Sachi Neurotherapeutics Demonstrate Excellent Efficacy and Safety Profile During Pre-Clinical Evaluation
Sachi recently released data on their proprietary discovery engine Nanoligomers™ that rapidly generates a novel family of molecules to selectively up- or down-regulate the expression of proteins of interest reversibly, by binding to targeted DNA or mRNA.
In their work with diverse pool of donor-derived primary human astrocytes and pre-clinical animal models, Sachi demonstrated the ability of sequence-specific Nanoligomers to be administered simply using inhalable or oral drugs, or as intravenous injections that can be designed to easily transport across the blood-brain barrier, and strongly curb neuroinflammation far exceeding their respective small-molecule counterparts. This data suggests Nanoligomers could be developed as potential best-in-class and first-in-class therapeutics to treat neuroinflammation by downregulation of several proinflammatory cytokines, inflammasomes, key transcription factors and their combinations, as upstream regulators and canonical pathway targets. Further, the facile route of administration, strong safety and biodistribution profile, and targeting multiple pathways through gene networks selectively (with minimal off-targeting) opens new avenues for treating neurodegenerative diseases such as Alzheimer’s Disease (AD), Parkinson’s Disease (PD), Multiple Sclerosis (MS), and Creutzfeldt-Jakob disease (CJD).
While translation of these molecules to demonstrate safety and human clinical efficacy is ongoing, Sachi Nanoligomer™ SB.NI_111 was recently shown to reverse PD progression and restore motor function in pre-clinical animal models, renewing hope in millions of patients suffering from these debilitating diseases for development of new and breakthrough medicine using Sachi’s revolutionary platform technology. Sachi plans to unveil the data and pre-clinical outcomes in the coming months to the broader drug-development community and pharmaceutical industry.
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