Rocket Pharma Announces First Patient Treated in Phase 1 Trial of RP-L301 Gene Therapy for Pyruvate Kinase Deficiency

Rocket Pharmaceuticals, Inc. announces that the first patient has received investigational therapy in the open-label, Phase 1 clinical trial of RP-L301, the Company’s lentiviral vector (LVV)-based gene therapy for the treatment of Pyruvate Kinase Deficiency (PKD), a rare monogenic red blood cell disorder.

“We are pleased to have treated the first patient in our Phase 1 trial of RP-L301, marking an important step forward in addressing a high unmet need for new therapies,” said Jonathan D. Schwartz, M.D., Chief Medical Officer and Senior Vice President of Rocket. “PKD is a genetic disorder characterized by red cell destruction and anemia that can be severe or even life-threatening. Children are often most severely affected, and the current treatment options—chronic transfusions and splenectomy—are associated with burdensome side effects including iron overload and end-organ damage. We believe gene therapy treatment with RP-L301 has the potential to be a safe and transformative approach to improve long-term patient outcomes.”

The global Phase 1 open-label, single-arm, clinical trial is expected to enroll six adult and pediatric transfusion-dependent PKD patients in the U.S. and Europe. The trial will be comprised of three cohorts to assess RP-L301 in young pediatric (age 8-11), older pediatric (age 12-17) and adult populations. The trial is designed to assess the safety, tolerability and preliminary efficacy of RP-L301, and initial safety evaluation will occur in the adult cohort before evaluation in pediatric patients. Lucile Packard Children’s Hospital Stanford is the lead site in the U.S. for adult and pediatric patients. Hospital Infantil Universitario Nino Jesus is the lead site in Europe for pediatrics and Hospital Universitario Fundacion Jimenez Diaz is the lead site in Europe for adult patients.