Rocket Pharma pulls BLA for gene therapy RP-L102 in the USA
US late-stage biotech Rocket Pharmaceuticals (Nasdaq: RCKT) today announced via a Securities Exchange filing that it has voluntarily withdrawn its Biologics License Application (BLA) to the US Food and Drug Administration for mozafancogene autotemcel, also known as RP-L102, an investigational gene therapy for the treatment of Fanconi anemia.
The company stated that this decision is part of a previously announced corporate prioritization to focus resources on programs with clearer regulatory and commercial pathways. While the company maintains a strong liquidity position with a current ratio of 6.39 and more cash than debt on its balance sheet, InvestinPro data indicates it’s quickly burning through cash.
As part of this shift, Rocket Pharmaceuticals ceased new internal investment in RP-L102 and withdrew its Marketing Authorization Application with the European Medicines Agency in July 2025. The withdrawal of the US BLA follows these earlier actions.
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