Will Chou
CEO
Passage Bio
Passage Bio is a gene therapy company focused on developing genetic medicines for monogenic central nervous system diseases, specifically targeting conditions like frontotemporal dementia caused by progranulin gene mutations. Their lead clinical program, PBFT02, aims to provide a permanent treatment solution that alters the disease's progression and improves patient outcomes.
- Biotechnology
Updated 7 months ago
Funding
Raised to date
$154MRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Funding rounds are not available yet.
Founders
Product
Problem
Many central nervous system (CNS) diseases, particularly monogenic disorders, lack effective long-term treatments, leading to progressive neurodegeneration and significant impact on patients' lives. Traditional therapeutic approaches often fail to address the underlying genetic causes of these conditions.
Solution
Passage Bio is developing gene therapies designed to address the root genetic causes of monogenic CNS diseases. Their approach involves delivering a functional copy of the mutated gene to restore normal protein expression and function within the central nervous system. By targeting the underlying genetic defect, Passage Bio aims to provide durable, potentially curative treatments that halt or reverse disease progression, offering improved outcomes for patients with limited therapeutic options. Their lead program focuses on frontotemporal dementia (FTD) caused by progranulin (GRN) gene mutations.
Target Audience
The primary target audience includes patients diagnosed with monogenic CNS diseases, such as frontotemporal dementia with progranulin mutations (FTD-GRN), and their families, as well as physicians specializing in neurology and gene therapy.
Features
- PBFT02: A gene therapy candidate for FTD-GRN currently in Phase 1/2 clinical trials.
- Aims to restore progranulin protein levels in the brain through gene transfer.
- Utilizes an adeno-associated viral (AAV) vector for efficient gene delivery to CNS cells.
- Targets specific genetic mutations responsible for causing neurodegenerative diseases.