Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...

The disease leads to the progressive growth of fluid-filled cysts in the kidneys, often resulting in kidney failure and other ...

The Food and Drug Administration paused trials for two experimental gene therapies from Regenxbio after one child developed a ...

Studies of Hunter and Hurler syndromes on hold ...

A five-year-old patient dosed with RGX-111 has developed a brain tumour four years after being dosed with the gene therapy.

Lilly and German biotech Seamless Therapeutics will develop recombinase-based treatments for hearing loss.

University of California San Diego-led team has discovered that restoring a key cardiac protein called connexin‑43 in a mouse ...

Under the agreement, participating states will receive “discounts and rebates” from the drugmakers if the treatments don’t ...

Lilly inks gene therapy deal with Seamless Therapeutics to develop programmable recombinase treatments targeting genetic hearing loss worldwide.

(Yicai) Jan. 28 -- Chinese medical researchers have made a breakthrough in gene therapy that overcomes the limitations of ...

By Kamal Choudhury Jan 27 (Reuters) - Intellia Therapeutics said on Tuesday that the U.S. drug regulator has lifted a ...

After finding an intraventricular tumor in one clinical trial participant, the FDA placed a clinical hold on an experimental ...