Rocket Pharmaceuticals Announced That The U.S. Food And Drug Administration (Fda) Has Granted Regenerative Medicine Advanced Therapy (Rmat) Designation To Rp-L301, The Investigational Lentiviral-Based Gene Therapy For Pyruvate Kinase Deficiency (Pkd), A Rare Blood Disorder Characterised By Severe Anaemia And Excessive Red Blood Cell Breakdown.Results From The Rp-L301 Programme Demonstrated Robust Efficacy In Both Adult Patients For Up To 30 Months Post-Infusion Demonstrated By Normalised Haemoglobin (From Baseline Levels In The 7.0-7.5 G/Dl Range), Improved Haemolysis Parameters, Red Blood Cell Transfusion Independence And Improved Quality Of Life With Documented Improvements Via Formal Quality Of Life Assessments. Receiving Rmat Designation From The Fda For Rp-L301 Is A Major Achievement To Bring The First Potentially Curative Gene Therapy Treatment To Patients Living With Pkd Who Have High Unmet Need.Pyruvate Kinase Deficiency (Pkd) Is A Rare, Monogenic Red Blood Cell Disorder Resulting From A Mutation In The Pklr Gene Encoding For The Pyruvate Kinase Enzyme, A Key Component Of The Red Blood Cell Glycolytic Pathway. Patients With Pkd Have A High Unmet Medical Need, As Currently Available Treatments Include Splenectomy And Red Blood Cell Transfusions, Which Are Associated With Immune Defects And Chronic Iron Overload.
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