Tag: Pharmaceuticals

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