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 ...

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

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

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

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

In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...

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

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 ...

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

A new study in the peer-reviewed journal Human Gene Therapy indicates that DNA impurities derived from plasmid and host cell ...