Company expects up to six-month review period per FDA guidelines New longer-term clinical data demonstrating durable positive brain biochemical ...

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

Orphan Drug Designation applies to approximately 50,000 Patients who predominantly suffer from severe chronic pain due ...

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

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 ...

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 ...

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

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

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