Passage Bio, Inc. (NASDAQ:PASG), a clinical stage genetic medicines company focused on improving the lives of patients with neurodegenerative diseases, will present updated data from the ongoing global Phase 1/2 upliFT-D clinical trial evaluating PBFT02, an adeno-associated virus (AAV)-delivery gene therapy for the treatment of patients with frontotemporal dementia (FTD) with granulin (GRN) mutations, at the 14th International Conference on Frontotemporal Dementias (ISFTD2024). The poster presentation will include safety and biomarker data from all treated patients (n=5) in the first cohort of the study.

Juan Chavez, M.D., vice president of clinical development, will share the data during an oral presentation, Interim Safety and Biomarker Data From upliFT-D Trial of PBFT02 in FTD with GRN Mutations, on Friday, September 20, 2024 at 9:38 a.m. GMT.

"We are very encouraged by the positive Cohort 1 data from our upliFT-D trial demonstrating that intra-cisterna magna delivery of Dose 1 of PBFT02 resulted in robust and durable increases in CSF PRGN expression, with elevated levels maintained for up to one year after treatment," said Will Chou, M.D., president and chief executive officer of Passage Bio. "Furthermore, PBFT02 continued to be well-tolerated among all Cohort 1 patients who received enhanced immunosuppression, with no serious adverse events or evidence of clinically significant immune responses observed in these patients. These results underscore the potential of PBFT02 as a best-in-class progranulin-raising therapy and further solidify our strategy to advance this one-time treatment in additional neurodegenerative diseases. We look forward to showcasing these findings during our poster session, and are thankful for the participants, their caregivers, and clinical trial investigators for their support of this study."