Rocket Pharma Updates Positive Prelim Data from Phase 1

 Rocket Pharmaceuticals (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces updated preliminary clinical data from its Phase 1 trial of RP-L301 for the treatment of Pyruvate Kinase Deficiency (PKD), showing durable normalization of hemoglobin levels up to 6 months following therapy and similar 3-month trends in the second patient treated. Additionally, results showed sustained safety and tolerability of RP-L301 6 months after treatment.

"The positive updates on our PKD trial represent continued validation of RP-L301’s robust potential benefit in addressing the challenges of PKD. We are pleased to report that updated patient 1 results show that RP-L301 continues to safely normalize hemoglobin levels as demonstrated by an increase from an average of approximately 7.4 g/dL at baseline to 13.9 g/dL at 6-months post-treatment with no transfusion requirements after hematopoietic reconstitution," said Jonathan Schwartz, M.D. Chief Medical Officer and Senior Vice President of Rocket. "A similar trend in hemoglobin and bilirubin normalization were observed at three months post-treatment in patient 2. We are excited to share this data as results show the potential of RP-L301 to address the extensive burden PKD places on patients and their families. We look forward to presenting more comprehensive and updated data at a scientific venue this year."

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