Taysha nabs rare pediatric disease, orphan drug status

By The Science Advisory Board staff writers

October 27, 2020 -- Taysha Gene Therapies has received rare pediatric disease designation and orphan drug designation from the U.S. Food and Drug Administration (FDA) for TSHA-104, an adeno-associated virus 9 (AAV9)-based gene therapy candidate for surfeit locus protein 1 (SURF1)-associated Leigh syndrome.

Taysha plans to submit an investigational new drug application to the FDA for TSHA-104 in 2021.

Leigh syndrome is a severe neurological disorder that presents in the first year of life. Approximately 10%-15% of people with Leigh syndrome have a SURF1 mutation. The progressive disease leads to loss of mental and movement abilities and can result in death within two to three years.

Taysha has also secured pediatric disease designation for its GM2 gangliosidosis program, TSHA-102 for Rett syndrome and TSHA-118 for CLN1 disease.

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