Gene therapies have the potential to cure some diseases, but they are extraordinarily expensive. Location can also be a big hurdle for patients seeking this specialized care.

Gene therapies have the potential to cure some diseases, but they are extraordinarily expensive. Location can also be a big ...

CRISPR gene therapy Casgevy can nearly eliminate sickle cell crises, but difficult stem‑cell collection is delaying access ...

Researchers have used gene therapy to partially reprogram the brain cells that store memories in aging mice, reversing signs ...

"Gene editing takes centre stage in FDA’s new rare disease approval pathway" was originally created and published by ...

Officials laid out a proposal to spur development of customized treatments for patients with hard-to-treat diseases, ...

Through an AI-enabled platform, the partnership between two non-profit groups seeks to bring overlooked cell and gene ...

At an event on Monday, the agency officially unveiled long-awaited draft guidance meant to help speed the development of bespoke treatments for extremely rare diseases.

A version of this Priestley Medal address will be presented at the American Chemical Society Spring 2026 meeting ...

UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new ...

Forget Vertex Pharmaceuticals -- CRISPR Therapeutics is a better buy right now.

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...