{"id":389204,"date":"2023-12-21T04:12:13","date_gmt":"2023-12-21T09:12:13","guid":{"rendered":"https:\/\/platohealth.ai\/passage-bio-reports-preliminary-data-from-ftd-grn-gene-therapy-trial\/"},"modified":"2023-12-21T06:53:47","modified_gmt":"2023-12-21T11:53:47","slug":"passage-bio-reports-preliminary-data-from-ftd-grn-gene-therapy-trial","status":"publish","type":"post","link":"https:\/\/platohealth.ai\/passage-bio-reports-preliminary-data-from-ftd-grn-gene-therapy-trial\/","title":{"rendered":"Passage Bio reports preliminary data from FTD-GRN gene therapy trial\u00a0","gt_translate_keys":[{"key":"rendered","format":"text"}]},"content":{"rendered":"

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The trial is analysing PBFT02 to potentially treat frontotemporal dementia with granulin mutations. Credit: Kateryna Kon \/ Shutterstock.<\/figcaption><\/figure>\n<\/p><\/div>\n
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Passage Bio<\/a> has reported initial data from its three Cohort 1 patients in the Phase I\/II upliFT-D clinical trial of adeno-associated virus (AAV)-delivery gene therapy, PBFT02, to treat frontotemporal dementia (FTD) with granulin (GRN) mutations.<\/p>\n

Using an AAV1 viral vector, PBFT02 offers a functional copy of the GRN gene in people having mutations in the gene encoding progranulin.<\/p>\n

The international, open-label, multi-centre, dose-escalation trial is designed to evaluate a single injection of PBFT02 administered into the cisterna magna in FTD-GRN patients aged 35 to 75 years.<\/p>\n

Two dose levels of the gene therapy will be analysed in the trial. <\/p>\n

Evaluating the tolerability and safety of PBFT02 is the trial\u2019s primary endpoint while disease biomarkers and clinical outcome measures will comprise secondary endpoints.<\/p>\n

As per the preliminary safety and biomarker data, three Cohort 1 subjects in the trial who received dose one of the gene therapy, had a significant increase in supraphysiologic cerebrospinal fluid (CSF) progranulin levels in these patients 30 days after treatment.<\/p>\n

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