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

About this update from Rocket Pharmaceuticals, Inc.

—Longer-Term Data from the Registration-Enabling Phase 1/2 Trial for Leukocyte Adhesion Deficiency-I Demonstrate Safety and Efficacy of RP-L201— —Preclinical Data From RP-L401 for Infantile Malignant Osteopetrosis Support Accelerated Clinical Development— —Results Provide Further Validation for Rocket’s “Process B” Lentiviral Platform— NEW YORK--(BUSINESS WIRE)-- 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 expression to 4-10% is associated with survival into adulthood. The two patients enrolled in our Phase 1 trial demonstrated restored CD18 expression substantially exceeding this threshold. In addition, we continue to observe a durable treatment effect in the patient followed through one year, with improvement of multiple disease-related skin lesions after therapy and no further requirements for prophylactic anti-infectives.” Dr. Schwartz continued, “In addition, preclinical results in Infantile Malignant Osteopetrosis represent an early positive signal of in vivo efficacy that support evaluation of RP-L401 in a Phase 1 trial. IMO is a devastating bone resorption disorder resulting in skeletal deformities, neurologic abnormalities and bone marrow failure. Rocket has developed RP-L401 as a potential treatment option to prevent the devastating morbidity and childhood mortality associated with IMO. Preclinical data indicate that even a modest leve...

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