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Driving Innovation.

Bringing life-saving drugs to patients with rare diseases
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Pipeline

Life-saving drugs.

At OrphAI Therapeutics, we are focused on identifying and developing treatments for rare orphan conditions. We use a variety of methods to identify promising assets to add to our pipeline. Our current clinical stage drug candidates target several orphan disorders including amyotrophic lateral sclerosis, pulmonary hypertension, bronchiolitis obliterans post lung transplant, pulmonary sarcoidosis, Ewing Sarcoma, Rhabdoid Tumor, and SWI/SNF mutated or dysregulated cancer.

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