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Taysha Gene Therapies' Rett Syndrome Candidate Shows Encouraging Action In Animal Studies, Human Trial To Start By 2021

05/10/2021 13:17
  • 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.