Path to Commercialization ofthe first "off the shelf" T cell product

ABSTRACT The adoptive transfer of virus specific T cells has produced remarkable clinical results in patients with viral diseaseHoweverbroader implementation of this therapy has been limited by theiprohibitive production costsiicomplexity of manufactureiiiprolonged time for preparation and product releaseandivthe requirement for individualizedpatient specific productsOver the pastyears we have systematically addressed these problemswe simplified and refined our manufacturing technologyremoved biohazardous components and employed a new cell expansion platform using a gas permeable culture device which promotes the proliferation and survival of large cell numbers in a GMP compliant closed system with minimal technician interventionFinallywe have established the clinical benefit associated with the infusion of partially HLA matched virus specific T cells that are prospectively generated and bankedmaking them available for immediateoff the shelfuseThuswith the purpose of moving beyond highly specialized academic centers we established a Baylor College of Medicine affiliated company called ViraCyte with the goal of commercializingoff the shelfvirusspecific T cellsOur productViralym Bis a bank of T cell lines with specificity for BK viruswhichin immunocompromised individuals including childrenis responsible for severe clinical syndromes like hemorragic cystitis and nephropathy that can lead to significant morbidity and prolonged hospital stayOur therapy is intended for the treatment of drug resistant infections diseasea condition that afflicts less thanpersons in the United States and for which there is no standard of careThusin the current application we will test the safety and potential for anti viral activity of Viralym B in allogeneic hematopoietic stem cell transplant recipientsIn additionwe will further de risk the transfer of this technology to a private sector contract manufacturing organizationCMOby transitioning our cell production from an open to an entirely closed system and validating our scale up manufacture protocolSuccess of this application will lay the foundation for a Phase IIb study to confirm the efficacy ofoff the shelfViralym B cells and facilitate market approvalthereby moving T cell therapy for BK virus into the public domain as a standard of care Project narrative ViraCyte was created as a collaborative effort between scientistsclinicians and entrepreneurs to bring antiviral T cell therapy to patients with severe and intractable viral infectionsIn the current application we propose undertaking necessary steps required to commercialize the firstoff the shelfT cell therapy productViralymBVirus specific Lymphocytes directed against BK virusthat is intended for use in immunocompromised patients with severe and refractory BK virus infectionUpon completionthis study will provide crucial data that will allow us to move towards product approval and fill the largely unmet need for effective therapeutic options in this patient group