Details

Our gene therapy product candidate, PBFT02, an AAV1 viral vector to deliver a modified DNA encoding the granulin gene (GRN) to a patient’s cells. The vector will be delivered directly to the cerebrospinal fluid by a single injection to the cisterna magna (ICM injection). The goal of this vector and delivery approach is to provide higher-than-normal levels of the progranulin protein (PGRN) to the CNS to overcome the progranulin deficiency in GRN gene mutation carriers.