Scientists engineered a way to deliver genes directly to lung and airway cells using a nasal spray * AAV.CCP.16 -// a viral ...

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

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

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

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

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

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

Wilson's new company, Philadelphia-based Gemma Biotherapeutics, plans to start testing gene therapies in Brazil this year.

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

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

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 ...

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