A central nervous system (CNS) tumor has prompted the FDA to place clinical holds on two Regenxbio gene therapies, including ...

The development of a brain tumor in a study participant led regulators to suspend a Hurler syndrome therapy in early testing ...

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

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

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

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

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

When Genenta Science listed on the Nasdaq at the tail end of 2021, the Milan-based biotech was squarely focused on its pipeline of cell-based gene therapies. | Four years after listing on the Nasdaq, ...

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

A new CRISPR-based one-off procedure that lowers "bad" cholesterol has been approved to enter Phase I human trial. If ...

After a spate of patient deaths in 2025 linked to the company’s Duchenne gene therapy, Sarepta shared new data showing benefits of the therapy three years after dosing.

Teenage boy revels in ‘freedom’ after £1.65 million gene-editing treatment - Adam Rehman underwent treatment after previously relying on monthly blood transfusions ...