Rocket Pharmaceuticals Inc. (RCKT) has achieved a major regulatory milestone as the FDA has granted accelerated approval to KRESLADI, the company's one-time gene therapy for severe Leukocyte Adhesion Deficiency-I.
Leukocyte Adhesion Deficiency-I (LAD-I) is a rare and life-threatening immunodeficiency caused by the absence of functional CD18 protein, leading to recurrent infections, impaired wound healing, and high early-childhood mortality.
KRESLADI (marnetegragene autotemcel) is an ex vivo lentiviral gene therapy designed to correct the underlying genetic defect by restoring CD18 expression in patients' own hematopoietic stem cells.
The approval is based on clinical data demonstrating restoration of CD18 and CD11a expression on neutrophils, reflecting improved immune function, and clinical benefits in treated pediatric patients. These biomarkers serve as surrogate endpoints reasonably likely to predict clinical benefit, with responses sustained through 24 months post-infusion and supported by reductions in infection-related complications.
This marks Rocket's second attempt to secure approval for KRESLADI. The FDA previously issued a Complete Response Letter (CRL) on June 28, 2024, requesting limited additional Chemistry Manufacturing and Controls (CMC) information to complete its review. Rocket resubmitted the BLA last year after addressing the agency's concerns.
KRESLADI received multiple expedited designations- including Orphan Drug, Rare Pediatric Disease, Regenerative Medicine Advanced Therapy (RMAT), and Fast Track- reflecting the high unmet need and the therapy's potential to deliver meaningful clinical benefit.
With approval, Rocket also received a Rare Pediatric Disease Priority Review Voucher, adding further strategic value to the program.
As part of the accelerated approval, Rocket must conduct confirmatory studies to verify long-term clinical benefit, and continued approval will depend on the outcomes of these post-marketing trials.
According to the FDA, KRESLADI marks the first gene therapy ever approved for LAD-I, offering a potential curative alternative for children who otherwise rely on high-risk allogeneic stem cell transplantation.
We alerted our readers on January 15, 2026, when RCKT was trading at $3.96.
RCKT has traded between $2.19 and $8.26 over the past year. The stock closed Thursday's trading at $4.69, up 0.21%. In pre-market, the stock rose further to $5.13, up 9.38%.
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