Development of an intellectual property (IP) strategy for the commercial advancement of a Direct Phase II SBIR-funded human platelet bioreactor

PROJECT SUMMARY ABSTRACT Platelet BioGenesis is developing a microfluidic bioreactor to produce human platelets at clinical scale Platelets are the andapos band aidsandapos of the bloodstream responsible for clot formation and blood vessel repair Low platelet count is a significant consequence of cancer treatment transplant and surgery for which plate lets are a critical first line therapy to prevent mortality due to uncontrolled bleeding Platelets are de rived exclusively from volunteer donors Risk of bacterial growth during room temperature storage limits plate let shelf life to days of which are expended by bacterial screening and during transport to hospitals As a result blood centers typically do not have more than a day platelet inventory available for transfu sion making platelet unit inventory especially vulnerable to routine depletion To address this ma jor unmet need we are developing a platelet bioreactor that reproduces key features of adult bone marrow physiological microenvironment to trigger platelet production from human induced pluripotent stem cells hiP SCs a replenishable source of progenitor cells which can be stored frozen for years at clinical commercial scale This SBIR CRP Program proposal for Technical Assistance SB outlines a comprehensive intellectual property strategy for Platelet BioGenesis to develop a proprietary patent protected technology to produce cGMP compliant human bioreactor derived platelets at commercial scale for clinical use in specific aims Aim Understanding the Patent Landscape We will work with IP counsel to identify and assess licens ing various third party patents and patent applications related to megakaryocyte differentiation protocols and the platelet bioreactor perform further patent searches landscape and or freedom to operate to ensure that we are able to commercialize our technology without infringement and or identify any additional third party patents that may be licensed by us to further improve our competitive commercial position Aim Protecting Proprietary Technology to Enable Commercialization We will work with IP counsel to identify intellectual property generated by us and will formulate a strategy for protecting such intellectual prop erty either by filing patent applications or ensuring that the technology is maintained as a trade secret Aim IP Agreements We will work with the IP counsel to review our non disclosure agreements em ployment or consultant agreements to ensure the ownership and protection of our intellectual property and negotiate and enter into license agreements with cGMP compliant hiPSC suppliers for access to relevant cell lines cGMP hiPSC line master banks will be maintained and differentiated into megakaryocytes under estab lished supply agreements using proprietary cell culture protocols PROJECT NARRATIVE Human platelets are presently derived exclusively from volunteer donors and have a shelf life of days mak ing platelet unit inventory sensitive to depletion and putting platelet recipients at risk of sepsis and viruses dur ing surgery pregnancy and birth cancer HIV burn treatment and transplant There are currently no licensed therapeutics that immediately increase platelet counts and insufficient platelet supply to meet projected US demand M platelet units year in Platelet BioGenesis has identified cGMP compliant hiPSC lines validated megakaryocyte differentiation protocol and platelet storage permissive media and established a mi crofluidic bioreactor to rapidly trigger functional platelet production at clinical commercial scale We now seek to develop a comprehensive IP strategy for linking these key technologies which is necessary to advance bio reactor derived platelets bdPLTs for clinical use