Seelos Therapeutics acquires license for gene therapy program
Seelos Therapeutics announced the exclusive worldwide licensing of a gene therapy program targeting the regulation of the SNCA gene, which encodes alpha-synuclein expression, from Duke University. Seelos plans to study this approach, named SLS-004, initially in Parkinson's Disease. At present, there is no effective treatment to prevent PD or halt its progression. The SNCA gene has been implicated as a highly significant risk factor for PD. In addition, accumulating evidence suggests that elevated levels of wild-type alpha-synuclein are causative in the pathogenesis of PD. The role of SNCA overexpression in PD pathogenesis and the need to maintain normal physiological levels of alpha-synuclein protein emphasize the so-far unmet need to develop new therapeutic strategies, such as SLS-004, targeting the regulatory mechanisms of SNCA expression. SLS-004 is an all-in-one lentiviral vector, for targeted DNA-methylation editing within intron. The system is based on CRISPR-dCas9 fused with the catalytic domain of DNA methyltransferase 3A (DNMT3A), an enzyme that is responsible for DNA methylation. The system was delivered to dopaminergic neurons derived from human induced pluripotent stem cells from a PD patient. As a result, the expression of SNCA was modified and disease-related cellular-phenotypes characteristics of the neurons were reversed.