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OrphAI Therapeutics

Guilford, Connecticut, United Statesorphai-therapeutics.com ↗

Ownership: Private
Development stage: Phase 2

Overview

The company has three investigational therapies in development. AIT-101, a first-in-class PIKfyve inhibitor, recently completed a Phase 2a clinical trial in ALS. LAM-001, a proprietary inhaled form of rapamycin, is currently in Phase 2 for BOS and PAH. AIT-102, a proprietary analogue of mithramycin, is in preclinical development for SWI/SNF mutated or dysregulated tumors.

Therapeutic areas: PulmonologyCardiovascularOncologyRare DiseasesNeurology
Modalities: Small molecule drugsNon-degrading molecular glue
Industry: Biotech

Drug pipeline

3 assets

apilimod

2 more assets in the full pipeline.

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