Researchers are making strides in improving gene therapies for genetic diseases, particularly chronic kidney disease, using adeno-associated virus, or AAV, vectors. While AAV-based treatments have ...

The firm said it plans to share data with regulatory agencies from the Phase III STEER trial after it met its primary endpoint.

Their study showed that the adeno-associated virus 9 (AAV9)-packaged cardiac bridging integrator 1 (cBIN1) gene therapy increased the amount of blood the heart can pump and dramatically improved ...

Jaguar Gene Therapy has received clearance from the US Food and Drug Administration (FDA) to administer JAG201, a gene ...

ETX201 expresses a miRNA that targets the UBE3A-ATS to knock down its expression and is delivered using a clinically-validated adeno-associated virus serotype 9 (AAV9) vector. This potential one ...