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SG001

Development stage: Preclinical
Lead developer: Shenji Changhua
Modality: Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
Administration: Intrathecal

Overview

SG001 is an adeno-associated virus (AAV)-based gene therapy candidate developed by Shenji Changhua (also known as SineuGene Therapeutics). It is designed to treat a range of neurological and neurodegenerative conditions, including amyotrophic lateral sclerosis (ALS), Alzheimer's disease (AD), Parkinson's disease (PD), and stroke. While the specific genetic target and mechanism of action remain undisclosed, the program is currently in the preclinical and proof-of-concept stages of development, aiming to leverage AAV vector technology for therapeutic gene delivery to the central nervous system.

Targets

PDCD1 (Programmed cell death protein 1 receptor)Tripartite motif-containing protein 72

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