Groundbreaking new research shows that it is possible to use gene therapy to restore hearing in both children and adults.

One 7-year-old girl who received a traditional cochlear implant in one ear and the gene therapy in another, recovered almost ...

Sarepta Therapeutics will cut 500 jobs and add a serious warning on the label of its muscle-disorder gene therapy Elevidys, the drugmaker said on Wednesday, following the recent deaths of two patients ...

Ultragenyx Pharmaceuticals RARE announced that the FDA has issued a Complete Response Letter (CRL) for its biologics license ...

While the CRL derails a near-term clearance, one analyst described the rejection as more of “a speed bump to approval, rather ...

This is the first time such results have been achieved in both children and adult patients born with a specific type of ...

Ultragenyx's UX111 gene therapy faces FDA delay due to facility issues; analysts adjust price targets amid uncertain ...

Gene therapy can improve hearing in children and adults with congenital deafness or severe hearing impairment, a new study involving researchers at Karolinska Institutet reports.

It is trying to treat a devastating lifelong disease by fixing it at its source. We are able to fix the actual genetic ...

The FDA cited manufacturing issues but did not flag problems with Ultragenyx’s data package for UX111, with the biotech ...

A team of researchers from the San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET, Milan), led by Nadia Coltella and ...

But her case is caused by a rare genetic mutation, so medications that work for 90% of people with cystic fibrosis won’t help her. The same dynamic plays out in other genetic conditions.