A 13-year-old boy with a rare genetic condition called Danon disease, which affects only about 300 families worldwide, was diagnosed with severe heart failure and required a heart transplant. His mother, who also has the disease, and he are among the few documented African-American cases. The story highlights the importance of support systems for young transplant patients, leading to the creation of a virtual community called Transplant Teenz to help teenagers cope with their condition.
A patient died following an experimental gene therapy trial by Rocket Pharmaceuticals for Danon disease, prompting the FDA to halt the study and investigate the cause, with concerns centered around a novel immune suppression agent used in the treatment regimen.
Rocket Pharmaceuticals' pivotal gene therapy trial for Danon disease has been put on hold by the FDA following a patient's death linked to complications from the therapy, prompting the company to review its safety protocols and extend its cash runway while in discussions with regulators.
A participant in Rocket Pharmaceuticals' gene therapy trial for Danon disease died, with the company investigating the cause, possibly related to immune suppression drugs used before therapy.
Rocket Pharmaceuticals announced an update on its Phase 2 trial of RP-A501 for Danon disease after a patient experienced a serious adverse event and subsequently passed away, leading to a clinical hold by the FDA. The company is investigating the cause, working with regulators and stakeholders, and has paused dosing to ensure patient safety. The trial aims to evaluate the efficacy of RP-A501, a gene therapy targeting cardiac function in Danon disease, with ongoing efforts to resume the study.
Rocket Pharmaceuticals plans to raise $175 million through a stock sale after the FDA agreed to its proposal for accelerated approval of its gene therapy for Danon disease based on a 12-patient study. The company's stock surged by 42% following the announcement. Danon disease is a rare X-linked disorder that weakens the heart and skeletal muscles, with males typically living until around 20 years old and females until around 35 or 40. Rocket's treatment delivers a functional version of the LAMP2 gene using a viral capsid called an AAV.
