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Taysha Gene Therapies Inc (NASDAQ: TSHA) has announced the online publication of new preclinical data for TSHA-102 in Rett syndrome. The data will be included in the May edition of Brain, a peer-reviewed neurological science journal.
Rett syndrome is a rare, severe neurological disorder that affects mostly girls. Symptoms include impairments in language and coordination and repetitive movements. Those affected often have slower growth, difficulty walking, and smaller head size.
The preclinical study evaluated the safety and efficacy of regulated miniMECP2 gene transfer, TSHA-102, compared to unregulated full-length MECP2 (AAV9/MECP2) and unregulated miniMECP2 (AAV9/miniMECP2).
TSHA-102 extended knockout survival by 56% via intrathecal delivery. In contrast, the unregulated miniMECP2 gene transfer failed to extend knockout survival at either dose tested significantly.
Additionally, the unregulated full-length MECP2 construct did not demonstrate a significant extension in survival and was associated with an unacceptable toxicity profile in wild-type mice.
In addition to survival, behavioral side effects were explored. AAV9/MECP2- and AAV9/miniMECP2-treated wild-type mice had a significantly higher mean (worse) aggregate behavioral severity score than observed for saline-treated mice.
TSHA-102-treated wild-type mice had a significantly lower (better) mean aggregate severity score versus those of AAV9/MECP2- and AAV9/miniMECP2-treated mice at most time points.
No significant difference was observed between saline- and TSHA-102-treated wild-type mice.
The company plans to file an Investigational New Drug/Clinical Trial Application for TSHA-102 in the second half of this year, followed by a Phase 1/2 trial by year-end 2021.
Price Action: TSHA shares are down 2.5% at $20.67 during the market trading hours on the last check Monday.
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