Posted by Michael Wonder on 28 Oct 2020
Taysha Gene Therapies receives rare paediatric disease designation and orphan drug designation for TSHA-104 to treat SURF1 associated Leigh syndrome
27 October 2020 - Taysha anticipated to submit Investigational new drug application for TSHA-104 to FDA in 2021.
Taysha Gene Therapies today announced that it has received rare paediatric disease designation and orphan drug designation from the U.S. FDA for TSHA-104, an AAV9-based gene therapy in development for SURF1-associated Leigh syndrome.
