Drugs / TSHA-105
last change Jul 2026 re-read 3 minutes ago

TSHA-105

Gene therapy (AAV / viral vector) targets SLC13A5 via restoration

Developed for
developmental and epileptic encephalopathy, 25
Investigated by
TESS Research Foundation

Trials 1

20272028202920302031
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1/2 NCT07102524 Jul 2026 → Dec 2030 expected developmental and epileptic encephalopathy, 25 TESS Research Foundation Recruiting No outcome recorded

News releases announcing trial results or a regulatory action · 6

DateIssuerRelease
2021-12-06 Taysha Gene Therapies, Inc. Results Taysha Gene Therapies Announces Late-Breaking Abstract and Poster Presentation on Positive Preclinical Data For TSHA-105 Demonstrating Therapeutic Potential for the Treatment of Epilepsy Caused by SLCA13A5 Deficiency tayshagtx.com ↗
TSHA-105 normalized electroencephalogram (EEG) and citrate levels and improved survival in both early treated juvenile and later treated adult preclinical mouse models of SLC13A5 deficiency
2021-08-25 Taysha Gene Therapies, Inc. Regulatory Taysha Gene Therapies Receives Orphan Drug Designation for TSHA-105 for the Treatment of Epilepsy Caused by SLC13A5 Deficiency From the European Commission tayshagtx.com ↗
today announced that it has been granted orphan drug designation from the European Commission for TSHA-105, an AAV9-based gene therapy in development for SLC13A5-related epilepsy.
2021-05-11 Taysha Gene Therapies, Inc. Results Taysha Gene Therapies Reports First Quarter 2021 Financial Results and Provides a Corporate Update tayshagtx.com ↗
TSHA-105 significantly reduced plasma citrate levels, normalized EEG brain activity, and reduced the number of seizures and seizure susceptibility in SLC13A5 knockout mice
2021-05-05 Taysha Gene Therapies, Inc. Results Taysha Gene Therapies Announces Multiple Data Presentations and Workshop Presentations at the 24th Annual Meeting of the American Society of Gene & Cell Therapy tayshagtx.com ↗
TSHA-105 significantly reduced plasma citrate levels, normalized EEG brain activity, and reduced the number of seizures and seizure susceptibility in SLC13A5 knockout mice
2021-04-14 Taysha Gene Therapies, Inc. Results Taysha Gene Therapies Announces New Data on Multiple Preclinical Programs and Upcoming R&D Day tayshagtx.com ↗
TSHA-105 significantly reduced plasma citrate levels, normalized EEG brain activity, and reduced the number of seizures and seizure susceptibility in SLC13A5 knockout mice
2021-01-19 Taysha Gene Therapies, Inc. Regulatory Taysha Gene Therapies Receives Rare Pediatric Disease and Orphan Drug Designations for TSHA-105 for the Treatment of Epilepsy Caused by SLC13A5 Deficiency tayshagtx.com ↗
today announced that it has received both rare pediatric disease and orphan drug designations from the U.S. Food and Drug Administration (FDA) for TSHA-105, an AAV9-based gene therapy in development for SLC13A5-related epilepsy.

Evidence & citations 5 cited values

Every value below carries the sentence it was read from. 1 source stands behind the page.

FieldValueCited text
Known as TSHA-105 ClinicalTrials.gov intervention name — accepted as the source's own label NCT07102524 ↗
Action Restore “expresses the fully functional form of SLC13A5” NCT07102524 ↗
Modality Gene therapy (AAV / viral vector) “AAV9-based gene therapy vector” NCT07102524 ↗
Route Intrathecal “delivered intrathecally” NCT07102524 ↗
Target SLC13A5 “expresses the fully functional form of SLC13A5” NCT07102524 ↗