Rocket’s KRESLADI Gene Therapy For Rare Immune Disorder LAD-I Receives FDA BLA Acceptance

Rocket Pharmaceuticals, Inc., a late-stage biotechnology company focused on developing gene therapies for rare and life-threatening disorders, announced that the U.S. Food and Drug Administration (FDA) has accepted the resubmission of its Biologics License Application (BLA) for KRESLADI™ (marnetegragene autotemcel; marne-cel). This lentiviral vector-based investigational gene therapy is designed to treat severe Leukocyte Adhesion Deficiency Type 1 (LAD-I). This rare immune disorder leads to recurrent, life-threatening infections and is nearly always fatal in childhood without a stem cell transplant. The FDA has set a PDUFA date of March 28, 2026, for its decision.

“We value the continued dialogue with the FDA and believe the BLA moves Rocket closer to our goal of delivering a one-time gene therapy to patients facing the devastating effects of severe LAD-I. For these patients, survival beyond childhood is uncommon. Bone marrow transplant is currently the only treatment option, has substantial morbidity, mortality, and cost, and may not be available in time for these children,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma. “As we approach our new PDUFA date, we are focused on the opportunity to make this therapy available to patients who need it most.”

The BLA submission is supported by strong efficacy and safety results from a global Phase 1/2 clinical trial, in which KRESLADI™ achieved 100% overall survival at 12 months post-infusion and met all primary and secondary endpoints. The treatment was well tolerated, with no serious treatment-related adverse events. Patients experienced fewer severe infections, significant healing of skin lesions, and restoration of wound-healing functions, demonstrating the therapy’s potential to address key clinical symptoms of LAD-I.

If approved, Rocket would qualify for a Rare Pediatric Disease Priority Review Voucher (PRV). KRESLADI™ utilizes patient-derived stem cells that have been genetically modified with a lentiviral vector to deliver a functional ITGB2 gene, thereby restoring the production of CD18 — a protein crucial for immune cell adhesion and defense against infection. The program has received RMAT, Rare Pediatric, and Fast Track designations in the U.S., as well as PRIME and ATMP designations in the EU, alongside Orphan Drug status in both regions. The therapy was originally licensed from CIEMAT and research partners in Spain and the U.K., with the vector developed collaboratively by University College London and CIEMAT.