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
Nr. of Employees
medium (51-250)
OrphAI Therapeutics
Guilford, Connecticut, United States, North America
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.
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
Sponsor-led clinical trial execution
Design and operational conduct of sponsor-led clinical studies including protocol implementation and trial oversight.
Sponsor-led clinical trial execution
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
Key Technologies
- Biomarker assays and translational biomarkers
- Clinical protocol development
- ClinicalTrials.gov registry and public study reporting
- Preclinical candidate development