DANON DISEASE

Rocket Pharmaceuticals Announces Positive Updates from Phase 1 Clinical Trial of RP-A501 in Danon Disease

Retrieved on: 
Monday, November 15, 2021
Health, Stem Cells, Clinical Trials, Other Health, RP-A501, Danon Disease, Rocket Pharmaceuticals, Inc., RP-A501, DANON DISEASE, ROCKET PHARMACEUTICALS, INC.

Male patients living with Danon Disease suffer a heavy disease burden and face rapidly progressive heart failure in their teenage years.

Key Points: 
  • Male patients living with Danon Disease suffer a heavy disease burden and face rapidly progressive heart failure in their teenage years.
  • The clinical data to date also demonstrate encouraging results including clinical stabilization of this rapidly progressive disease.
  • Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition.
  • Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes.

Rocket Pharmaceuticals Announces Danon Disease Presentation at the American Heart Association (AHA) Scientific Sessions 2021

Retrieved on: 
Monday, November 8, 2021
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Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that updated data from the ongoing Phase 1 clinical trial of RP-A501 in Danon Disease will be presented at the American Heart Association (AHA) Scientific Sessions 2021 taking place virtually November 13-15, 2021.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that updated data from the ongoing Phase 1 clinical trial of RP-A501 in Danon Disease will be presented at the American Heart Association (AHA) Scientific Sessions 2021 taking place virtually November 13-15, 2021.
  • RP-A501 is an investigational gene therapy product being developed for Danon Disease and the first potential gene therapy for monogenic heart failure.
  • Danon Disease is a rare X-linked inherited disorder caused by mutations in the gene encoding lysosome-associated membrane protein 2 (LAMP-2), an important mediator of autophagy.
  • Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition.

Rocket Pharmaceuticals Presents Clinical Data from RP-A501 Trial in Danon Disease at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2021

Retrieved on: 
Tuesday, September 14, 2021
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Steroid-induced myopathy was observed in two of the three patients >2 weeks after dosing, which also resolved.

Key Points: 
  • Steroid-induced myopathy was observed in two of the three patients >2 weeks after dosing, which also resolved.
  • In these two patients, a substantial improvement of a key marker of heart failure, B-type natriuretic peptide (BNP), was also observed.
  • RP-A501 is an investigational gene therapy product being developed for Danon Disease and the first potential gene therapy for monogenic heart failure.
  • Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition.

Rocket Pharmaceuticals Announces FDA Lifts Clinical Hold on Danon Disease Trial of RP-A501

Retrieved on: 
Monday, August 16, 2021
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We are grateful for the collaboration between the FDA and our team in reaching agreement on protocol updates allowing us to resume patient enrollment in our Danon Disease trial.

Key Points: 
  • We are grateful for the collaboration between the FDA and our team in reaching agreement on protocol updates allowing us to resume patient enrollment in our Danon Disease trial.
  • We look forward to progressing this critical work on behalf of all Danon patients, said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma.
  • Rockets Danon Disease program was placed on clinical hold by the FDA in May of 2021 to modify the study protocol and other supporting documents with revised guidelines for patient selection and safety management.
  • Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition.