PHILADELPHIA, PA — Passage Bio, Inc. (Nasdaq: PASG) recently shared its first-quarter financials and highlighted recent advancements in its clinical trials. Among the key updates, interim data from its upliFT-D trial for treating frontotemporal dementia with granulin mutations (FTD-GRN) showed promising results.
The trial’s preliminary findings indicated that the initial dose of their gene therapy, PBFT02, effectively increased cerebrospinal fluid (CSF) progranulin levels in patients six months post-treatment. This is significant because progranulin is crucial for normal brain function, and its deficiency is linked to neurodegeneration in FTD-GRN patients.
Will Chou, M.D., President and CEO of Passage Bio, expressed optimism about these developments. “The consistent progranulin response…gives us confidence in the impact our therapy may have on patients at the current dose,” he stated. This positive outcome signals a potential breakthrough in treating a form of dementia that currently lacks effective therapies.
The company also announced the completion of dosing for the first cohort in the upliFT-D trial and plans to release comprehensive safety and biomarker data in the second half of 2024. Additionally, Passage Bio is preparing to begin dosing in a second cohort and has initiated discussions with the FDA regarding treatment pathways for another variant of the disease, FTD-C9orf72, using PBFT02.
These developments come as good news not only for patients suffering from these debilitating conditions but also for the broader medical community seeking new treatments for genetic brain disorders. With a robust financial position, Passage Bio expects to continue its research and development efforts well into Q4 2025.
This progress spotlights the potential of gene therapy in addressing complex neurological diseases, offering hope where traditional pharmacological interventions have fallen short. As Passage Bio moves forward with its clinical trials, the medical community eagerly anticipates further results that could herald a new era in the treatment of frontotemporal dementia and related conditions.
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