Preclinical Study Backs Seelos' Gene Therapy for Dementia with Lewy Bodies
09 Jun 2022
Gene TherapymRNA
Seelos Therapeutics announced in vitro data on Thursday supporting its gene therapy, SLS-004, which is intended for the treatment of dementia with Lewy bodies (DLB).
Dementia with Lewy bodies, also known as Lewy body dementia, is a type of progressive dementia associated with declines in cognition, motor ability and independent functioning. It's also associated with cholinergic dysfunction, which can affect many aspects of brain functioning, including sleep and sensory processing.
DLB is the third most common type of dementia after Alzheimer’s disease (AD) and vascular dementia. The condition is caused by the buildup of Lewy bodies in the brain, which are mainly made up of alpha-synuclein proteins.
Seelos is hoping to make a difference for people with DLB using its gene therapy program, SLS-004, which utilizes CRISPR-dCas9. The company tested SLS-004 in cholinergic neurons from the areas of the brain cortex that are usually affected by DLB.
The preclinical study results showed that a single dose of SLS-004 was associated with a 19% downregulation of mRNA and a 40% reduction of alpha-synuclein, following a two-week period showing the drug’s potential to reduce the presence of Lewy bodies.
"This current in vitro study extends the existing CRISPR program for Parkinson's disease to DLB as both disorders are synucleinopathies although affecting different neurons in separate regions of the brain. Our team's observation of a meaningful efficacy with a new CRISPR technology focused on cholinergic neurons in striatum for DLB is exciting indeed, as it reinforces our earlier findings in a Parkinson's disease model," Raj Mehra, Ph.D., Seelos chairman and CEO, said in a press release. "Results producing statistically significant reductions in mRNA and alpha-synuclein are clinically meaningful. We plan to advance into additional preclinical studies in DLB and Parkinson's and expect additional data in the second half of this year."
SLS-004 modulates the expression of the SNCA gene, which has been implicated in the pathology of Lewy bodies in Parkinson’s disease and AD, as it encodes for alpha-synuclein. In previous studies of SLS-004, the therapeutic was able to produce a 30% downregulation of SNCA overexpression in dopaminergic neurons in a PD patient. The reduction was sufficient to ameliorate disease-related cellular phenotypes. Seelos plans to announce additional data from its Parkinson’s disease model in the second half of 2022.
Armed with new data supporting SLS-004’s development, Seelos plans to advance studies of SLS-004 in DLB in preclinical models and will disclose further developments of the therapy in the future.
Seelos also announced in May that it had received a notice of allowance in the U.S. for an additional patent for its candidate SLS-007, a peptide inhibitor designed to inhibit alpha-synuclein protein aggregation in patients with PD. The patent now covers the methodology for treating several neurodegenerative diseases, including AD, Parkinson’s disease, DLV and multiple system atrophy.
The drug is currently being evaluated in preclinical studies to establish target engagement and pharmacokinetic and pharmacodynamic pro Like SLS-004, data from the study is also anticipated in the second half of 2022.
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