FDA Accepts Rocket Pharma’s Gene Therapy for Severe LAD-I

CRANBURY, N.J. – October 14, 2025 – Rocket Pharmaceuticals, Inc., a late-stage biotechnology company developing genetic therapies for rare disorders, announced today that the U.S. Food and Drug Administration (FDA) has accepted the resubmission of the Biologics License Application (BLA) for KRESLADI (marnetegragene autotemcel; marne-cel). KRESLADI is a lentiviral vector (LV)-based investigational gene therapy designed to treat severe Leukocyte Adhesion Deficiency-I (LAD-I), a rare, life-threatening genetic immune disorder. The FDA has set a PDUFA date of March 28, 2026.

Gene Therapy Addresses Fatal Childhood Condition

Severe LAD-I is an ultra-rare pediatric disease caused by mutations in the ITGB2 gene, which is critical for immune cells (leukocytes) to fight infection and heal wounds. Without an allogeneic hematopoietic stem cell transplant, the disorder is near-uniformly fatal in childhood.

The BLA is supported by positive data from the global Phase 1/2 study of KRESLADI. The study demonstrated 100% overall survival at 12 months post-infusion (and for the entire duration of follow-up) for all enrolled patients. All primary and secondary endpoints were met, and the therapy was well tolerated, with no treatment-related serious adverse events reported.

The clinical data showed substantial reductions in the incidence of significant infections compared to pre-treatment levels.

Evidence of improvement in skin lesions and restoration of wound-healing capabilities was also observed.

Expediting Access for Patients

KRESLADI is a one-time gene therapy that uses the patient’s own (autologous) hematopoietic stem cells, genetically modified with an LV vector to deliver a functional copy of the ITGB2 gene.

Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma, emphasized the importance of the BLA acceptance, noting that bone marrow transplant is currently the only treatment option for severe LAD-I, which carries substantial morbidity, mortality, and cost.

Rocket is eligible to receive a Rare Pediatric Disease Priority Review Voucher (PRV) upon KRESLADI approval. The therapy has previously received FDA Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric, and Fast Track designations in the U.S.