Four patients with infantile-onset Pompe’s disease received a single intravenous injection of an adeno-associated virus serotype 9 vector carrying codon-optimized complementary DNA encoding human acid ...

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 ...

Researchers in the lab of Hans-Peter Kiem, MD, PhD, at Fred Hutch Cancer Center have devised a method that could one day treat genetic hematologic disorders by correcting how the body makes blood ...

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 ...

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 ...

The antibiotic, effective against resistant respiratory infections, represents a historic leap in India’s pursuit of ...

A patient has died in a clinical trial evaluating a Rocket Pharmaceuticals gene therapy shortly after complications emerged that led the FDA to pause the study. An inquiry into the fatality is ...

Cirrus’ lead program is a novel AAV gene therapy designed to restore IRAK-M, a central regulator of the eye’s immune homeostasis, so as to prevent AMD progression and preserve central vision IRAK-M ...