Rocket Pharmaceuticals Presents Positive LAD-l Clinical Update and Comprehensive IMO Preclinical Review at the European Society for Immunodeficiencies 2020 Meeting – The Baytown Sun

NEW YORK–(BUSINESS WIRE)–Oct 16, 2020–

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces clinical data at the European Society for Immunodeficiencies (ESID) 2020 Meeting being held virtually October 14-17, 2020. An oral presentation provides positive longer-term follow-up data from the Phase 1/2 clinical trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I). An e-poster highlights preclinical study data on RP-L401 for Infantile Malignant Osteopetrosis (IMO) supporting clinical development of the trial.

“Today, Rocket presents positive results from our LAD-I gene therapy trial demonstrating further clinical benefit in this severely affected patient population,” said Jonathan Schwartz, M.D. Chief Medical Officer and Senior Vice President of Rocket. “Patients with LAD-I have markedly diminished expression of the integrin CD18 and suffer from life-threatening bacterial and fungal infections. Natural history studies indicate that an increase in CD18
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