SLS-004

SLS-004 is an investigational gene therapy designed to regulate the expression of the SNCA gene, which encodes alpha-synuclein, a protein implicated in the pathogenesis of Parkinson’s disease and other synucleinopathies such as dementia with Lewy bodies. The therapy uses a modified lentiviral vector to deliver DNA methyltransferase 3A (DNMT3A) guided by CRISPR-dCas9 technology to specifically methylate a region of the SNCA gene, thereby reducing its expression and subsequent alpha-synuclein production. Preclinical studies have shown that SLS-004 can reduce alpha-synuclein levels by approximately 30–40% in neuronal models derived from patients and animal models, leading to improved neuronal survival and reduced pathological changes associated with Parkinson’s disease. The drug is being developed by Seelos Therapeutics in collaboration with Duke University[1][4][5][6][7][9].