AddGraft Therapeutics
AddGraft Therapeutics cell therapy platform is building stem cell based bio factories capable of producing and deploying any protein directly into a patient’s bloodstream. AddGraft hopes to address some of the major problems with current cell therapies including toxicity, transgene capacity, dose flexibility / control, stable long term expression, and complicated CMC with an ex vivo based method to introducing genetically modified stem cells. In preclinical mouse models, Addgraft has demonstrated the ability to use their platform to treat multiple disease categories including Neurological Disorders, Metabolic Disorders, and Enzyme Deficiencies. For their beachhead, they are targeting the rare disease PKU with a therapy that has demonstrated great pre clinical efficacy and hopes to solve for some of the major problems associated with current PKU treatment.