The Latest Cardiac and Vascular News
CRANBURY, N.J.–(BUSINESS WIRE)–Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast […]
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 […]
First clinical gene therapy program for PKP2-ACM, a devastating inherited heart disease affecting approximately 50,000 people in the U.S. and EU Robust preclinical proof of concept studies showed RP-A601 decreased arrhythmias and increased survival in the PKP2 knockout mouse model Initiating […]
Continued to advance leading pipeline of AAV cardiology and LV hematology assets; all 2023 milestones remain on track Updates on all four clinical programs and PKP2-ACM preclinical program to be presented at the 26th Annual Meeting of the American Society of Gene […]
RP-A601 for Arrhythmogenic Cardiomyopathy due to PKP2 pathogenic variants (PKP2-ACM) represents meaningful commercial opportunity with estimated prevalence of 50,000 adults and children in the U.S. and EU; IND filing anticipated in Q2 2023 In support of Phase 2 pivotal study, […]
Acquisition further extends Rocket’s leadership in AAV-based cardiac gene therapy and expands focus and capabilities to BAG3-associated dilated cardiomyopathy CRANBURY, N.J.–(BUSINESS WIRE)–Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for […]
— Positive updates from Phase 1 clinical trial in Danon Disease showed RP-A501 was well tolerated with evidence of durable treatment effect and disease improvement for both pediatric patients and adult patients with up to nine and 36 months of follow-up, respectively— […]
CRANBURY, N.J.–(BUSINESS WIRE)–Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of investigational genetic therapies for rare childhood disorders with high unmet need, announced today that it has commenced an underwritten public offering of […]
— Pediatric efficacy data for both patients show initial improvements across clinical, functional and biomarker endpoints with six to nine months of follow-up; positive results including protein expression obtained at three and six months consistent with adult cohorts at similar timeframe— […]
