Get all premium features to the best stock analysis tool

Rocket Pharmaceuticals, Inc. (RCKT) Discusses FDA Approval of KRESLADI Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1 Transcript

Rocket Pharmaceuticals (NASDAQ:RCKT) announced that the US Food and Drug Administration (FDA) has granted accelerated approval for KRESLADI (marnetegragene autotemcel), the company's gene therapy for children with severe leukocyte adhesion deficiency-I (LAD-I) caused by biallelic variants in ITGB2. The therapy is intended for patients without an available human leukocyte antigen-matched sibling donor for allogeneic stem cell transplant.

Rocket Pharmaceuticals shares jumped 10% on Friday before the bell after the U.S. Food and Drug Administration approved its ​gene therapy for a rare and often fatal immune ‌disorder in children, marking the first regulatory green light for a treatment of the condition.

CRANBURY, N.J.--(BUSINESS WIRE)--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 the U.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 defici.

Rocket Pharmaceuticals, Inc. (RCKT) Presents at TD Cowen 46th Annual Health Care Conference Transcript

The broker maintains its 'outperform' rating and $16 price target on shares trading at $4.97 Wedbush Securities has reiterated its bullish stance on Rocket Pharmaceuticals, the rare disease gene therapy company, after its fourth-quarter update confirmed that patient dosing in its pivotal Phase 2 study of RP-A501 for Danon disease will resume in the first half of 2026. Danon disease is a rare and life-threatening inherited condition affecting the heart, muscles and brain, for which no approved gene therapy yet exists.

Rocket Pharmaceuticals reports narrower-than-estimated Q4 loss. The company provides key updates on its pipeline candidates.