💡 Discover AIT-101: OrphAI Therapeutics' groundbreaking treatment for ALS has now earned Orphan Drug Designation in the EU! With its unique ability to clear toxic aggregates, AIT-101 is leading the charge against ALS, offering hope to patients worldwide.

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OrphAI Therapeutics has been granted Orphan Drug Designation by the EU for AIT-101, aimed at treating amyotrophic lateral sclerosis (ALS). Following its US FDA approval, this marks a significant step toward battling this neurodegenerative disease.

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OrphAI Therapeutics Appoints Jay Fine, PhD to Board of Directors

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'We have grown into a clinically focused company with a mission to bring life-saving drugs to patients with rare diseases. Our new name, OrphAI Therapeutics, echoes our progress & our legacy in leveraging AI for drug development.' - Brigette Roberts, CEO

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OrphAI Therapeutics will be at the 22nd Annual NEALS (Northeast ALS Consortium) meeting being held October 4 - 6, 2023 in Clearwater Florida.

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AI Therapeutics is now OrphAI Therapeutics Inc. This rebrand signifies our sharpened focus on developing treatments for orphan diseases. Visit our newly launched site: OrphAI-Therapeutics.com

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Exciting News! AI Therapeutics has rebranded to OrphAI Therapeutics Inc. as we further our commitment to treating rare diseases. Our new name captures our evolution & dedication to those in need.

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Our 24-week study aims to evaluate LAM-001's safety, tolerability, and efficacy in 15 adults with advanced PAH. Primary endpoints include changes in peak oxygen uptake, among other hemodynamic metrics.

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What sets LAM-001 apart? Dr. Aaron Waxman, Principal Investigator, explains: 'Unlike current therapies, LAM-001 addresses the cellular remodeling that is the root cause of PAH by modulating both the mTOR and BMPR2 pathways.'

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The 48-week study at UCSF will involve 30 adults newly diagnosed with BOS. Objectives? Safety, efficacy, and progression-free survival of LAM-001. Secondary endpoints include improvements in FEV1, quality of life measures, and more. Stay tuned for results!
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BOS is a significant challenge post-lung transplant, affecting nearly 50% of patients by 5 years and is a leading cause of death. But, LAM-001 could be a game-changer by directly targeting lung tissue!

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Exciting news from AI Therapeutics, we have launched a Phase 2 study at UCSF for LAM-001, an inhaled form of sirolimus, aimed at treating bronchiolitis obliterans syndrome (BOS) in post-lung transplant patients. #LungHealth #ClinicalTrials

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Mr. Scheer, our new Chairman, brings decades of experience to AI Thera, having founded, built and advised multiple #biotech companies, including Achillion, Viropharma, OraPharma and Esperion Therapeutics which were cumulatively acquired for nearly $7B.

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David Scheer, a seasoned company builder and advisor with a proven track record of creating value in the biotechnology industry, has been appointed Chairman of AI Therapeutics Board of Directors.

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AI Therapeutics Announces Appointment of Biotechnology Industry Leader David Scheer as Chairman of the Board of Director

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Join AI Therapeutics at the American Thoracic Society International Conference! Dr. Antje Prasse will present a co-authored poster on mTOR activity in sarcoidosis patients. Learn more about our work developing novel therapeutics for rare diseases #ATS2023

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Exciting news from AI Therapeutics! Dr. Antje Prasse will present at the 2023 American Thoracic Society International Conference. The poster, co-authored by the company, will highlight high mTOR activity in sarcoidosis patients. #ATS2023

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AI Therapeutics Announces Poster Presentation at the 2023 American Thoracic Society International Conference

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“This study provides early clinical evidence of AIT-101’s ability to clear the toxic protein aggregates that may contribute to the adverse pathology of ALS and provides a strong rationale for further clinical studies.”

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