Rocket Pharmaceuticals Presents Positive Data from LV Hematology Portfolio at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT)
“The positive updates presented at this year’s annual meeting demonstrate the sustained safety and efficacy across the totality of our LV hematology portfolio,” said Jonathan Schwartz, M.D., Chief Medical & Gene Therapy Officer, Rocket Pharmaceuticals.
- “The positive updates presented at this year’s annual meeting demonstrate the sustained safety and efficacy across the totality of our LV hematology portfolio,” said Jonathan Schwartz, M.D., Chief Medical & Gene Therapy Officer, Rocket Pharmaceuticals.
- In our pivotal studies of RP-L102 for Fanconi Anemia, we continue to see maintained genetic and phenotypic correction combined with hematologic stabilization.
- Additionally, our Phase 1 study of RP-L301 for PKD shows sustained clinically meaningful hemoglobin improvement in all patients.
- The oral presentation includes positive, updated data (cut-off September 11, 2023) from the global Phase 1/2 pivotal studies of RP-L102, Rocket’s ex vivo LV gene therapy candidate for FA.