Neuronal ceroid lipofuscinosis gene therapy - University of Texas Southwestern Medical Center
Alternative Names: TSHA-121Latest Information Update: 10 May 2023
At a glance
- Originator University of Texas Southwestern Medical Center
- Developer Taysha Gene Therapies; University of Texas Southwestern Medical Center
- Class Gene therapies
- Mechanism of Action Gene transference
-
Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
Highest Development Phases
- Suspended Neuronal ceroid lipofuscinosis
Most Recent Events
- 10 May 2023 Suspended - Phase-I/II for Neuronal ceroid lipofuscinosis in USA (Intrathecal) due to limited financial and management resources
- 31 Dec 2022 Taysha Gene Therapies has patent pending CLN7-encoding transgene packaged in an AAV vector technology in-licensed from The Board of Regents of The University of Texas System in the US
- 28 Apr 2022 Phase-I/II clinical trials in Neuronal ceroid lipofuscinosis in USA (Intrathecal) before April 2022 (Taysha Gene Therapies, Form 10K, April 2022)