An experimental gene therapy treatment created at UCLA gave a "bubble girl" born with a rare genetic disorder a new life.

The donation from Don and Anne Edwards aims to speed research, treatment and diagnosis for children with rare and genetic ...

From rare diseases to chronic conditions, epigenetic editing unlocks new ways to control gene activity without altering the genetic code.

The FDA on Tuesday approved etuvetidigene autotemcel (Waskyra) as the first cell-based gene therapy for patients 6 months and ...

The rate of severe infections decreased from 2 infections to 0.2 infections per PYO after treatment with etuvetidigene autotemcel.

On November 25, 2025, Sarepta Therapeutics filed two petitions for inter partes review (“IPR”) challenging Genzyme’s patents relating to ...

The firm plans to seek accelerated approval from the FDA next year, while it launches a Phase III trial to support other regulatory applications.

Wedbush upgrades Sarepta Therapeutics, citing overdone selloff and potential upside as FDA could re-approval Elevidys for non ...

Bristol Myers Squibb said it will enroll more patients in a key Phase 3 trial studying its drug Cobenfy in psychosis associated with Alzheimer’s disease, after the company found “irregularities due to ...

For the scientific enterprise, 2025 was marked by setbacks and challenges. But scientists are no strangers to adversity—the ability to overcome obstacles is built into our training. In turn, research ...

Capricor Therapeutics’ stock has soared by more than 370% after a pivotal trial of its Duchenne muscular dystrophy (DMD) cell ...

A regimen of three immune-suppressing drugs may improve the effectiveness of gene therapy for people with DMD, research in ...