The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by injection three-to-six times each week.

The California Institute for Regenerative Medicine has awarded a $6 million grant to USC investigators pioneering a new first-of-its-kind genetic therapy for glioblastoma, a severe form of brain ...

A £1.65 million treatment has been approved for use for some NHS patients, offering some with an inherited blood disorder ...

Campaigners reacted with joy as health officials approved a gene-editing therapy for certain children and adults with severe sickle cell disorder. It is thought that 1,700 people could be eligible ...

The announcement was made at Arab Health 2025 where Sidra Medicine will highlight its international patient service programmes ...

Announcing a new article publication for BIO Integration journal. Neurologic disorders currently affect approximately 100 million people worldwide.

Sangamo (SGMO) reported positive updated data from a Phase 1/2 study of its gene therapy candidate ST-920 in the treatment of Fabry disease. Read more here.

LONDON, Jan 31 (Reuters) - Britain's National Health Service (NHS) will provide a cutting-edge gene therapy that aims to cure sickle cell disease, the National Institute for Health and Care ...

In gene therapy, a person's faulty genes are either replaced or modified to treat or prevent disease. With roughly two dozen products on the market and hundreds of clinical trials in progress or ...