D25AC00322

DAMAGE OF A SINGLE ORGAN ACCOUNTS FOR THE MAJORITY OF HUMAN ILLNESSES WITH LIVER DISEASE AFFECTING OVER 500 MILLION PEOPLE AND ACCOUNTING FOR OVER 2 MILLION ANNUAL DEATHS WORLDWIDE. AT THIS TIME THE ONLY CURATIVE THERAPY IS ORTHOTOPIC TRANSPLANT OF A DONOR LIVER. HOWEVER DUE TO THE SEVERE SHORTAGE OF DONOR ORGANS TRANSPLANTS ARE LIMITED TO ONLY A FRACTION OF THE POPULATION WHO MIGHT BENEFIT FROM LIVER REPLACEMENT THERAPY LARGELY DUE TO THE LOW AVAILABILITY AND HIGH COST AND COMPLEX SURGERY AND LIFELONG IMMUNOSUPPRESSION REQUIREMENTS. AS SUCH MILLIONS OF PATIENTS ARE LEFT WITHOUT AN ACCESSIBLE SOLUTION.THE IMPLANT TEAM AIMS TO ADDRESS THIS CHALLENGE BY ESTABLISHING THE FIRST OFF THE SHELF ENGINEERED PRODUCT TO ADDRESS LIVER FAILURE. THE SOLUTION IS BUILT ON SEVERAL NEXT GENERATION TECHNOLOGIES INCLUDING TOOLS TO DRIVE INDUCIBLE PLURIPOTENT STEM CELL DIFFERENTIATION INTO ALL REQUIRED CELLS OF THE LIVER AND IMMUNE ENGINEERING TO ALLOW ALLOGENEIC CELLS TO EVADE IMMUNE REJECTION AND BIOREACTORS TO FACILITATE MANUFACTURING AND MATURATION OF CELLS AND ENGINEERED TISSUES AT LOW COST AND SIMPLE BIOFABRICATION TECHNOLOGIES TO BUILD CLINICAL SCALE CONSTRUCTS. IMPLANT WILL GENERATE A UNIVERSAL AND TRANSPLANT READY GRAFT THAT COOPERATES WITH THE NATIVE LIVER TO MAINTAIN HOMEOSTASIS AND SOMETIMES EVEN LEADING TO ITS RECOVERY AND REGENERATION THROUGH METABOLIC OFFLOADING AND BRINGING HOPE TO MILLIONS OF PATIENTS FOR WHOM THE CURRENT LIVER TRANSPLANT PARADIGM FAILS.THE PROJECT WILL CULMINATE WITH A GMP MANUFACTURED BIOBANK OF CELLS AND UNIVERSAL LIVER TISSUES WITH EFFECTIVENESS DEMONSTRATED IN HUMANIZED MICE AND HUMANIZED PIG MODELS TO SUPPORT TECHNOLOGY TRANSLATION TOWARDS CLINICAL TRIALS. BEYOND IMPLANT WE EXPECT THE PATH TO REGULATORY APPROVAL TO PROGRESS THROUGH A NARROW INDICATION ULTIMATELY INTO SERVING THE MILLIONS OF PATIENTS WITH SERIOUS LIVER DISEASES. OVERALL THIS PROJECT AIMS TO PROVIDE A SOLUTION THAT WILL REVOLUTIONIZE THE TREATMENT OF LIVER DISEASE AND SERVING ALL POPULATIONS MANY OF WHOM ARE CURRENTLY EXCLUDED FROM TRANSPLANT.

President And Fellows Of Harvard College

SIGNIFICANT HEALTH IMPACT FOR THE FUTUREOF ORGAN TRANSPLANTATION AND PAVE THE WAY FOR THE NECESSARY NEXT STEPS IN SCALING TISSUE AND ORGANENGINEERING.

93.384 - ADVANCED RESEARCH PROJECTS AGENCY for HEALTH (ARPA-H)

Interior Business Center (IBC) [DOI]

Suffolk, Massachusetts United States

County-Wide

D-AQD-FA-25-013