Dose 1 of PBFT02 achieved supraphysiologic CSF progranulin levels in each of the first three treated patients at 30 days after treatmentElevated progranulin levels were sustained at up to six months post-treatmentDose 1 of PBFT02 was generally well-tolerated in patients who received an enhanced steroid regimen for immunosuppression Pipeline to focus on continued advancement of PBFT02 in FTD-GRN and explore PBFT02 in multiple additional adult neurodegenerative diseases Pursuing potential partnership opportunities for clinical-stage pediatric lysosomal storage disease programs including GM1 gangliosidosisManagement to host a webcast presentation to review interim FTD data today at 8:30 a.m.

治疗后30天，前三名接受治疗的患者中，剂量1的PBFT02均达到超生理CSF前颗粒蛋白水平。治疗后长达六个月，前颗粒蛋白水平持续升高。对于接受增强类固醇免疫抑制方案的患者，剂量1的PBFT02通常耐受性良好，以关注PBFT02在FTD-GRN中的持续进展并探索PBFT02在多种其他成人神经退行性疾病中的应用，寻求临床阶段小儿溶酶体贮积病项目（包括GM1神经节苷脂管理）的潜在合作机会，以在今天上午8:30主持网络广播演讲，回顾中期FTD数据。

ET PHILADELPHIA, Dec. 20, 2023 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic medicines company focused on improving the lives of patients with neurodegenerative diseases, today announced initial safety and biomarker data from three Cohort 1 patients in the ongoing 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.

ET PHILADELPHIA，2023年12月20日（global NEWSWIRE）--Passage Bio，Inc.（纳斯达克：PASG），一家专注于改善神经退行性疾病患者生活的临床阶段遗传药物公司，今天宣布了正在进行的全球1/2期UPLATION-D临床试验评估PBFT02的三名队列1患者的初始安全性和生物标志物数据，腺相关病毒（AAV）递送基因疗法，用于治疗具有颗粒蛋白（GRN）突变的额颞叶痴呆（FTD）患者。

FTD is a form of early onset dementia with no approved disease-modifying therapies. Additionally, the company shared updated strategic priorities aimed at further optimizing its portfolio for the treatment of neurodegenerative conditions. 'We are proud to announce initial clinical data from our upliFT-D clinical trial, which showcases the ability of PBFT02 to elevate CSF progranulin to supraphysiologic levels at the lowest tested dose, Dose 1, up to six months post-treatment.

FTD是一种早发性痴呆，没有批准的疾病缓解疗法。此外，该公司分享了最新的战略重点，旨在进一步优化其治疗神经退行性疾病的投资组合。”我们很自豪地宣布了我们的UPLATION-D临床试验的初步临床数据，该试验显示了PBFT02在治疗后6个月内以最低测试剂量（剂量1）将CSF颗粒蛋白前体提高至超生理水平的能力。

We believe these data, surpassing our expectations based on preclinical non-human primate models, validate the .

我们相信这些数据超过了我们基于临床前非人灵长类动物模型的预期，验证了。