Posted by Michael Wonder on 28 Aug 2020
Taysha Gene Therapies receives orphan drug designation and rare paediatric disease designation for TSHA-101 for GM2 gangliosidosis
27 August 2020 - TSHA-101 anticipated to advance into the clinic by the end of 2020.
Taysha Gene Therapies today announced that it has received orphan drug designation and rare paediatric disease designation from the U.S. FDA for TSHA-101, an AAV9-based gene therapy in development for GM2 gangliosidosis.
The company anticipates TSHA-101 to enter the clinic by the end of 2020.
