Rocket Pharmaceuticals Withdraws Gene Therapy Application for Fanconi Anemia

PRINCETON, NJ –Oct 3, 2025–Rocket Pharmaceuticals announced the voluntary withdrawal of its rolling Biologics License Application (BLA) for its gene therapy candidate, RP-L102 (Fanca-cel), intended to treat Fanconi anemia.

The company confirmed the withdrawal in an Oct. 3 filing with the Securities and Exchange Commission (SEC), stating the decision was due to “business and strategic considerations” stemming from a pipeline and workforce restructuring initiated in July. Rocket emphasized that the decision was not based on any safety or efficacy concerns regarding the gene therapy.

Strategic Pivot and Reprioritization

Rocket had initiated the rolling BLA for the gene therapy RP-L102 earlier this year. However, the official withdrawal confirms a strategic pivot by Rocket to focus resources on programs with “the clearest regulatory and commercial pathways.” This restructuring primarily aims to shift the company’s focus away from most of its hematology assets to concentrate instead on its cardiovascular pipeline, including its programs that utilize the AAV delivery platform.

The company stated that the withdrawal of the BLA preserves its ability to “re-engage with regulators at a later date” and will explore “external partnership opportunities” that could potentially enable the advancement of RP-L102, a non-AAV gene therapy, in the future.

Continuing Challenges with AAV Programs

This move follows a period of regulatory challenges for the company, particularly concerning its AAV-based assets:

• Kresladi: Last year, the FDA denied the BLA for Kresladi (for severe leukocyte adhesion deficiency-I) due to manufacturing queries. Rocket now expects to resubmit that BLA by the end of 2025.
• RP-A50: In May, a pivotal trial for the AAV9 gene therapy RP-A50 (for Danon disease), which uses a specific AAV serotype, was placed on a clinical hold, which was only lifted last month. The challenges faced by the company, including those with the AAV program RP-A50, highlight the rigorous regulatory scrutiny applied to the manufacturing and safety of AAV-delivered genetic medicines. The company’s continued commitment to its cardiovascular pipeline is largely focused on AAV therapies.

The company previously announced laying off 30% of its staff as part of its strategic realignment to prioritize key programs, particularly its AAV-driven pipeline.