Gene Therapy Pipeline Update: Rocket Pharmaceuticals Receives IND Clearance for Gene Therapy for Pyruvate Kinase Deficiency

Rocket Pharmaceuticals, a New York-based, clinical-stage company, developing first-in-class gene therapies for rare and devastating inherited diseases announced clearance of Investigational New Drug (IND) by the FDA for RP-L301 gene therapy. RP-L301 is a lentiviral vector (LVV)-based gene therapy for the treatment of Pyruvate Kinase Deficiency (PKD), a genetic rare disease. Last month, the company received the Investigational Medicinal Product Dossier (IMPD) clearance from the Spanish Agency for Medicines and Health Products (AEMPS) for RP-L301

According to the company’s CEO, “RP-L301 represents the first gene therapy candidate in development for PKD, a hematologic disorder in which the current treatment options, chronic blood transfusions, and splenectomy, are associated with burdensome side effects and end-organ damage.”

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Last month, the company registered a phase I clinical trial (NCT04105166), for which the recruitments are yet to begin. The drug is being developed for PKD deficiency, a genetic disorder, which is caused by a mutation in the PKLR gene. This gene contains instructions for creating a specialized protein (enzyme) known as pyruvate kinase, which is vital for the breakdown of adenosine triphosphate (ATP) – the energy-carrying-molecule.

Over the last few years, gene therapies have proven to be effective tools in curing genetic disorders. Yesterday, Homology Medicines, a company using proprietary gene editing and gene therapy platform announced the presentation of preclinical data supporting its investigational gene therapy programs for the treatment of metachromatic leukodystrophy (MLD) and phenylketonuria (PKU).

The ongoing success ride of the approved gene therapies has increased expectations in the overall gene therapy market, which is expected to be around $12 billion by 2030.

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