Seelos Therapeutics Inc. (NASDAQ:SEEL) has announced the commencement of a preclinical SLS-004 study in Parkinson’s disease through a lentiviral vector that is all in one targeting the synuclein alpha gene.

Seelos begins preclinical trial early than anticipated

The company is developing a bi-modular viral system that harbors an endogenous synuclein alpha transgene as well as an inducible controlled repressive CRISPR/Cas9-unit.  It aims at achieving constitutive activation and also inducible suppression for PD-associated pathologies.

According to Seelos CEO and Chairman Raj Mehra, Ph. D, the commencement of the preclinical study early than anticipated is a major and very significant step. Mehra added that there has been an increase in the synuclein-alpha approach in the treatment of Parkinson’s disease. As a result, the prospect of Seloos initiating a preclinical study, which is its first gene therapy program, is a massive step. 

Previous studies have demonstrated that DNA methylation enrichment at the first intron of the alpha-synuclein gene SNCA via SLS-004 enhanced precise and robust SNCA expression repression, consistent with freeing PD-phenotypes. The SNCA gene encodes alpha-synuclein expression, and it is a highly significant PD risk factor. 

Alpha-synuclein protein plays a role in the pathogenesis of Parkinson’s disease

There is growing evidence that indicates the elevated levels of alpha-synuclein are the causative factors in PD pathogenesis. The need to have normal physiological alpha-synuclein levels and the role of overexpression of SNCA in the pathogenesis of PD is indicated the level of unmet need in developing novel therapeutic strategies like SLS-004 that can target the regulatory mechanisms of expression of SNCA. 

One of the areas with a lot of interest in Parkinson’s researchers is the alpha-synuclein protein, which is a major component of Lewy neuritis and Lewy bodies. These are protein clumps that play a pathological role in synucleinopathies like Parkinson’s disease, multiple system atrophy and dementia with Lewy bodies. For years since its discovery, most Parkinson’s disease researchers have been focusing on the alpha-synuclein protein trying to characterize its role in the pathology of PD and its role for targeting for potential neuroprotective therapies.