OrphAI Therapeutics


OrphAI Therapeutics is dedicated to developing life-saving drugs for patients in need, focusing on treatments for conditions with unmet medical needs such as pulmonary hypertension, post lung transplant bronchiolitis obliterans, and transcriptionally dysregulated cancers. The company advances innovative medicines through clinical trials and aims to address critical health challenges.

Industries

artificial-intelligence
biotechnology
genetics
medical
therapeutics

Nr. of Employees

medium (51-250)

OrphAI Therapeutics

101 College Street, Suite 210, New Haven, CT 06510


Patents

Rapamycin for the treatment of lymphangioleiomyomatosis

US-12171874-B2

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Combination therapy with apilimod and glutamatergic agents

US-11957688-B2

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Rapamycin for the treatment of lymphangioleiomyomatosis

US-11744797-B2

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Inhalable rapamycin formulation for treating age-related conditions

US-11648199-B2

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Inhalable rapamycin formulation for the treatment of pulmonary hypertension

US-11491143-B2

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Combination therapy with apilimod and glutamatergic agents

US-11439649-B2

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Products

LAM-001

Investigational small-molecule drug candidate under clinical development for Group 1 and Group 3 pulmonary hypertension and for bronchiolitis obliterans syndrome post lung transplant.

AIT-102

Acquired cancer drug candidate being advanced for transcriptionally dysregulated tumors.

AIT-101

Investigational therapy previously developed for amyotrophic lateral sclerosis (ALS); further development on hold pending non-dilutive funding. Prior biomarker-driven Phase 2a data reported.


Services

Design and operational conduct of sponsor-led clinical studies including protocol implementation and trial oversight.

Expertise Areas

  • Clinical trial management
  • Biomarker-driven clinical development
  • Non-clinical and preclinical development
  • Regulatory strategy and orphan drug designation
  • Show More (3)

Key Technologies

  • Biomarker assays and translational biomarkers
  • Clinical protocol development
  • ClinicalTrials.gov registry and public study reporting
  • Preclinical candidate development
  • Show More (2)

News & Updates

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