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Verge Genomics PIKfyve inhibitor (peripheral indications)
- Development stage
- Preclinical
- Lead developer
- Verge Genomics
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
This program involves the development of small molecule inhibitors targeting the PIKfyve kinase for unspecified peripheral (non-CNS) indications. PIKfyve is a lipid kinase that plays a critical role in regulating the endolysosomal system, which is responsible for protein trafficking and degradation within cells. Dysfunction in this pathway is linked to various diseases characterized by impaired protein homeostasis. The program was identified using Verge Genomics' proprietary AI-powered CONVERGE platform, which integrates human tissue data to discover novel therapeutic targets. While Verge's lead PIKfyve inhibitor, VRG50635, is currently in clinical trials for amyotrophic lateral sclerosis (ALS), this peripheral program is in the earlier stages of preclinical development.
- Other names
- PIKfyve program (peripheral indications)
02
Targets
PIKFYVE (Phosphoinositide kinase, FYVE-type zinc finger containing enzyme)
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