NEXGEN-PD

帕金森氏病(PD)是一种主要的神经退行性疾病，目前还没有确定的治疗方法来改变疾病的病理，也没有早期诊断的手段。针对a Syn聚集体的疫苗是治疗PD疾病的一条有希望的途径，但当前一代的PD疫苗使用的是传统配方，其免疫原性有限，需要大量的佐剂才能达到效果。NexGen的专有WiSITE疫苗平台是新型葡萄糖结合疫苗(GNCV)中的第一个，这种疫苗在皮内注射并专门配制，以利用皮肤树突状细胞(DC)产生比传统疫苗更强大和更特异的免疫反应。这些更强的免疫反应可以大幅减少佐剂，同时提高治疗效果。NexGen将识别和表征针对Syn(PD-WISITs)的候选WiSIT构建体，并开发一种新的基于细胞外小泡(EV)的生物标记分析，该方法能够使用液体活检早期诊断PD，适用于护理点使用。PD-WISITs的安全性和有效性将在临床前得到验证，然后转化为人类第一个I/Ib期临床试验，以及基于EV的新型生物标记分析。NexGen的结果将是廉价而有效的早期帕金森病疾病修正治疗的非凡成就，以及一种诊断和指导前驱/早期帕金森病治疗的新的生物标记物检测。此外，将在临床上展示GNCV技术，该技术有可能对治疗多种其他疾病产生变革，对数百万人的健康产生深远影响。

Parkinson’s Disease (PD) is a major neurodegenerative disorder with no established treatment modalities capable of modifying diseasepathology, and no means of early diagnosis. Vaccines targeting aSyn aggregates are a promising route to disease-modifying therapyfor PD, but the current generation of PD vaccines utilise conventional formulations, which are limited in their immunogenicity andrequire substantial quantities of adjuvant to achieve efficacy. NEXGEN’s proprietary WISIT vaccine platform is the first of the novelclass of gluconeoconjugate vaccines (GNCVs), which are administered intradermally and specifically formulated to leverage skindendritic cells (DCs) to generate substantially stronger and more specific immune responses than conventional vaccines. These strongerimmune responses allow substantial reduction in adjuvants, while simultaneously increasing therapy efficacy. NEXGEN will identify andcharacterise candidate WISIT constructs targeting aSyn (PD-WISITs) and develop a novel extracellular vesicle (EV)-based biomarkerassay that enables early diagnosis of PD using liquid biopsies, suitable for point of care use. Safety and efficacy of PD-WISITs will bedemonstrated preclinically, before being translated to first-in-human Phase I/Ib clinical trials, along with the novel EV-based biomarkerassay. The results of NEXGEN will be the extraordinary accomplishments of cheap and effective disease-modifying treatment of earlyPD and a novel biomarker assay to diagnose and guide prodromal/early PD treatment. Further still, GNCV technology will be clinicallydemonstrated, which hasthe potential to be transformative to the treatment of a wide range of additional diseases, resulting in far-reachingimpacts to the health of millions.