Project Grant R44GM134729

SIMPLE AND EFFECTIVE METHOD FOR CELL SEPARATION DURING PRECLINICAL RESEARCH AND CELL THERAPY MANUFACTURING - PROJECT SUMMARY PROTEIOS IS AN EMERGING STARTUP DRIVEN TO REDUCE THE RISING COST OF HEALTHCARE. PRESCRIPTION DRUG COSTS ARE THE FASTEST RISING COMPONENT AND ARE CONTRIBUTING TO THE DRAMATIC INCREASE IN HEALTHCARE COSTS. THE WORLDWIDE FOCUS OF PHARMACY AND BIOTECHNOLOGY IS NOW IN TWO AREAS: 1) BIOPHARMACEUTICAL DEVELOPMENT AND PRODUCTION AND 2) CELL THERAPIES. CELL THERAPIES HAVE DEMONSTRATED ENORMOUS PROMISE FOR TRANSFORMING THE TREATMENT OF CANCER AND OTHER DEBILITATING DISEASES, AND THE FDA HAS ALREADY APPROVED THEIR USE FOR THE TREATMENT OF LEUKEMIA AND LYMPHOMA. HOWEVER, SOME CELL THERAPIES CAN COST AS MUCH AS $350,000 PER TREATMENT, WHICH HAS MADE THESE LIFE-SAVING TECHNOLOGIES INACCESSIBLE TO MANY PATIENTS. THE OBJECTIVE OF THIS PROPOSAL IS TO PROVIDE PRECLINICAL RESEARCHERS WITH A SIMPLE AND COST-EFFECTIVE CELL SEPARATION TOOL TO ISOLATE AND STUDY CELL SUBPOPULATIONS FOR THEIR POTENTIAL USE AS NEW CELL THERAPEUTICS. PROTEIOS' CELL SEPARATION TECHNOLOGY WILL ALSO BE EMPLOYED DURING CELL THERAPY MANUFACTURING; PROTEIOS WILL DEVELOP CELL SEPARATION CARTRIDGES FOR USE WITH ITS CGMP CELL THERAPY MANUFACTURING INSTRUMENT CURRENTLY UNDER DEVELOPMENT. OUR GOAL FOR PHASE II OF THE SBIR AWARD IS TO DEVELOP NEW PROTEIOS CHIMERAS FOR THE 15 MOST COMMONLY STUDIED CELL SUBPOPULATIONS USED FOR AUTOLOGOUS AND ALLOGENEIC CELL THERAPIES. CHIMERAS FORM A CRITICAL COMPONENT OF THE PROTEIOS CHIMERA PLATFORM THAT WILL EMPOWER OUR PRECLINICAL RESEARCH R & D CELL SEPARATIONS KITS AND CELL SEPARATION CARTRIDGES FOR MANUFACTURING. THE PLATFORM PROVIDES A TAG-FREE, ANTIBODY-FREE ISOLATION PLATFORM BASED ON PROPRIETARY PROTEIN TECHNOLOGY AND NUCLEIC ACID APTAMERS BY IMMUNOAFFINITY CAPTURE OF CELLS THAT EXPRESS CLUSTER OF DIFFERENTIATION (CD) MARKER PROTEINS. OUR APPROACH AVOIDS CHALLENGING AND COSTLY STEPS WHERE ANTIBODIES OR OTHER PROTEINS ARE COVALENTLY MODIFIED TO BIND TO BEADS OR SMALL MOLECULES. ADDITIONALLY, OUR APPROACH ELIMINATES COMMONLY USED REAGENTS THAT CAUSE OXIDATIVE DAMAGE TO CELLS. WHEN COMMERCIALIZED, THIS APPROACH WILL ALLEVIATE SOME OF THE CONCERNS WITH CURRENT TECHNOLOGIES - SUCH AS HIGH COST, LONG DEVELOPMENT TIMES, LACK OF AUTOMATION AND CLINICAL-GRADE REAGENTS, AND COMPLICATED, MULTISTEP CHEMISTRY. THE DEVELOPMENT OF PRODUCTS FOR CELL ISOLATION USING THE PROTEIOS CHIMERA PLATFORM HAS THE POTENTIAL TO REDUCE THE COST OF CELL SEPARATIONS BY MORE THAN TENFOLD. FURTHERMORE, THIS TECHNOLOGY COULD TRANSFORM THE PRODUCTION PROCESS FOR THERAPEUTIC CELLS TO MAKE LIFE-SAVING CELL THERAPIES AVAILABLE TO MORE INDIVIDUALS.