GLOBAL RESEARCH ARCHIVE
Previewing Some Thoughts Into TSHA's Upcoming Catalysts; Reiterating as a Top Pick
Research evidence excerpt
Previewing Some Thoughts Into TSHA's Upcoming Catalysts; Reiterating as a Top Pick
June 9, 2026
How Do We Cross-Compare NGNE (NC) and TSHA's Constructs/Therapies?
●Most investors we speak to have a clear view that intrathecal
administration will be viewed substantially more favorably than ICV
in a commercial world, by ease in performing/done in outpatient
setting, safety, more favorable reimbursement, etc.
●And importantly intrathecal administration doesn't come with an
efficacy trade off. Investors originally on the fence on this have started
to de-emphasize this debate now that TSHA has shown clear benefits
in the clinic.
●A more recent topic asked has been around construct differences. And
it's been our view that TSHA's double-stranded construct with a CNS-
specific promoter may allow for a rapid onset of action (more rapid
transcription of the transgene).
How to Cross-Compare Data Across NGNE (NC) and TSHA?
●Big picture, both company datasets have validated that AAV9-based
gene therapy is a successful treatment option for Rett syndrome.
●But cross-comparing data can be difficult for many reasons, and we
think investors should be understanding of several key differences.
●First on patient ages. We believe the strongest data to interpret is in
patients over the age of six where third party data suggests is the
earliest one can look at to remove any bias that some milestones/
improvements occur by chance (link to Cantor note).
●Next on the rigor of data collection where Taysha's includes clear
definitions of milestones, rigorous baseline collection, outside raters
adjudicating milestones, etc.
●We would note that Taysha also discloses additional skills and
improvements that are separate from the "milestones" but still carry
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