Rocket Pharma: Positive Data from LV Hematology and AAV Cardiovascular Gene Therapy Programs

 

• Robust and sustained efficacy in both adult PKD patients up to 30 months post-RP-L301; first pediatric patient results suggest efficacy similar to adult cohort with initial greater than five-point increase in hemoglobin
• Sustained genetic correction observed in eight of 12 evaluable Fanconi Anemia patients, and phenotypic correction and concomitant hematologic stabilization observed in seven of 12 patients 12-42 months after RP-L102 in Phase 2 pivotal trial
• 100% overall survival at 12 months post-RP-L201 and favorable safety profile for all LAD-I patients with 12-24 months of follow-up
• Robust preclinical proof of concept studies showed RP-A601 for PKP2-ACM decreased arrhythmias and increased survival

https://www.biospace.com/article/releases/rocket-pharmaceuticals-presents-positive-data-from-lv-hematology-and-aav-cardiovascular-gene-therapy-programs-at-the-26th-annual-meeting-of-the-american-society-of-gene-and-cell-therapy-asgct-/