Drug intelligence / Profile preview

ALT-001

Development stage
Preclinical
Lead developer
Alterras Therapeutics
Modality
Small Molecules
01

Overview

ALT-001 is a small molecule mitophagy enhancer originally developed by **Alterras Therapeutics**. It functions by stimulating the **ULK1/Rab9-mediated alternative mitophagy** pathway, a cellular process essential for the selective clearance of damaged or dysfunctional mitochondria. By restoring mitophagic flux, ALT-001 aims to reduce neuroinflammation and cellular stress. The drug is currently in preclinical development for several indications, including **pulmonary arterial hypertension (PAH)** and **Alzheimer's disease-related cognitive impairment**. Research has also demonstrated its potential to suppress **herpes simplex virus type 1 (HSV-1)** infection and alleviate associated neuroinflammation by improving mitochondrial health in microglia.

02

Targets

ULK1RAB9A (Ras-related protein Rab-9A)

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