FDA accepts Rocket’s BLA resubmission for immune disorder gene therapy

The U.S. FDA has accepted Rocket Pharmaceuticals’ BLA resubmission for Kresladi, a lentiviral vector-based investigational gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I), a rare genetic immune disorder.

The PDUFA date set by the FDA is March 28, 2026. Rocket is eligible for a rare pediatric disease priority review voucher, should Kresladi be approved.

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.

Back in July, 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 Rocket received for Kresladi in June 2024.