SynaptixBio: Development of antisense oligonucleotide therapeutics for the treatment of rare paediatric TUBB4A-related leukodystrophies

SynaptixBio：开发反义寡核苷酸疗法，用于治疗罕见的儿科 TUBB4A 相关脑白质营养不良

基本信息

批准号：
10067962
负责人：
金额：
$ 63.53万
依托单位：
SYNAPTIXBIO LTD
依托单位国家：
英国
项目类别：
Collaborative R&D
财政年份：
2023
资助国家：
英国
起止时间：
2023 至无数据
项目状态：
未结题

来源：
https://gtr.ukri.org/projects?ref=10067962
关键词：
SynaptixBio Development antisense oligonucleotide therapeutics

项目摘要

SynaptixBio Ltd is a UK-based rare disease biotech SME pioneering ground-breaking development of therapeutics for treatment of TUBB4A-related leukodystrophies - the diseases affect the central nervous system (CNS) in both adults (milder) and children (the most common and severe).Rare diseases affect less than 1:2,000 people and are often severely disabling, chronic, and life-threatening, with misdiagnosis commonly occurring. TUBB4A-related leukodystrophies are a group of newly described neurodegenerative diseases which are genetic, debilitating, and life-limiting orphan diseases. The incidence is estimated at 1:100,000 to 1:200,000 and makes up 9% of all leukodystrophies.At present, no curative treatment exists for TUBB4A-related leukodystrophies and there are no clinical trials currently ongoing or scheduled for TUBB4A-leukodystrophy patients across the world.In the absence of disease-modifying treatments, SynaptixBio will develop and validate a life-changing treatment for TUBB4A-related leukodystrophy patients. SynaptixBio will achieve this by developing ASO therapeutics targeting the source of pathogenesis by altering TUBB4A mRNA and reducing protein expression in the cell. This project will build on the momentum that recent advances (12 ASO therapeutics have been approved to date) in the understanding of ASO pharmacology have provided for translating these therapeutics into the clinic with improved drug efficacy and safety.Once developed, SynaptixBio's treatment is expected to improve overall patient outcomes and standard-of-care, delivering transformative effects by opening new markets, generating revenues and investments which benefits the UK economy.

SynaptixBio LTD是一种基于英国的罕见疾病生物技术SME先验开发的治疗方法，用于治疗与TUBB4A相关的白细胞营养不良症治疗 - 疾病会影响中枢神经系统（CNS）（CNS）（CNS）（CNS）（MINDER）和儿童（最常见和疾病）（最常见的疾病），经常与1：2,000的人相比，疾病和疾病经常会受到疾病的影响。误诊通常发生。与TUBB4A相关的白细胞营养不良是一组新描述的神经退行性疾病，这些疾病是遗传，衰弱和生命的孤儿疾病。发病率估计为1：100,000至1：200,000，占所有白细胞营养不良的9％。目前，与TUBB4A相关的白细胞营养不良的治疗尚无治疗治疗与TUBB4A相关的白细胞营养不良患者的治疗。 SynaptixBio将通过开发针对发病源的ASO疗法来通过改变TUBB4A mRNA并减少细胞中的蛋白质表达来实现这一目标。该项目将基于对ASO药理学的理解的最新进展（12种ASO治疗学的批准）的势头，该项目提供了将这些治疗剂转化为诊所，并具有改善的药物疗效和安全性。一开始，SynaptixBio的治疗方法预计可以通过开放的整体效果来改善新市场，并改善了新的市场，并改善了新的市场，并改善了新市场，并改善了新市场，并改善了新的效果。