PHILADELPHIA, PA — Passage Bio, Inc. (Nasdaq: PASG) announced that the first patient was recently 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.
“Dosing the first patient in our upliFT-D trial is an important milestone for the Passage Bio team and for the PBFT02 program, our first program in the clinic for adults,” said Edgar (Chip) Cale, interim chief executive officer of Passage Bio. “We look forward to continuing our important work to develop PBFT02 as a potential treatment option for the thousands of people living with FTD-GRN. We are grateful for the support from the families and clinical trial investigators who have chosen to participate in our studies.”
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 we are hopeful this trial will provide evidence that PBFT02 could become a meaningful treatment option for adults living with FTD-GRN,” said Mark Forman, M.D., Ph.D., chief medical officer of Passage Bio. “Our approach, which employs the AAV1 vector and ICM administration, provides a potential opportunity to achieve higher than normal levels of PGRN in the CNS, thereby overcoming the PGRN deficiency in GRN mutation carriers with a diagnosis of early symptomatic FTD-GRN. We look forward to building on our preclinical data with this Phase 1/2 trial.”
FTD is one of the more common causes of early-onset dementia. In approximately 5 to 10 percent of individuals with FTD–3,000 to 6,000 individuals in the United States–the disease occurs because of mutations in the GRN gene, causing a deficiency of PGRN. FTD causes impairment in behavior, language and executive function, as well as changes in personal and social conduct including loss of inhibition, apathy, and social withdrawal. Progression of FTD results in an average survival of eight years after the onset of symptoms.
The U.S. Food and Drug Administration (FDA) has granted PBFT02 Fast Track and Orphan Drug designations. PBFT02 has also received an Orphan designation from the European Commission.
upliFT-D continues to enroll patients with early symptomatic FTD-GRN. If you are interested in a referral to a clinical trial site, contact Passage Bio here to learn more.
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