A Novel Target for CAR T Cell Therapy Using Precision Oncology Approaches

Project Summary This year cancer is expected to claimmillion lives worldwideWorld Health OrganizationItrepresents the leading cause of death in the United States and its prevalence continues to groweach yearConventional treatment options tend to be limited in efficacy with highly toxic sideeffectsNewermonoclonal antibody based therapies have offered promise to for some types ofcancerparticularly blood tumorsbut these often fall short as wellTypicallythese treatments are delivered based on the location of the tumor and do not consider whether theindividual patient actually expresses the targetIn additionthe most common tumor antigens tendto have significant expression on healthy tissues as wellcreatingon targetoff tumortoxicitiesRecent advances in nucleotide sequencing technology have created a proliferation inprecision oncology capabilitiesThese new capabilities have allowed patients and their tumors tobe stratifiedknowing exactly which patientstumors express druggable targetsThese advanceshave created a gap in the field between the ability to identify unique tumor markers and theability deliver therapies based on those markersThe maturation of precision oncology establishesan urgent need for development of therapeutics to novel targets intended to treat cancers based ontheir genetic composition rather than their locationUsing proprietary computational biology techniqueswe have populated a list of antigens that arevery highly expressed on small subsetsof tumors but are not expressed or have very lowexpression on healthy tissuesWith this Phase I SBIRwe hope to advance our first therapeuticsto one of these targets through pre clinical proof of concept experiments and lead candidateselectionThese experiments will drive the selection of the optimal format of this therapeuticand will justify the IND enabling pre clinical work required to prepare these novel therapeuticsfor delivery to patients Project Narrative Despite recent advances in the diagnosis and treatment of canceroverAmericans will die from the disease this yearWe are creating new therapies toward unique targetsexpressed only on cancer cellsand establishing a critical pathway for identifying which patients have tumors expressing these targetsIf successfulthis work will provide an accelerated route for creating new cancer drugs and delivering them to the patients who need them