Gene therapy pioneer Jim Wilson and researcher Kiran Musunuru are using contrasting approaches to overcome “irrational ...

Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...

Scientists at the Allen Institute have made a major breakthrough in research for people living with Dravet Syndrome, which is ...

A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...

A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

Shares of Sarepta Therapeutics have struggled for the past year due to concerns about the commercial potential of its gene ...

The death of a 16-year-old boy evokes a lengthy battle to realize the benefit of cutting-edge gene therapy while also ...

By replacing the defective gene associated with Dravet syndrome in mice, scientists successfully alleviated symptoms without ...

By Rishika Sadam HYDERABAD (Reuters) -Indian vaccine maker Bharat Biotech said on Thursday it had invested $75 million in its first cell and gene therapy (CGT) facility in the southern Indian state of ...

A pair of privately-held developers of editing-based therapies have some successes to show for their recent efforts to ...

The research, taking place at the Allen Institute for Brain Science, offers new hope to families fighting against Dravet Syndrome, a severe form of epilepsy that devastates childhood development.

To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as ...