Taysha Gene Therapies is a clinical-stage company developing TSHA-102, a one-time AAV9 gene therapy given into the spinal fluid for Rett syndrome; it is not approved.
Taysha's case rests on TSHA-102, a one-time gene therapy given into the spinal fluid for Rett syndrome. The pivotal REVEAL trial dosed 17 patients and is single-arm, with each patient as her own control, and topline data are expected in the first half of 2027. Across 33 patients there were no severe treatment-related serious adverse events, and cash of $455.4 million is expected to last into the second half of 2028.
Thesis checklist
0 risks need attention
Why it is in this state
The evidence, what would weaken it and the source filing
Why it is in this state
The evidence, what would weaken it and the source filing
Why it is in this state
The evidence, what would weaken it and the source filing
Next decision point
Tests: The REVEAL interim analysis supports a BLA
Do enough patients gain milestones, and does FDA accept the data for a BLA?
Strong, inconclusive and weak outcomes
What to look for in the result and how each would move the checklist
Price and material events
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Dosing is complete in Taysha's pivotal Rett syndrome trial; 6-month interim data and FDA feedback on the BLA path come in 1H 2027
TSHA-102 in Rett syndrome heads toward BLA on a 100% response rate; the 6-month interim analysis will determine whether that signal is registrational
FDA Breakthrough Therapy Designation granted for TSHA-102
About this history: every entry shows when the Thesis moved and what it said. The reasoning behind each move (the evidence, the analysis and the source filing) and the earnings write-ups are part of a subscription.
Dates, trials and completed outcomes are open to everyone. What each upcoming milestone hinges on is part of a subscription.
Disease areas: Neurology and pain
Catalyst calendar · Upcoming FDA decisions · Thesis changes
Competitive landscape
7 peers tracked
Cash: Market-data provider estimate; measurement period unavailable.
Debt: Market-data provider estimate; a complete comparable filing balance is unavailable.
Quarterly burn: Estimated from trailing free cash flow; measurement period unavailable.
Net cash and estimated runway require verified matching measurement periods. Missing debt components are not assumed to be zero.
Runway: No underlying guidance available.