RP-L301 / Rocket Pharma - LARVOL DELTA

RP-L301, a Lentiviral-Mediated Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase 2 Clinical Trial Design (ASH 2024) - P2 | "Mitapivat, an allosteric PKLR activator, is approved for the treatment of adult patients, however hemoglobin increases of at least 1.5 g/dL were infrequent in splenectomized patients and/or those with more severe anemia...Autologous CD34+ hematopoietic cells are mobilized using granulocyte-colony stimulating factor (G-CSF) and plerixafor and then collected via apheresis, followed by CD34+ cell enrichment by immunoselection and transduction with the LV containing coRPK. RP-L301 is infused following myeloablative busulfan conditioning...Approximately 10 patients will participate. Conclusion : Based on the favorable efficacy and safety data from the Phase 1 study, this registrational Phase 2 study is designed to enable comprehensive evaluation of the potential of RP-L301 autologous gene therapy to substantively reverse the disease phenotype in pediatric and adult patients with PKD."

Clinical • Gene therapy • P2 data • Anemia • Cardiovascular • Gene Therapies • Hematological Disorders • Hepatology • Infectious Disease • Pediatrics • CD34

Gene Therapy for Adult and Pediatric Patients with Severe Pyruvate Kinase Deficiency: Results from a Global Study of RP-L301 (ESGCT 2024) - P1 | "Myeloablative therapeutic drug monitoring-guided busulfan was administered prior to RP-L301 intravenous infusion. RP-L301 is a potential treatment for patients with severe PKD, including those who did not derive benefit from available therapies (including splenectomy and mitapivat). Robust and sustained efficacy up to the latest follow-up visit (up to 36 months post-treatment) is demonstrated by clinically significant improvement in hemoglobin and absence of transfusion requirements."

Clinical • Gene therapy • Anemia • Fatigue • Gene Therapies • Pediatrics

RP-L301-0119: Gene Therapy for Pyruvate Kinase Deficiency (PKD) (clinicaltrials.gov) - P1 | N=4 | Completed | Sponsor: Rocket Pharmaceuticals Inc. | Active, not recruiting ➔ Completed

Trial completion • Gene Therapies • Pediatrics

CHANGING THE NATURAL HISTORY OF PYRUVATE KINASE DEFICIENCY WITH LENTIVIRAL-MEDIATED GENE THERAPY: RESULTS OF A GLOBAL PHASE 1 STUDY (EHA 2024) - P1 | "Myeloablative therapeutic drug monitoring-guided busulfan was administered prior to RP-L301 IVinfusion. RP-L301 is a potential treatment for patients with severe PKD, including those who didnot derive benefit from available therapies (including splenectomy and mitapivat). Robust and sustainedefficacy up to 36 months post-treatment is demonstrated by normalized Hb (adult cohort) and clinicallysignificant Hb increases (pediatric cohort) with both cohorts demonstrating improved hemolysis parameters,with no transfusion requirements after engraftment."

Clinical • Gene therapy • P1 data • Anemia • Fatigue • Gene Therapies • Hematological Disorders • Pediatrics • HP

RP-L301-0124: Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency (clinicaltrials.gov) - P2 | N=10 | Not yet recruiting | Sponsor: Rocket Pharmaceuticals Inc.

New P2 trial • Gene Therapies