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 ...

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

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

Intellia Therapeutics said on Tuesday that the U.S. drug regulator has lifted a clinical hold on one of its late-stage gene ...

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

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, ...

Opus Genetics is conducting a trial for OPGx-MERTK gene therapy in Abu Dhabi, targeting MERTK-related retinitis pigmentosa ...

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

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

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.

Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for a progressive muscle‑wasting disorder showed sustained benefit in ...