Posted by Michael Wonder on 15 Oct 2020
Taysha Gene Therapies receives rare paediatric disease designation and orphan drug designation for TSHA-102 as a treatment for Rett syndrome
14 October 2020 - Program leverages novel miRARE platform technology used to control transgene expression on a cellular basis.
Taysha Gene Therapies today announced that it has received rare paediatric disease designation and orphan drug designation from the U.S. FDA for TSHA-102, an AAV9-based gene therapy in development for the treatment of Rett syndrome.
Taysha anticipates that it will submit an Investigational new drug application for TSHA-102 to the FDA in 2021.
