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

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

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

Mutations in BRCA2—a gene known to repair damaged DNA and suppress tumor formation—can predict an individual's predisposition ...

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 ...

LUXTURNA has significant market potential due to the high unmet need for effective treatments in rare genetic conditions. With its FDA approv ...

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

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

In this interview, News Medical speaks with Olivier Negre, Chief Scientific Officer at Smart Immune, about how immunotherapy ...

Jonathon Whitton, AuD, PhD, VP, auditory global program head, Regeneron, discusses regulatory challenges for DB-OTO, Regeneron’s AAV-based gene therapy for hearing loss.

Opens in a new tab or window A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy delandistrogene moxeparvovec (Elevidys), Sarepta ...