SG004

SG004 is an adeno-associated virus (AAV)-based gene therapy candidate developed by Shenji Changhua (Beijing) Biotechnology for the treatment of Amyotrophic Lateral Sclerosis (ALS). The program specifically targets the *C9ORF72* gene, which is the most common genetic cause of ALS and frontotemporal dementia (FTD). Hexanucleotide repeat expansions in the *C9ORF72* gene lead to neurodegeneration through toxic gain-of-function mechanisms, including the formation of RNA foci and dipeptide repeat proteins, as well as a potential loss-of-function of the normal C9ORF72 protein. SG004 is designed to address these pathological processes using an AAV vector delivery system. As of early 2024, the program is in the preclinical and drug discovery stages of development.