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

Treatment with gene therapy candidate BB-301 led to improvements in swallowing ability for the first three OPMD patients in a ...

Marks’ resignation leaves the field without a regulator many view as “integral” to its progress over the last decade.

At such a critical moment in US history, we need reporters on the ground ... progress after he became the first infant to ...

Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing early investors to ...

The arc of biotechnology is one of breakthroughs and struggles, writes Tim Hunt, CEO of the Alliance for Regenerative ...

Sebastien Beauzile, 21, is the first New Yorker to have received the breakthrough Lyfgenia treatment, according to the New ...

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

In the time since the Novartis gene therapy Zolgensma was approved for babies with spinal muscular atrophy, other treatments ...