Taysha TSHA-102 data: safe in wild-type mice, extends survival >4x in Rett model

Taysha Gene Therapies, Inc.

New wild-type mouse data: TSHA-102 showed no toxicity or survival reduction over 36 weeks, no impact on neurobehavioral function.
In Mecp2–/Y knockout mice, TSHA-102 led to 47% survival at 36 weeks vs median 8.1 weeks in vehicle group (>4-fold extension, p<0.0001).
TSHA-102 significantly delayed onset of severe Rett phenotypes: severe clasping from ~7 to 21 weeks, abnormal gait from ~8 to 20 weeks.
Dosing of first adult patient in Phase 1/2 REVEAL trial expected Q2 2023; initial safety data anticipated Q2 2023.
TSHA-102 uses miRARE technology to regulate MeCP2 expression, addressing risks of both under- and overexpression in mosaic X-chromosome silencing.

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