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CRANBURY, N.J. - The U.S. Food and Drug Administration has accepted Rocket Pharmaceuticals’ (NASDAQ:RCKT) resubmitted Biologics License Application for KRESLADI (marnetegragene autotemcel), a gene therapy targeting severe Leukocyte Adhesion Deficiency-I (LAD-I), the company announced in a press release. According to InvestingPro data, the clinical-stage company maintains a strong liquidity position with a current ratio of 6.39, holding more cash than debt on its balance sheet.
The FDA set March 28, 2026, as the Prescription Drug User Fee Act target action date for the therapy, which aims to treat the rare genetic immune disorder that is typically fatal in childhood without a bone marrow transplant.
The BLA submission is supported by data from a global Phase 1/2 study that demonstrated 100% overall survival at 12 months post-infusion for all enrolled patients. According to the company, the trial met all primary and secondary endpoints, with patients showing substantial reductions in significant infections compared to pre-treatment levels.
"We value the continued dialogue with the FDA and believe the BLA moves Rocket closer to our goal of delivering a one-time gene therapy to patients facing the devastating effects of severe LAD-I," said Gaurav Shah, Chief Executive Officer of Rocket Pharmaceuticals. Despite showing a significant 10.5% return over the last week, the company’s stock, currently trading at $3.47, has experienced substantial volatility, with analysts setting price targets ranging from $2 to $16.
KRESLADI is an investigational therapy that uses modified patient-derived stem cells to deliver a functional copy of the ITGB2 gene, which produces a key protein that helps white blood cells fight infection.
The therapy holds several regulatory designations, including Regenerative Medicine Advanced Therapy, Rare Pediatric, and Fast Track designations in the U.S., as well as PRIME and Advanced Therapy Medicinal Product designations in the EU. InvestingPro analysis indicates the company is currently undervalued, though it faces challenges with cash burn and profitability. Get access to 8 more exclusive ProTips and comprehensive financial analysis through InvestingPro’s detailed research report.
If approved, Rocket would be eligible to receive a Rare Pediatric Disease Priority Review Voucher, which can be used to expedite review of a future drug application or sold to another company. With a market capitalization of $374 million and analysts not anticipating profitability this year, investors can access detailed financial health metrics and growth projections through InvestingPro’s comprehensive research platform.
In other recent news, Rocket Pharmaceuticals announced that it has voluntarily withdrawn its Biologics License Application (BLA) for RP-L102, an investigational gene therapy for Fanconi Anemia, from the U.S. Food and Drug Administration. This decision aligns with the company’s strategic shift to focus on programs with clearer regulatory and commercial pathways. As a result, Rocket Pharmaceuticals has ceased new internal investment in RP-L102. Additionally, the company had previously withdrawn its Marketing Authorization Application with the European Medicines Agency in July 2025. In related developments, Leerink Partners lowered its price target for Rocket Pharmaceuticals to $7.00 from $9.00, maintaining a Market Perform rating. Meanwhile, Cantor Fitzgerald reiterated an Overweight rating with a price target of $8.00, despite recent challenges in the company’s Danon disease clinical trial. Furthermore, Rocket Pharmaceuticals reported board changes, with Pedro Granadillo and Dr. Gotham Makker resigning from their positions. The company clarified that these resignations were not due to any disagreements with its operations or policies.
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