Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) is one of the best NASDAQ stocks under $5 to buy now. On October 14, Rocket Pharmaceuticals announced that the US FDA accepted the resubmission of the Biologics License Application/BLA for KRESLADI (marnetegragene autotemcel; marne-cel).
KRESLADI is a lentiviral vector/LV-based investigational gene therapy intended for the treatment of severe Leukocyte Adhesion Deficiency-I (LAD-I), which is a rare genetic immune disorder. The BLA resubmission is supported by positive clinical efficacy and safety data from the global Phase 1/2 study of KRESLADI.
The study achieved 100% overall survival at 12 months post-infusion (and for the entire duration of follow-up) for all enrolled patients. All primary and secondary endpoints were met, and the therapy was well tolerated with no treatment-related serious adverse events reported. Data showed substantial reductions in the incidence of significant infections compared to pre-treatment levels, along with evidence of improved skin lesions and restored wound-healing capabilities.
Rocket Pharmaceuticals Inc., together with its subsidiaries, operates as a late-stage biotechnology company that develops gene therapies for rare and devastating diseases in the US.
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This article is originally published at Insider Monkey.
