AI Therapeutics Inc., a clinical-stage biopharmaceutical company in Guilford, Connecticut, developing novel therapeutics for rare diseases, recently announced the commencement of an investigator initiated Phase 2 study at UC San Francisco (UCSF) for its novel inhaled form of sirolimus, LAM-001, for the treatment of bronchiolitis obliterans syndrome (BOS) in patients post-lung transplant. BOS remains the leading cause of death after transplantation and affects approximately 50% of all lung transplant recipients by 5 years. Worldwide, 4,600 lung transplantations are performed each year.
“BOS is a major complication in lung transplant patients for which there are no approved therapies. BOS pathology is driven by inflammation of the small airways, fibroproliferation and abnormal regeneration of the epithelium. Progressive narrowing of these airways leads to difficulty breathing and the ultimate need for re-transplantation or death,” said Steven Hays, M.D., medical director of the UCSF Lung Transplant Program, and principal investigator of the trial. “While retrospective analyses have shown that oral sirolimus can improve survival in BOS patients, systemic toxicities have limited its widespread adoption. An inhaled version of sirolimus has the promise of delivering the drug directly to the lung tissue where it is needed while potentially reducing systemic exposures and concomitant toxicities.”
AI Therapeutics was founded by Dr. Jonathan Rothberg, serial entrepreneur and recipient of the National Medal of Technology and Innovation for inventing high-speed “Next-Gen” DNA sequencing, with the goal of utilizing artificial intelligence to accelerate the clinical development of drugs for rare diseases. The company is building out an expansive rare disease pipeline with the help of its Guardian Angel™ Platform, a suite of artificial intelligence tools that use deep learning to understand complex disease biology and the action of potential new therapeutics.
