Drug intelligence / Profile preview

CTx-TFEB

Development stage
Preclinical
Lead developer
Coave Therapeutics
Modality
Gene Therapies
01

Overview

**CTx-TFEB** is a gene therapy developed by Coave Therapeutics using its proprietary ALIGATER platform, which employs conjugated AAV (coAAV) vectors for targeted delivery of the **transcription factor EB (TFEB)** gene to neurons in the brain and spinal cord. TFEB acts as a master regulator of the autophagy-lysosomal pathway (ALP), promoting the clearance of toxic protein aggregates such as alpha-synuclein in alpha-synucleinopathies and misfolded proteins in ALS. This approach aims to halt neurodegeneration by enhancing lysosomal biogenesis and autophagy, with demonstrated preclinical efficacy in reducing aggregates, preserving dopaminergic neurons, and improving phenotypes in models of Multiple System Atrophy (MSA), Parkinson's disease (PD), and amyotrophic lateral sclerosis (ALS).[1][2][3][5][7]

02

Targets

TFEB (Transcription factor EB)

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