Seelos Therapeutics Inc (NASDAQ:SEEL) said it has initiated a preclinical study of SLS-004 in Parkinson’s disease (PD) through an all-in-one lentiviral vector targeting the synuclein alpha (SNCA) gene.
Seelos said it is constructing a bimodular viral system harboring an endogenous alpha-synuclein (α-synuclein) transgene and inducible regulated repressive CRISPR/Cas9-unit to achieve constitutive activation and inducible suppression of PD-related pathologies.
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α-synuclein is a protein of interest to Parkinson's researchers because it is a major constituent of Lewy bodies and Lewy neurites, protein clumps that are the pathological hallmark of synucleinopathies, such as Parkinson's disease, dementia with Lewy bodies (DLB) and multiple system atrophy (MSA).
The company said that it had been previously shown that enrichment in DNA methylation at intron 1 of the α-synuclein gene SNCA, through SLS-004, facilitated "robust and precise repression of SNCA expression". The SNCA gene has been implicated as a highly significant risk factor for PD.
In a statement, Seelos chairman and chief executive Raj Mehra said: “There has been a high level of interest in the alpha-synuclein approach to Parkinson's and beginning further work on our first gene therapy program is exciting. Initiating this preclinical study earlier than expected is also very significant."
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