Seelos Therapeutics Inc, a clinical-stage biopharmaceutical company focused on the development of therapies for central nervous system disorders and rare diseases, announces it was selected to receive a grant from The Michael J. Fox Foundation for Parkinson’s Research to advance preclinical research and development of its gene therapy delivered SLS-004 program.
“The selection of SLS-004 to receive grant funding from The Michael J. Fox Foundation is a strong validation of our program and should significantly raise the profile of our program,” said Raj Mehra, Ph.D., Chairman and CEO of Seelos. “We look forward to sharing additional data from our ongoing preclinical studies later this year.”
Parkinson’s disease (PD) is the second most common neurodegenerative disorder in the world and currently, there is no effective treatment to prevent PD or to halt its progression. The SNCA gene has been implicated as a highly significant genetic risk factor for PD. In addition, accumulating evidence has suggested that elevated levels of alpha-synuclein (α-synuclein) are causative in the pathogenesis of PD. Patients with impaired regulation of the SNCA gene show as high as 200% expression of α-synuclein protein. A reduction of 25%-50% in SNCA mRNA and protein expression should be sufficient to restore normal physiological levels of SNCA.
In July 2021, Seelos announced positive in vivo data demonstrating down-regulation of SNCA mRNA and protein expression from a study of SLS-004 in an in-vivo rodent model utilizing CRISPR-dCas9 gene therapy technology. A single dose of SLS-004 produced a therapeutically desirable 27% reduction in SNCA mRNA and a 40% reduction in SNCA protein expression.
Additionally in June 2022, Seelos released data demonstrating a statistically significant (p<0.01) 19% downregulation of mRNA and a ~40% reduction of α-synuclein in an in vitro study of SLS-004 in dementia with Lewy bodies (DLB).
[Source(s): Seelos Therapeutics Inc, PR Newswire]