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First FDA-approved gene therapy for children with severe LAD-I due to biallelic variants in ITGB2
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Severe LAD-I is an ultra-rare, life-threatening pediatric genetic immunodeficiency characterized by recurrent infections and high early-childhood mortality without treatment
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FDA grants Rare Pediatric Disease Priority Review Voucher
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Company to host conference call today,March 27at8:30 AM ET
CRANBURY, N.J.--(BUSINESS WIRE)--Mar. 27, 2026-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that theU.S. Food and Drug Administration(FDA) has granted accelerated approval for KRESLADI™ (marnetegragene autotemcel), an autologous hematopoietic stem cell-based gene therapy indicated 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. This indication is approved under accelerated approval 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, the FDA granted Rocket a Rare Pediatric Disease Priority Review Voucher (PRV), a program designed to encourage development of therapies for rare pediatric diseases. The Company intends to evaluate strategic options to monetize the PRV in a manner designed to enhance financial flexibility and maximize shareholder value.
“The approval of KRESLADI represents an important milestone for the severe LAD-I community,” saidGaurav Shah, M.D., Chief Executive Officer,Rocket Pharmaceuticals. “This approval reflects the dedication of patients, families, investigators, and regulators who have worked together to advance research of this ultra-rare disease. We look forward to making KRESLADI available to eligible patients inthe United States.”
LAD-I is an ultra-rare genetic pediatric disease caused by mutations in the ITGB2 gene encoding for CD18, a key protein that is expressed along CD11 integrins to facilitate leukocyte adhesion to the blood vessel wall and migration to tissues to confine and clear infections and orchestrate wound repair. Patients with severe LAD-I typically show very diminished CD11a expression. Infants with severe LAD-I suffer from recurrent, life-threatening bacterial, and fungal infections that respond poorly to antimicrobials and require frequent hospitalizations. In theU.S., the incidence of LAD-I is estimated to range from approximately one in 100,000 to one in 200,000 live births, with roughly two-thirds of affected patients classified as having the severe form of the disease.
“As a clinician, I have seen firsthand the serious impact that severe LAD-I can have on young children and their families,” saidDonald B. Kohn, M.D., Principal Investigator of the Phase 1/2 study and Distinguished Professor of Microbiology, Immunology & Molecular Genetics at theUniversity of California, Los Angeles(UCLA). “The approval of KRESLADI represents the culmination of many years of scientific research and clinical collaboration aimed at addressing the underlying cause of this devastating disease.”
“The approval of KRESLADI represents a significant development for individuals affected by severe LAD-I and the broader primary immunodeficiency community,” saidVanessa Tenembaum, Chief Executive Officer of theJeffrey Modell Foundation, a global nonprofit organization dedicated to early diagnosis and treatments for primary immunodeficiency. “For families impacted by this rare and serious disease this approval underscores the importance of continued efforts to improve outcomes for patients with primary immunodeficiencies.”
More information is available for patients, families, and healthcare providers in theU.S.at www.KRESLADI.com, including full Prescribing Information.
Research supporting the development of KRESLADI was made possible in part by funding from theCalifornia Institute for Regenerative Medicine(Grant Number CLIN2-11480).
AboutRocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully integrated biotechnology company advancing gene therapies for rare and devastating cardiovascular diseases, with additional programs in hematology and immunology. Rocket’s cardiovascular pipeline includes three clinical stage programs that each target one of the major inherited cardiomyopathy subtypes: hypertrophic, arrhythmogenic, and dilated cardiomyopathies. Together these conditions represent more than 100,000 patients in the U.S. and EU. The Company’s platform is supported by proprietary AAV manufacturing capabilities, multi-year efficacy and safety data in cardiac gene therapy, and experience treating several cardiac patients across late-stage AAV programs.