Seelos Therapeutics has signed an agreement with Duke University in North Carolina to conduct a pre-clinical study of SLS-004, one of its gene therapy candidates, as a treatment for Parkinson’s disease.
The proof-of-concept study will seek to determine if the investigational therapy can prevent or delay Parkinson’s disease and also test its safety and effectiveness. If warranted, future studies will investigate it further and seek to validate SLS-004 in other pre-clinical models.
The company signed a Sponsored Research Agreement with Duke University, where SLS-004 was developed, to use its MPTP-induced Parkinson’s mouse model. MPTP is a neurotoxin that causes brain inflammation, and often is used to study Parkinson’s.
SLS-004 consists of a modified, harmless virus, called a lentivirus, that delivers the DNA methyltransferase 3A (DNMT3A) enzyme and CRISPR-dCas9 gene-editing components to cells.
Methylation is the addition of the chemical methyl group to specific locations along DNA. This regulates gene activity by preventing genes from being made into proteins, effectively turning them off.
Cells use methylation naturally throughout development and as a defense against foreign DNA. SLS-004 seeks to fine-tune this process by using gene-editing technology to specifically target SNCA.
Seelos hopes that turning down the production of alpha-synuclein will stop or limit toxic clumps of that protein from forming inside nerve cells. These clumps, also called Lewy bodies, damage and eventually kill the cells.
“We are extremely pleased to begin the in vivo target engagement study with SLS-004 at Duke as it builds onto the work we began in the spring focused on designing a vector capable of inducing and suppressing Parkinson’s related phenotypes,” Raj Mehra PhD, chairman and CEO of Seelos, said in a press release.
“Initiating this next portion of the studies at Duke should help us further validate this approach in Parkinson’s,” he added.
Seelos also began designing a vector that can alternately amplify and reduce alpha-synuclein activity. This vector aims to suppress gene activity more effectively by carrying another DNA methyltransferase, DNMT3L, in addition to DNMT3A.
The company hopes this research will deliver a new, effective, and precise tool to reverse disorders involving SNCA, as well as a new way to treat Parkinson’s.
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