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Taysha Gene Therapies Inc (TSHA) Q3 2024 Earnings Call Highlights: Clinical Progress and ... [Yahoo! Finance]

Taysha Gene Therapies, Inc. (TSHA) 
Company Research Source: Yahoo! Finance
Q : How is Taysha Gene Therapies considering endpoints for their trials given the heterogeneity of Rett syndrome? A : Sean Nolan, CEO, explained that they are considering the heterogeneity by potentially using patients as their own controls, supported by natural history data. They are focusing on milestones and functional gains, which can vary among patients but are consistent in showing improvement across the board. Q : Can you provide more context on the natural history analysis and its use as a comparator in trials? A : Sean Nolan stated that while the natural history data does not indicate a uniform progression like in SMA, it does show consistent patterns in loss or lack of gain of certain functions. This data will be used to inform trial design and endpoints, with further discussions planned with the FDA. Q : What is the safety profile of the high dose of TSHA 102, and is there a window for adverse events? A : Sukumar Nagendran, President & Head of R&D, noted that the high Show less Read more
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