TSHA-105 / Taysha Gene Therapies - LARVOL DELTA

Intrathecal Gene Therapy For SLC13A5 Citrate Transporter Disorder (clinicaltrials.gov) - P1/2 | N=8 | Recruiting | Sponsor: TESS Research Foundation | Not yet recruiting ➔ Recruiting

Enrollment open • Gene Therapies

Intrathecal Gene Therapy For SLC13A5 Citrate Transporter Disorder (clinicaltrials.gov) - P1/2 | N=2 | Not yet recruiting | Sponsor: TESS Research Foundation | Initiation date: Dec 2025 ➔ Aug 2026

Trial initiation date • Gene Therapies

Broad CNS Biodistribution of AAV9-based Gene Therapies Delivered by Intrathecal Lumbar Puncture in Non-Human Primates (ESGCT 2024) - "During preclinical characterization of investigational gene therapies, biodistribution in NHPs of TSHA-101, TSHA-102, TSHA-105 and TSHA-120 was evaluated after administration by the IT (all products) or ICM (TSHA-102 only) routes. Overall, biodistribution analysis in Taysha’s NHP studies showed that both the IT and ICM routes led to comparable widespread AAV9 distribution throughout the CNS, achieving brain levels of ∼3 – 5 × 105 vg/µg at comparable doses and timing. These findings support the use of lumbar IT administration as an effective, procedurally simple approach for rAAV9 dosing of the CNS."

Gene therapy • Gene Therapies

Intrathecal Gene Therapy For SLC13A5 Citrate Transporter Disorder (clinicaltrials.gov) - P1/2 | N=2 | Not yet recruiting | Sponsor: Elpida Therapeutics SPC

New P1/2 trial • Gene Therapies

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 (Businesswire) - "Taysha Gene Therapies, Inc...announced a late-breaking abstract and poster presentation by Dr. Rachel Bailey, Assistant Professor at UT Southwestern Medical Center on positive preclinical data for TSHA-105, an AAV9-based gene therapy in development for SLC13A5-related epilepsy at the American Epilepsy Society Annual Meeting on December 6th 2021...An IND/CTA filing for TSHA-105 for the treatment of SLC13A5-related epilepsy is expected in 2022."

IND • Preclinical • CNS Disorders • Epilepsy

Taysha Gene Therapies Receives Orphan Drug Designation for TSHA-105 for the Treatment of Epilepsy Caused by SLC13A5 Deficiency From the European Commission (Businesswire) - "Taysha Gene Therapies, Inc...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."

Orphan drug • CNS Disorders • Epilepsy

Taysha Gene Therapies to Host Virtual R&D Day (Businesswire) - "Taysha Gene Therapies, Inc...announced that it will host its first two-day virtual research and development (R&D) day for analysts and investors. The event will be webcast live on June 28 and June 29, 2021 from 10:00 a.m. to 1:00 p.m. ET each day...TSHA-105 (SLC13A5 deficiency): AAV9-based gene therapy expressing human SLC13A5 protein to potentially treat SLC13A5 deficiency, a rare autosomal recessive epileptic encephalopathy characterized by the onset of seizures within the first few days of life. This gene replacement therapy program is currently in IND/CTA-enabling studies."

Clinical • CNS Disorders • Epilepsy

Taysha Gene Therapies Announces Multiple Data Presentations and Workshop Presentations at the 24th Annual Meeting of the American Society of Gene & Cell Therapy (Businesswire) - "Taysha Gene Therapies, Inc...announced that preclinical data from its investigational gene therapy programs will be presented at the 24th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), which will be held virtually May 11-14, 2021...TSHA-105-treated knockout mice demonstrated significantly decreased plasma citrate levels compared to knockout mice treated with vehicle. TSHA-105-treated knockout mice had reduced spike train activity and seizure frequency on electroencephalogram (EEG)."

Preclinical • CNS Disorders • Epilepsy

Taysha Gene Therapies Announces New Data on Multiple Preclinical Programs and Upcoming R&D Day (Businesswire) - “Taysha Gene Therapies, Inc….today announced new data for multiple preclinical programs and a planned R&D Day, which will be held in June 2021.…We have advanced five programs into IND/CTA-enabling studies, including TSHA-105 in SLC13A5 deficiency…In SLC13A5 knockout mice, treatment with...TSHA-105 normalized electroencephalogram (EEG) brain activity, reduced the number of seizures, and reduced seizure susceptibility compared to vehicle-treated controls…In preclinical studies, TSHA-110 caused a significant, dose-dependent reduction of GM2 accumulation at 20 weeks in mice that were dosed intrathecally at postnatal day 1 or at 6 weeks of age. Long-term follow up studies, which include bi-monthly behavioral, as well as biochemical and histological analyses, are currently ongoing.”

Preclinical • CNS Disorders • Epilepsy