For gene therapy to work well, therapeutic molecules need to be efficiently delivered to the correct locations in the body—a ...

While the medical world is melting down from the absolute apocalypse that is RFK Jr., it's good to celebrate that (at least ...

The FDA ordered a clinical hold on Rocket Pharmaceuticals' Danon disease trial after the adverse event was first disclosed.

Hot on the heels of a $150 million fundraise, Atsena Therapeutics is back with early-stage data for the company’s eye disease ...

Researchers from The University of Osaka find that mass photometry can distinguish between full and empty rAAV particles, ...

A personalized gene editing therapy was successfully used to treat an infant with CPS1 deficiency, a disease that causes ...

Qure divested for $25M, advanced AMT-130 with FDA approvals, and building infrastructure. A strong balance sheet backs ...

Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing ...

Find out if gene therapy has also find its way into skin diseases. With two approvals in the last 2 years, is it the start of ...

Scientists say they've put together a molecular toolkit that could be used to treat a variety of brain diseases, possibly ...

US researchers have engineered a novel gene therapy to target the airway and lungs via a nasal spray. For gene therapy to ...