The therapy is designed to address a particularly severe type of heart failure characterized by progressive enlargement of the heart’s ventricles and reduced ability to pump blood effectively.

The MUSIC-HFpEF trial is investigating SRD-002, a one-time gene therapy treatment for heart failure with preserved ejection fraction (HFpEF) delivered through a proprietary minimally invasive ...

First-in-human trial of AB-1002 designed to evaluate safety and preliminary efficacy in participants with New York Heart Association (NYHA) Class III heart failure Trial enrolled 11 participants ...

A new gene therapy shows promise in fighting Alzheimer’s by repairing brain cell function, not just clearing toxic proteins—potentially preserving memory even after symptoms appear.

Rocket Pharmaceuticals has shared early data from three patients that tie its gene therapy for an inherited heart disease to a well-tolerated safety profile, which has been heralded by analysts as ...

Biotechnology New Gene Therapy Reverses Three Diseases With Shots to the Bloodstream The treatments rescued damaged blood and immune cells in newborn mice with just one shot.

The mood at the annual meeting of the American Society of Cell and Gene Therapy isn’t amazing. The biotech market has been bad for years. But many researchers tried to project confidence.

Sarepta Therapeutics on Sunday said there had been a second reported case of acute liver failure resulting in death after a patient received the company's gene therapy for a rare form of muscular ...

Patients affected by BAG3 DCM have a mutation in the BAG3 gene and a deficiency in functioning BAG3 protein, resulting in early onset heart failure that progresses rapidly.

Sarepta Therapeutics said a second patient had died after receiving its $3.2 million gene therapy to treat Duchenne muscular dystrophy.

Rocket Pharmaceuticals adjusted the clinical trial design to include use of approved drug to avert an excessive immune response to its Danon disease gene therapy. The patient who died was one of ...

AskBio Presents Complete Results of Phase 1 Trial of AB-1002 Gene Therapy in Participants with Congestive Heart Failure at European Society of Cardiology Heart Failure Meeting ...