OrphAI Therapeutics

OrphAI Therapeutics' mission is to transform the lives of patients with rare diseases. The company is currently developing three investigational therapies across multiple orphan indications: AIT-101, a first-in-class PIKfyve inhibitor, which recently completed a Phase 2a clinical trial for amyotrophic lateral sclerosis (ALS); LAM-001, a proprietary inhaled formulation of rapamycin, currently in Phase 2 clinical trials for the treatment of Group 1 and Group 3 pulmonary hypertension and bronchiolitis obliterans syndrome (BOS); AIT-102, a proprietary analog of rapamycin, currently in Phase 2 clinical trials for the treatment of Group 1 and Group 3 pulmonary hypertension and bronchiolitis obliterans syndrome (BOS); and AIT-102, a patented analog of mycophenolic acid, in preclinical development for the treatment of SWI/SNF-mutant or dysregulated tumors.