Oligonucleotide therapeutics to transform IPF treatment

寡核苷酸疗法改变 IPF 治疗

• 批准号: 10055833
• 金额: $ 72.8万
• 依托单位: SENISCA 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=10055833
• 关键词: Oligonucleotide; therapeutics; transform; IPF; treatment

Here we propose a highly innovative RNA medicine project that will leverage the previous work carried out by SENISCA on the identification of a new and druggable set of target genes to enable significant senescence reversal effect in primary human cell lines. This project is a step forward towards the most innovative and fruitful avenue for novel therapeutics for Idiopathic Pulmonary Fibrosis (IPF), capable, for the first time, of attenuating the disease mechanism and with the potential to reverse the disease rather than mitigating symptoms only.IPF is the most common and aggressive form of interstitial lung disease, which can't be cured, reversed or stopped and current treatment options offer only a palliative solution, as they can only mildly slow disease progression and only in certain patients. Data shows that both incidence and prevalence of IPF are rising sharply and are predicted to continue to do so, leading to a crisis for healthcare systems.A strong team and advisory board have been established to carry out this project, which will ultimately lead to clinical trials of a new drug to stop advancement of the disease or potentially even reverse the fibrotic deposition that occurs during its progression.The expected outcomes are novel oligonucleotide therapeutics capable of reversing senescence in IPF-specific tissues. These new drugs will drive extension of the business strategy and scientific achievement of SENISCA which will contribute to the future generation of revenues and operational growth and strengthen the competitive position of SENISCA as a UK-based RNA medicine biotech.We expect significant benefits for the healthcare system (decrease of annual NHS costs up to ˜£2.5m in 3 years post launch), the UK economy (a reduction of working days lost because of IPF by ˜0.1%, with overall saving of estimated £32m for the UK economy) and a significant improvement of care outcome that will enable better quality of life and care for patients with IPF.

在这里，我们提出了一个高度创新的RNA医学项目，该项目将利用SENISCA先前在鉴定一组新的可药用靶基因方面所做的工作，以在原代人类细胞系中实现显着的衰老逆转效应。该项目是向特发性肺纤维化（IPF）新疗法最具创新性和成果的途径迈出的一步，首次能够减弱疾病机制，并有可能逆转疾病，而不仅仅是减轻症状。IPF是间质性肺病中最常见和最具侵袭性的形式，无法治愈，目前的治疗方案只能提供一种姑息性的解决办法，因为它们只能轻度减缓疾病进展，而且仅适用于某些患者。数据显示，IPF的发病率和患病率都在急剧上升，预计将继续上升，导致医疗保健系统的危机。已经成立了一个强大的团队和顾问委员会来执行这个项目，这将最终导致一种新药的临床试验，以阻止疾病的进展，甚至可能逆转在其进展过程中发生的纤维化沉积。能够逆转IPF特异性组织衰老的新寡核苷酸治疗剂。这些新药将推动SENISCA的业务战略和科学成就的扩展，这将有助于未来的收入和运营增长，并加强SENISCA作为英国RNA药物生物技术公司的竞争地位。（推出后3年内，NHS年度成本减少高达250万英镑），英国经济（因指规数损失的工作日减少0.1%，估计为英国经济节省3200万英镑）显著改善治疗结果，使IPF患者的生活质量和治疗更好。