Seelos Therapeutics Inc. (NASDAQ: SEEL) Release Encouraging SLS-004 Study Results Showing SNCA mRNA Downregulation

Seelos Therapeutics Inc. (NASDAQ: SEEL) has announced encouraging in vivo data showing SNCA mRNA and protein expression down-regulation a study evaluating SLS-004 in in-vivo animal models using CRISPR-dCas9 gene therapy tech.Data showed that a single SLS-004 dose resulted in therapeutically desirable SNCA mRNA reduction by 27% and around 40% SNCA protein expression reduction.

SNCA gene plays a role in PD pathogenesis

Parkinson’s disease (PD) is the world’s second most common neurological disorder, and there is presently no effective medication to treat or slow the progression of the disease. The SNCA gene is a major hereditary risk factor for PD. Furthermore, mounting evidence suggests that high levels of alpha-synuclein (-synuclein) have a role in the etiology of Parkinson’s disease. Patients with SNCA gene regulatory problems have up to 200 percent -synuclein protein expression. To restore normal physiological SNCA levels, a 25% to 50% reduction in SNCA mRNA and protein expression is necessary.

The study attained SNCA mRNA and SNCA protein expression downregulation.

Seelos Chairman and CEO Raj Mehra said, “We are highly encouraged by these preliminary findings demonstrating downregulation of SNCA mRNA and SNCA protein expression in this in vivo model. Overexpression of alpha-synuclein has been implicated as a highly significant risk factor for Parkinson’s and the accumulation of this protein is a pathological hallmark of synucleinopathies for additional diseases such as dementia with Lewy bodies and multiple system atrophy.”

Duke University School of Medicine Associate Professor Boris Kantor said that the SNCA downregulation effects attained with the innovative CRISPR-dCas9 platform in the in vivo rodents study are encouraging. Boris said they are planning further validation of the efficacy and safety of the lentivirus-based epigenome-editing model in a preclinical trial under the Sponsored Research Agreement with Seelos. The study aims to reverse PD-related pathology.

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