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TSHA-116

Development stage: Discontinued
Lead developer: Taysha Gene Therapies
Modality: Gene Therapies
Administration: Intrathecal

Overview

TSHA-116 is an investigational gene therapy developed originally by Taysha Gene Therapies for the treatment of Prader-Willi syndrome, a complex genetic disorder caused by the loss of function of paternal genes on chromosome 15. Evidence indicates it was designed as a potential disease-modifying therapy but its development has been discontinued. Mechanistic and molecular details of the agent are not publicly disclosed, but it was intended as an advanced epigenetic or gene-modifying intervention targeting the underlying genetic defects in Prader-Willi syndrome[5][9].

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