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Rocket Pharmaceuticals Presents Positive Updates on FA and LAD-I Gene Therapy Programs at the 23rd Annual Meeting of the American Society of Gene and Cell Therapy
—Follow-up of 12 to 36 Months for FA RP-L102 “Process A” Demonstrates Prolonged Engraftment and Continued Hematologic Correction— —Sustained Efficacy in
About this update from Rocket Pharmaceuticals, Inc.
[{"type":"text","content":"\n—Follow-up of 12 to 36 Months for FA RP-L102 “Process A” Demonstrates Prolonged Engraftment and Continued Hematologic Correction—\n\n\n—Sustained Efficacy in LAD-I Provides Initial Proof of Concept for Lentiviral Platform and “Process B” Manufacturing—\n\n\n—Longer-term “Process B” Data for FA and LAD-I on Track for End of Year—\n\n NEW YORK--(BUSINESS WIRE)--\nRocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders, today presents new clinical data supporting longer-term efficacy and durability of gene therapy for Fanconi Anemia (FA) and Leukocyte Adhesion Deficiency-I (LAD-I) at the 23rd Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) being held virtually May 12-15, 2020. Two oral presentations highlight updates from the company’s Phase 1/2 study of RP-L201 for the treatment of severe LAD-I and the Phase 1/2 study of RP-L102 “Process A” for the treatment of FA.\n\n\n“The latest additional data from both our LAD-I and FA programs demonstrate sustained engraftment and durable clinical impact,” said Jonathan D. Schwartz, M.D., Chief Medical Officer and Senior Vice President of Rocket. “These results further support the viability of gene therapy in LAD-I and FA, disorders in which bone marrow transplant is the primary curative option and is associated with high rates of toxicity.”\n\n\n“Patients with severe LAD-I have neutrophil CD18 expression of less than 2% of normal, with extremely high mortality in early childhood,” said Dr. Schwartz. “In this first patient treated with RP-L201 using ‘Process B’, an increase from less than 1% to 47% CD18 expression sustained over six months demonstrates that RP-L201 has the potential to correct the neutrophil deficiency that is the hallmark of LAD-I. We are also pleased with the continued visible improvement of multiple disease-related skin lesions. These results lend further support to the applicability of ‘Process B’ across the lentiviral portfolio. The second patient has also recently been treated, and we look forward to completing the Phase 1 portion of the registrational trial for this program.”\n\n\nDr. Schwartz continued, “In our FA program, patients followed for a year or more after treatment with RP-L102 ‘Process A’ continue to demonstrate durabl...