Seelos Therapeutics Says In Vivo Data Positive In Study Of Parkinson's Disease

(RTTNews) - Seelos Therapeutics, Inc. (SEEL) on Wednesday announced positive in vivo data from the study of SLS-004 in Parkinson's disease pathology.

An in-vivo rodent model study of SLS-004 using CRISPR-dCas9 gene therapy technology showed that a single dose of SLS-004 produced therapeutically desirable 27% reduction in SNCA mRNA and 40% reduction in SNCA protein expression.

SLS-004 is an epigenome-editing approach to modulate expression of SNCA gene mediated by modification of DNA-methylation.

The SNCA gene has been implicated as a highly significant genetic risk factor for Parkinson's disease. Seelos said, a 25%-50% reduction in SNCA mRNA and protein expression should be sufficient to restore normal physiological levels of SNCA.

"The effect of SNCA downregulation achieved with our innovative CRISPR-dCas9 platform technology in this in vivo animal study is very promising and we plan to further validate the safety and efficacy of our lentivirus-based epigenome-editing approach in a full-range preclinical study under our Sponsored Research Agreement with Seelos, with the aim of reversing the Parkinson's disease-related pathology," said Boris Kantor, Associate Research Professor, Duke University School of Medicine.