Passage Bio, Inc. announced that the first patient has been dosed in the 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. FTD is a form of early onset dementia with no approved disease-modifying therapies. The upliFT-D clinical study evaluates PBFT02 as a single dose delivered via intra-cisterna magna (ICM) injection.

This gene therapy uses an AAV1 viral vector to deliver a functional copy of the GRN gene encoding progranulin (PGRN) to a patient's cells. PGRN is a complex and highly conserved protein thought to have multiple roles in cell biology, development and inflammation. Emerging evidence suggests that PGRN deficiency may contribute to lysosomal dysfunction.

FTD-GRN is a devastating disease with no approved disease-modifying therapies, and are hopeful this trial will provide evidence that PBFT02 could become a meaningful treatment option for adults living with FTD-GRN. The upliFT-D clinical study evaluates PBFT02 as a single dose delivered via intra-cisterna magna (ICM) injection. This gene therapy uses an AAV1 viral vector to deliver a functional copy of the GRN gene encoding progranulin (PGRN) to a patient's cells.

PGRN is a complex and highly conserved protein thought to have multiple roles in cell biology, development and inflammation. Emerging evidence suggests that PGRN deficiency may contribute to lysosomal dysfunction.