Fred Hutch bioengineer Dr. Matthias Stephan is working to develop a special foam that could help make gene therapy for blood ...

Cincinnati Children's today celebrated the opening of a new state-of-art facility for its Applied Gene and Cell Therapy Center, which will enable the health system to increase clinical trials of ...

By adding a microRNA molecule to ALS models, Tel Aviv University, Ben-Gurion University and Weizmann Institute researchers ...

AAV-SLB101 is a rationally designed capsid developed for enhanced skeletal muscle, cardiac tropism, and reduced biodistribution to the liver.

Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to ...

Andelyn Biosciences has entered a non-exclusive worldwide license and collaboration agreement with Solid Biosciences Inc. for ...

Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment ...

Gene anticipates filing a biologics licence application (BLA) for detalimogene voraplasmid to the FDA in H2 2026.

Despite the best efforts of modern medicine, Huntington’s disease is a condition that still comes with a tragic prognosis.

Non-exclusive license providing Solid’s proprietary, next-generation capsid, AAV-SLB101, to Andelyn Biosciences, a full-service cell and gene ...

In a significant leap for gene therapy, scientists have discovered a method to reprogram the pancreas to produce GLP-1 ...