Rocket Pharmaceuticals stock price rises on FDA approval of gene therapy

LootboxPhobia · 2026-03-27T16:45:31+00:00

Rocket Pharmaceuticals' gene therapy KRESLADI receives FDA accelerated approval for the treatment of children with severe leukocyte adhesion deficiency type I, and the stock price rose 6%. This therapy is the first gene treatment targeting this disease and is expected to enhance the company's financial flexibility and shareholder value.

LootboxPhobia

2026-03-27 16:45:31

Abstract generation in progress

Investing.com – Rocket Pharmaceuticals (NASDAQ: RCKT) stock rose 6% on Friday after the U.S. Food and Drug Administration accelerated the approval of the company’s gene therapy KRESLADI for the treatment of pediatric patients with severe leukocyte adhesion deficiency type I.

KRESLADI is an autologous hematopoietic stem cell-based gene therapy designed for pediatric patients with severe LAD-I caused by ITGB2 biallelic variants, who are unable to receive an allogeneic hematopoietic stem cell transplant from a human leukocyte antigen-matched sibling donor. This approval marks the first time the FDA has approved a gene therapy for the treatment of children with severe LAD-I.

The accelerated approval is based on increased surface expression of neutrophil CD18 and CD11a. Confirmation of clinical benefit will depend on ongoing clinical studies and long-term follow-up data from post-marketing registries.

Severe LAD-I is an extremely rare, life-threatening pediatric genetic immunodeficiency disorder caused by mutations in the ITGB2 gene. The disease is characterized by recurrent infections, and without treatment, the early mortality rate in children is very high. In the U.S., the incidence of LAD-I is estimated to occur in approximately 1 in every 100,000 to 200,000 live births, with about two-thirds of patients classified as severe.

With this approval, the FDA granted Rocket Pharmaceuticals a priority review voucher for rare pediatric diseases. The company stated that it plans to evaluate strategic options to monetize this voucher to enhance financial flexibility and maximize shareholder value.

Rocket Pharmaceuticals CEO Gaurav Shah stated, “The approval of KRESLADI is a significant milestone for the severe LAD-I community.”

Research supporting the development of KRESLADI was partially funded by the California Institute for Regenerative Medicine.

This article was translated with the assistance of artificial intelligence. For more information, please see our terms of use.

View Original

This page may contain third-party content, which is provided for information purposes only (not representations/warranties) and should not be considered as an endorsement of its views by Gate, nor as financial or professional advice. See Disclaimer for details.

1 Likes