Seelos Therapeutics Inc. (NASDAQ:SEEL) And Duke University Enter Sponsored Research Agreement For SLS-004 Study

Seelos Therapeutics Inc. (NASDAQ:SEEL) and Duke University have entered a Sponsored Research Agreement (SRA). The agreement is for using an MPTP-induced Parkinson’s Diseases (PD) mouse model to create an in-vivo proof-of-concept trial showing that LV-dCas9-DNMT3A virus administration can delay and/or prevent PD as well as test safety and efficacy of SLS-004. Also, other subsequent pre-clinical models will be used in further validating this investigational product.

Seelos commences the SLS-004 study at Duke

Raj Mehra, the CEO and Chairman of Seelos, expressed his delights in the commencement of the in vivo target engagement SLS-004 study at Duke University. The study builds on the work the company commenced in the spring that focuses on designing a vector that can induce and suppress phenotypes related to Parkinson’s disease. Mehra said that the initiation of the next part of the study at Duke will help the company validate Parkinson’s disease approach.

The company has equally commenced work on the design of a vector that can induce and suppress PD-associated phenotypes through carrying of a unit which overexpresses the alpha-synuclein protein as well as a unit mediating inducible suppression of alpha-synuclein. This process aims at creating a next-generation suppressive unit that can carry an effective effector molecule compared to previously used, which are in DNA methyltransferase 3A and 3L (DNMT3A&L) form. The research will help advance a potential proprietary, precise, and effective tool for reversing SNCA pathologies providing a valuable new therapeutic approach for PD treatment.

SLS-004 meant to modulate SNCA gene overexpression

Seelos is developing SLS-004 as a novel epigenome editing strategy for modulating SNCA gene expression through DNA methylation modification. SLS-004 uses a collective lentiviral vector haboring dCAs9-DNA methyltransferase 3A. The vector helps in enriching DNA methylation within CpGs Island at SNCA intron 1 area. Most importantly, the system has shown to be effective in downregulation of SNCA overexpression by 30% in hiPSC-derived dopaminergic neurons in Parkinson diseases with SNCA locus triplication. Interestingly, SNCA expression reduction is mediated through a system developed to reduce disease-associated phenotypes sufficiently.