TSHA - Taysha Gene Therapies: Latest Rett Data Checks Momentum Road Ahead Rocky

2024-06-19 22:40:52 ET

Summary

• Taysha Gene Therapies stock fell over 25% after disappointing data from Phase 1/2 REVEAL study of TSHA-102 in Rett Syndrome patients.
• TSHA-102 is a gene therapy targeting Rett Syndrome, a rare neurodevelopmental disorder with no approved disease-modifying therapies.
• Despite some positive signs in initial data, the lack of a placebo arm and the need for more data to support approval are evident, given the recent approval of Dayvue.
• Taysha has limited funding and no other assets to fall back on should TSHA-102 not succeed - the risk of further losses is therefore significant.

Investment Overview

Taysha Gene Therapies ( TSHA ) stock hit the skids yesterday as the Dallas, Texas headquartered biotech reported data from its Phase 1/2 REVEAL study of its lead and only gene therapy TSHA-102 in patients with Rett Syndrome - an indication the company discusses as follows in its 2023 annual report / 10K submission :

a rare progressive neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene encoding methyl CpG-binding protein 2 (MeCP2), which is essential for regulating neuronal and synaptic function in the brain.

The disorder is characterized by loss of communication and hand function, slowing and/or regression of development, motor and respiratory impairment, seizures, intellectual disabilities and shortened life expectancy. Rett syndrome progression is divided into four key stages, beginning with early onset stagnation at 6 to 18 months of age followed by rapid regression, plateau and late motor deterioration.

Rett syndrome primarily occurs in females and is one of the most common genetic causes of severe intellectual disability.

For further details see: