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

For gene therapy to work well, therapeutic molecules need to be efficiently delivered to the correct locations in the body -- a job commonly given to adeno-associated viruses (AAV). To improve the AAV ...

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 ...

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

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

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

The Hemophilia B treatment landscape is entering a new era, marked by disruptive innovation, curative potential, and renewed global attention. Once viewed as a lifelong burden requiring constant ...

The dynamics of the Krabbe disease market are expected to shift in the coming years due to the emergence of a new pipeline, increasing awareness of t ...

Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on ...