Gene therapy may offer new hope for infants with rare, fatal disorder

In 1962, a pediatric neurology resident at Columbia's Neurological Institute and his colleagues in the College of Physicians ...
Fierce Biotech

uniQure's ballyhooed gene therapy for Huntington's hits FDA roadblock

The FDA is said to be making a U-turn on uniQure’s one-time gene therapy candidate for Huntington’s disease despite the treatment being hailed as a game-changer. | FDA is said to be making a U-turn on ...
FierceBiotech

Neurogene rethinks gene therapy trial after high dose leaves Rett patient in critical condition

Neurogene has dropped plans to keep studying the high dose of its Rett syndrome gene therapy because of an adverse event that has left a patient in critical condition. The discontinuation has pushed ...
Morningstar

Biologic Competition, Payer Pressures, and Gene Therapy Momentum Define a Pivotal Moment in nAMD and DME Management, According to Spherix Global Insights

Exton, Pennsylvania, July 10, 2025 (GLOBE NEWSWIRE) -- Recently released research from Spherix Global Insights provides a timely look at the increasingly competitive and complex treatment landscape ...
Medical Xpress

Gene therapy may be key to permanently putting HIV into dormant state

In a study of human immune cells infected with HIV, the virus that causes AIDS, scientists at Johns Hopkins Medicine say a molecule within HIV itself can be manipulated and amplified to force the ...
Medscape

Mutation-Agnostic Gene Therapy May Restore Vision in RP

A new approach to gene therapy for retinitis pigmentosa may help patients with advanced disease regain vision regardless of which genetic mutation is causing the condition. An optogenetic therapy ...
FiercePharma

As Pfizer backs out of hemophilia gene therapy space, CSL hopes Hemgenix is here to stay

After Pfizer let limited patient interest get the best of its hemophilia B gene therapy ambitions and discontinued Beqvez, CSL Behring naturally faced the question of whether it can hold its ground ...
The Bakersfield Californian

Biologic Competition, Payer Pressures, and Gene Therapy Momentum Define a Pivotal Moment in ...

Exton, Pennsylvania, July 10, 2025 (GLOBE NEWSWIRE) -- Recently released research from Spherix Global Insights provides a timely look at the increasingly competitive and complex treatment landscape ...