Leukocyte adhesion deficiency (LAD) represents a group of rare, genetically determined immunodeficiency disorders that impair the ability of white blood cells to adhere to vascular endothelium, ...
CRANBURY, N.J.--(BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare ...
Rocket's clinical programs using lentiviral vector (LV) based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and ...
WAYNE, Pa. and ROCKVILLE, Md., Aug. 02, 2022 (GLOBE NEWSWIRE) -- Avalo Therapeutics, Inc. (AVTX), announced the first patient has been dosed in the pivotal LADDER trial (A Phase 3, Randomized, ...
Prescription Drug User Fee Act (PDUFA) target action date is March 28, 2026 All primary and secondary endpoints were met, and KRESLADI™ was well tolerated Rocket is eligible for a Rare Pediatric ...
RP-L201 has received FDA Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric, Fast Track and Orphan Drug designations; Rocket eligible for Priority Review Voucher, if RP-L201 is approved ...
Rocket Pharmaceuticals’ flight path to approval for Kresladi has been delayed again after the FDA issued a complete response letter (CRL) requesting additional manufacturing information. The biotech ...
CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare ...
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Investigational gene therapy gives children with rare immune disorder a new lease on life
An investigational gene therapy has successfully restored immune function in all nine children treated with the rare and life-threatening immune disorder called severe leukocyte adhesion deficiency-I, ...
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