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

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

A new nasal spray-based gene therapy, using AAV.CPP.16 shows promise for treating lung diseases like COVID-19 and pulmonary ...

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

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

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 ...

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

Ask scientists which gene-editing tool is most needed to advance gene therapy, and they'd probably describe a system that's now close to realization in the labs of Samuel Sternberg at Columbia ...

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

Developed by Sarepta Therapeutics, Elevidys is a one-time, intravenous treatment that uses adeno-associated virus (AAV) ...

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