Drug intelligence / Profile preview

TGTX-101

Development stage: Preclinical
Lead developer: Taysha Gene Therapies
Modality: Gene Therapies
Administration: Intrathecal

Overview

**TGTX-101** is a gene replacement therapy developed by Taysha Gene Therapies using an AAV vector to deliver a functional gene copy for the treatment of GM2-gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), a rare monogenic lysosomal storage disorder caused by HEXA or HEXB gene mutations leading to GM2 ganglioside accumulation in neurons. As of 2021, the program was planned to advance into clinical development with an IND filing targeted by year-end, positioning it as the lead candidate in Taysha's pipeline of over 15 AAV-based therapies for CNS diseases developed in partnership with UT Southwestern.

Targets

HEXB (Beta-hexosaminidase subunit beta)HEXA (Hexosaminidase subunit alpha)

Beyond the preview

Go deeper on TGTX-101.

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Clinical trials

Follow clinical development from study design and recruitment through results.

Trial phase
Status
Readouts

Indications & development

Explore development by indication, patient population, and geography.

Indications
Development status
Countries

Licensing & deals

Trace asset ownership, licensing agreements, and commercial partnerships.

Partners
Deal terms
Milestones

Patents & exclusivity

Explore the patent landscape and regulatory exclusivity around an asset.

Patents
Expiration dates
Exclusivity

Competitive landscape

Compare development programs by target, modality, and indication.

Competing assets
Targets
Development stage

Research & analysis

Connect source evidence and development news to your research questions.

Publications
News
Analysis

Bring the full picture into focus.

See how Gosset can support your research on TGTX-101.

Explore the full profile