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Rocket Pharmaceuticals Announces IMPD Clearance of RP-L301 Gene Therapy for Pyruvate Kinase Deficiency
–Rocket’s Largest Lentiviral Pipeline Opportunity with an Estimated Prevalence of 3,000 to 8,000 Patients in the U.S. and EU– –Phase 1 Clinical Trial to
About this update from Rocket Pharmaceuticals, Inc.
[{"type":"text","content":"\n –Rocket’s Largest Lentiviral Pipeline Opportunity with an Estimated Prevalence of 3,000 to 8,000 Patients in the U.S. and EU–\n\n\n–Phase 1 Clinical Trial to Commence in the Fourth Quarter–\n\n NEW YORK--(BUSINESS WIRE)--\nRocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a leading U.S.-based multi-platform clinical-stage gene therapy company, today announces Investigational Medicinal Product Dossier (IMPD) clearance from the Spanish Agency for Medicines and Health Products (AEMPS) for RP-L301. RP-L301 is the Company’s lentiviral vector (LVV)-based gene therapy for the treatment of Pyruvate Kinase Deficiency (PKD) that was in-licensed from the Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER) and Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD).\n\n\n“We are excited to advance the first gene therapy for PKD to the clinic following the clearance of the IMPD from the AEMPS for RP-L301,” said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. “This news is particularly timely for Rocket as it follows our sponsorship and participation in the Patient Focused Drug Development Meeting on PKD held by NORD last week, as well as the 3rd Annual PKD Patient’s Forum in May. At these events, we gained insights into the impact of PKD on the daily life of patients, and the challenges of current therapies. We continue to be highly committed to working closely with patients, families and physicians in addressing many of these issues with a potentially curative gene therapy option.”\n\n\n“In preclinical models, RP-L301 reverses the hemolytic phenotype, including normalization of splenomegaly and reduction of anemia and reticulocytosis, when at least 20-30% of bone marrow progenitor cells are genetically corrected,” said Dr. José-Carlos Segovia, scientific leader of the project and PKD Scientific Advisor. “We look forward to investigating the potential of this new, single-administration treatment option for patients who are desperately in need of promising and potentially definitive therapy.”\n\n\nThe planned open-label, single-arm, Phase 1 clinical trial of RP-L301 will enroll a total of six adult and pediatric transfusion-dependent PKD patients in Europe and the U.S. The trial will be separated int...