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Stock impact report

Rocket Pharmaceuticals secures FDA approval for gene therapy KRESLADI [Yahoo! Finance]

Rocket Pharmaceuticals, Inc. (RCKT) 
Last rocket pharmaceuticals, inc. earnings: 3/5 07:00 am Check Earnings Report
US:NASDAQ Investor Relations: rocketpharma.com
Company Research Source: Yahoo! Finance
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. The approval, based on increased neutrophil CD18 and CD11a surface expression, is conditional and requires confirmation of long-term clinical benefit through ongoing studies and a post-marketing registry. Alongside the approval, Rocket received a Rare Pediatric Disease Priority Review Voucher (PRV), which the company may monetize to enhance financial flexibility. Severe LAD-I is an ultra-rare genetic immunodeficiency that leads to recurrent, life-threatening infections in early childhood. Incidence in the US is estimated at one in 100,000 to 2 Show less Read more
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