Company expects up to six-month review period per FDA guidelines New longer-term clinical data demonstrating durable positive brain biochemical ...

Scientists at the Broad Institute and Mass General Brigham have built a generative AI model that creates short DNA segments ...

It’s now possible to treat inherited blood diseases, such as sickle cell disease, with gene editing. Blood stem cells are extracted from the patient, modified, and infused back into their bone ...

Market OverviewThe global cell and gene therapy market is undergoing rapid and transformative growth, reflecting its increasing importance in modern healthcare.The market is projected to expand at a ...

Fractyl Health is developing a shot that would program the body to make more of the GLP-1 hormone naturally, a risky bet that it can provide a longer-lasting benefit than blockbuster weight-loss drugs ...

A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the burden of lifelong transfusions. A single one-time gene therapy could free ...

US biotech firm Fractyl Health is developing Rejuva, an experimental gene therapy that may mimic GLP-1 drugs for years after ...

A revolutionary treatment brings new hope to those suffering from a devastating rare skin disorder, while showcasing an innovative funding model that could transform rare disease research In a ...

Fractyl Health, Inc. announced promising preclinical results for its gene therapy RJVA-001 at the 2025 ASGCT Annual Meeting, showing that a single dose leads to substantial metabolic improvements ...

Retiree Chip Hailey, who has a severe form of hemophilia B, participated in a groundbreaking gene therapy trial. Hailey experienced a life-threatening delay in treatment after a car accident, ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.