JAG301

JAG301 is an investigational AAV-based gene therapy being developed by Jaguar Gene Therapy for Type 1 diabetes, designed to restore endogenous insulin production by reprogramming pancreatic cells. Using an AAV9 vector to deliver the transcription factor gene PAX4 to the pancreas, JAG301 aims to convert glucagon-secreting alpha cells into functional insulin-producing beta cells via transdifferentiation, thereby addressing the loss of beta cell mass that underlies Type 1 diabetes rather than simply replacing insulin.[1][5][13][14][19] The program is currently in preclinical development, supported by exclusively licensed technology and proof-of-concept data from Tulane University, and is being positioned for use in a subpopulation of newly diagnosed Type 1 diabetes patients.[1][8][13][14]