The FDA previously placed two clinical studies on hold, including the Phase III trial in which the liver toxicity occurred.

Scientists at The University of Texas at Austin have developed a revolutionary gene-editing method using bacterial retrons ...

Your heart will not just give you fear and uncertainty after a heart attack; it can leave scar tissue that stiffens your ...

Innovation Passport award facilitates accelerated time to market and helps expedite patient access to transformative new ...

Children with hereditary deafness regained their hearing thanks to a type of gene therapy, a new study published on Wednesday found. In a clinical trial, co-led by investigators from Mass Eye and Ear, ...

A new West Coast biotech has emerged into the busy in vivo cell therapy space, this time with the backing of Nobel Prize-winning CRISPR pioneer Jennifer Doudna, Ph.D. Azalea Therapeutics has bloomed ...

However, in late September 2025, scientists successfully treated Huntington’s for the first time in a ground-breaking study.

In a world first, a bespoke gene-editing therapy benefited one child. Now researchers plan to launch a clinical trial of the ...

Researchers in the United Kingdom say they have successfully trialed what could become the world's first gene therapy for Huntington's disease – a fatal neurodegenerative disorder that is typically ...

Emily Kramer-Golinkoff can’t get enough oxygen with each breath. Advanced cystic fibrosis makes even simple things like walking or showering arduous and exhausting. She has the most common fatal ...

Sarepta stock crashed Tuesday after the biotech company said two of its muscular dystrophy drugs failed confirmatory tests.

The patient who was hospitalized after receiving Intellia Therapeutics’ CRISPR therapy nex-z has died. | The patient who was ...