Rocket Pharma withdraws BLA for RP-L102 (Fanconi anemia gene therapy) as part of corporate prioritization

ROCKET PHARMACEUTICALS, INC.

Voluntarily withdrew BLA for RP-L102 (mozafancogene autotemcel) from FDA on October 3, 2025.
Withdrawal follows prior MAA withdrawal to European Medicines Agency in July 2025 and cessation of new internal investment in RP-L102 in July 2025.
Decision based on business and strategic considerations; no safety or efficacy concerns noted.
Company will explore external partnership opportunities to potentially advance RP-L102 in the future.
Rocket is focusing resources on programs with clearest regulatory and commercial pathways.

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