CRISPR gene therapy Casgevy can nearly eliminate sickle cell crises, but difficult stem‑cell collection is delaying access ...

A gene therapy that patients breathe in has been found to shrink lung tumours by inserting immune-boosting genes into ...

The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are ...

Cell and gene therapies , or CGT , have come a long way since they were first introduced. In the last few decades, both cell therapy -- the transplantation of living cells -- and gene therapy -- the ...

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Gene therapy approaches include gene replacement, suppression, and editing, each matched to specific genetic mechanisms in hereditary hearing loss. Preclinical studies in rodent models show promise, ...

Life Biosciences is pushing cellular rejuvenation into the clinic with ER-100, an experimental gene therapy designed to restore vision by reviving damaged neurons in the eye. Rather than slowing ...

A lower court had previously ruled for Sarepta in the companies’ long-running dispute, finding that REGENXBIO’s AAV patent ...

People looking to lose weight and lower their blood sugar may someday be able to get a single injection that turns their cells into tiny factories churning out a protein that is essentially the active ...

A patient has died in a clinical trial evaluating a Rocket Pharmaceuticals gene therapy shortly after complications emerged that led the FDA to pause the study. An inquiry into the fatality is ...

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.