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

In the last year, some drugmakers have pulled back from the sector, including Pfizer, which recently stopped selling its gene ...

With a storied history in vaccine development, India's Bharat Biotech is shaking things up and answering the call of advanced ...

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

The Company is currently analyzing the case and will update the prescribing information for Elevidys to note this development.

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

A baby boy with a rare illness is making “incredible” progress after he became the first ever patient to receive a new gene ...

Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases 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 ...

Gene therapy, which promises a possible cure for rare diseases like sickle cell, is losing early investors to higher-reward ...

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