Rockets gets FDA approval for first LAD-I gene therapy

The U.S. FDA has granted accelerated approval to Rocket Pharmaceuticals’ autologous hematopoietic stem cell-based gene therapy, branded Kresladi, for the treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant — marking the first FDA approved gene therapy for the ultra-rare genetic immunodeficiency.

This accelerated approval was based on increase in neutrophil CD18 and CD11a surface expression. Confirmation of clinical benefit will be based on the evaluation of longer-term follow-up data of treated patients in the ongoing clinical study and through a post-marketing registry.

With the approval of Kresladi, Rocket was granted a rare pediatric disease priority review voucher.

LAD-I predisposes patients to recurrent and fatal infections and is near-uniformly fatal in childhood without an allogeneic hematopoietic stem cell transplant. Kresladi contains autologous hematopoietic stem cells that have been genetically modified with a lentiviral vector to deliver a functional copy of the ITGB2 gene, which encodes for the beta-2 integrin component CD18, a key protein that facilitates leukocyte adhesion and enables their extravasation from blood vessels to fight infection.

The approval for Kresladi was hard-fought. In July 2025, the Rocket unveiled a strategic re-org and pipeline prioritization that was partially aimed at enabling the company to resolve the CMC deficiencies noted in the CRL that the company initially received for Kresladi in June 2024. Rocket resubmitted the BLA for Kresladi in October 2025.