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Find Clinical Drug Pipeline Developments & Deals by Verge Genomics
VRG50635 is a potential best-in-class, small molecule inhibitor of PIKfyve, a therapeutic target for ALS. It is under phase 1 clinical development for the treatment of amyotrophic lateral sclerosis.
Under the collaboration, Verge will use CONVERGE®, its all-in-human, AI-powered platform, to identify novel therapeutic targets by using data generated from postmortem brain tissue provided by schizophrenia patients and healthy donors.
The collaboration aims to develop a disease model to validate novel targets identified by Verge for Parkinson’s disease (PD), by leveraging Stem Pharm's cutting-edge human-first technologies and expertise.
The collaboration aims to identify novel drug targets for rare neurodegenerative and neuromuscular diseases by leveraging CONVERGE, a full-stack platform that combines highly predictive human tissue datasets with machine learning to find new targets.
VRG50635 is a potential best-in-class small molecule inhibitor of PIKfyve for treatment of ALS. PIKfyve is a new therapeutic target for ALS discovered using CONVERGE™, the company’s all-in-human, AI-powered platform.
VRG50635 is a potent PIKfyve inhibitor that restores endolysosomal function in ALS patient neurons and has shown efficacy in multiple preclinical studies in ALS-relevant models of motor neuron degeneration.
Funding from the Series B will be used to exponentially grow the number of preclinical and clinical programs in Verge’s pipeline and advance its lead programs through clinical testing.
In an in vivo study of SARS-CoV-2 infected hamsters, following four-day treatment, animals treated with VRG101 at 50 mg/kg showed significant improvement in lung pathology compared to vehicle controls.