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Passage Bio Receives FDA Clearance of IND Application for PBFT02 Gene Therapy Candidate for Treatment of Patients with Frontotemporal Dementia with Granulin Mutations
– Second Product Candidate Expected to Enter Clinic in First Half of 2021 – Preclinical Data Underscore Treatment Potential for PBFT02 in Frontotemporal
About this update from Passage Bio, Inc.
[{"type":"text","content":"– Second Product Candidate Expected to Enter Clinic in First Half of 2021 – Preclinical Data Underscore Treatment Potential for PBFT02 in Frontotemporal Dementia with Granulin (GRN) Mutations, a Devastating, Progressive Disorder Impacting Adults with No Approved Disease-Modifying Therapy Options PHILADELPHIA, Jan. 28, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders, today announced that the U.S. Food and Drug Administration (FDA) has cleared an investigational new drug (IND) application for PBFT02, an adeno-associated virus (AAV)-delivery gene therapy that is being studied for the treatment of patients with Frontotemporal Dementia (FTD) with granulin (GRN) mutations. FTD is a debilitating form of early onset dementia that currently has no approved disease-modifying therapies. “We are pleased to be advancing our second therapy into clinical development in our quest to bring transformative medicines to patients who need them,” said Bruce Goldsmith, Ph.D., chief executive officer of Passage Bio. “FTD can have a devastating impact on a person’s quality of life and create a substantial caregiving and economic burden for families. We are excited to investigate the potential of PBFT02 as a treatment for FTD-GRN as we initiate our clinical development program in the coming months.” FTD is one of the more common causes of early-onset (midlife) dementia, causing impairment in behavior, language and executive function, and occurs at similar frequency to Alzheimer’s disease in patients younger than 65 years. In approximately 5 to 10 percent of individuals with FTD – 3,000 to 6,000 in the United States – the disease occurs because of mutations in the GRN gene, causing a deficiency of progranulin (PGRN). PGRN is a complex and highly conserved protein. The mechanism by which PGRN deficiency results in FTD is uncertain, but increasing evidence points to PGRN’s role in lysosomal function. The rapid progression of FTD results in an average survival of eight years after onset of symptoms. Passage Bio is developing PBFT02 to treat FTD-GRN as a single dose delivered via intra-cisterna magna (ICM) injection. The gene therapy utilizes an AAV1 viral vector to deliver a modified DNA encoding the GRN gene to a p...