Taysha Gene Therapies’ (NASDAQ:TSHA) TSHA-103 received FDA rare pediatric and orphan drug designations for the treatment of SLC6A1-related epilepsy.
SLC6A1-related epilepsy is caused by the loss of function of one copy of the SLC6A1 gene. TSHA-103 is an adeno-associated virus serotype 9-base gene therapy.
“Haploinsufficiency in the SLC6A1 gene has been identified as a cause of genetic epilepsy, yet there remains a lack of approved disease-modifying therapies,” Stephen Gray, Ph.D., Taysha’s CSO, said in a statement.
“The designations highlight the innovation of TSHA-103 and the importance of developing a treatment for patients living with this devastating disease,” he added.
The company’s TSHA-101, TSHA-102, TSHA-118 and TSHA-104 gene therapy candidates previously received rare pediatric and orphan drug designations for the treatment of GM2 gangliosidosis, Rett syndrome, infantile Batten disease and Leigh syndrome, respectively.