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SG003

Development stage: Preclinical
Lead developer: SineuGene Therapeutics
Modality: Gene Therapies
Administration: Intrathecal

Overview

SG003 is an adeno-associated virus (AAV)-mediated gene therapy developed by Shenji Changhua (Beijing) Biotechnology Co., Ltd. for the treatment of amyotrophic lateral sclerosis (ALS) specifically associated with mutations in the superoxide dismutase 1 (SOD1) gene. The therapy is designed to target and modulate the expression of the pathogenic SOD1 protein, which is known to aggregate and cause motor neuron death in this subset of ALS patients. By utilizing an AAV vector to deliver genetic material, SG003 aims to reduce the toxic gain-of-function associated with mutant SOD1. The program is currently in the IND-enabling and Investigator-Initiated Trial (IIT) stages of development.

Other names: Shenji Changhua SOD1-AAV

Targets

Misfolded SOD1

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