therapy RP-L102 was being tested in patients with

Fanconi anemia, characterized by impaired bone marrow function

which leads to a decrease in the production of blood cells.

The company said the move was driven by business strategy

and not by concerns about the treatment safety or efficacy.

Rocket said it will consider external partnership

opportunities for RP-L102.

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