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

Studies of Hunter and Hurler syndromes on hold ...

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

Emil Kakkis and Ultragenyx Pharmaceutical are taking another swing at a potentially deadly genetic disease with their ...

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

By Mariam Sunny Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy slowed disease ...

Scientists have achieved a genetic feat once thought impossible: removing an entire extra human chromosome using gene-editing ...

Sanofi’s participation will support the progression of Sensorion’s genetic medicine pipeline.

The EMA’s positive opinion on Kygevvi marks a turning point for patients with TK2d, an ultra-rare genetic muscle disease previously managed only with supportive care.

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

Apis Capital Advisors’ Apis Flagship Fund released its Q4 2025 investor letter. A copy of the letter can be downloaded here. Apis Flagship Fund delivered strong performance in the fourth quarter of ...