Intellegens: Developing a novel ML-driven tool to advance Oligonucleotide-therapeutic characterisation and manufacturing.

Intellegens：开发一种新颖的机器学习驱动工具来推进寡核苷酸治疗的表征和制造。

• 批准号: 10063439
• 金额: $ 184.8万
• 依托单位: INTELLEGENS LIMITED
• 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=10063439
• 关键词: Intellegens; Developing; novel; ML; driven

Intellegens Ltd is seeking to develop a novel ML-powered digital tool to provide organisations with a user interface for oligonucleotide characterisation and impurity prediction. Intellegens will collaborate with the Centre for Process Innovation (CPI) to build an extensive database of associated structures and intelligent look-up logic defined and coded into the software to create specific profiles for target molecules.Nucleic Acid Therapies (NATs) is a major emerging new class of medicines, offering enormous potential to treat a range of common and rare diseases with typically reduced timescales for development that can be quicker than other classes of drugs. In addition, NATs provide opportunities to tailor treatments to individual patients based on their genome sequence. However, the broader clinical application of NATs can be limited by current methods of manufacture and delivery.Oligonucleotides are segmented into several classes, including antisense, ribozymes, aptamers, miRNA, CPG/Immunostimulatory and RNAi. Many therapeutic areas and indications are currently targeted by oligo therapies from oncology (29%), Central Nervous System (CNS) disorders (12%) and genetic disorders (9%) to cardiovascular, respiratory, ophthalmology, gastrointestinal and infectious diseases. The global oligonucleotide development pipeline currently has 660 in preclinical, 70 phase-1, 94 phase-2, 19 phase-3 and 13 marketed, with a larger proportion targeting patient populations \>500k. 40% of approvals have occurred within 5-years (e.g. Inclisiran), providing a positive signal/confidence that oligonucleotides are a valid therapeutic area \[NICE,2021\]. This is encouraging more investment at the discovery end.Impurity prediction tools designed for small molecules are not sophisticated enough to predict impurities for oligonucleotides. With improved mechanistic understanding and screening approaches, more potent oligo candidates with better safety profiles can be identified for treatment of an increasing range of diseases and patient populations. The recent surge in approved oligonucleotide therapeutics indicates imminent potential as oligos are of intermediate size with much-improved selectivity towards the target and fewer off-target effects than small molecules \[Thakur,2022\].Intellegens addresses the unmet market need for a digital tool capable of impurity prediction and characterisation of the large and complex nature of oligonucleotides. Subsequently, reducing development costs and facilitating manufacturing scale-up.

Intellegens Ltd 正在寻求开发一种新型机器学习驱动的数字工具，为组织提供用于寡核苷酸表征和杂质预测的用户界面。 Intellegens 将与流程创新中心 (CPI) 合作，建立一个广泛的数据库，其中包含相关结构和智能查找逻辑，这些结构和智能查找逻辑已定义并编码到软件中，以创建目标分子的特定配置文件。核酸疗法 (NAT) 是一种重要的新兴新型药物，具有治疗一系列常见和罕见疾病的巨大潜力，并且通常可以缩短开发时间，比其他药物更快。 其他类别的药物。此外，NAT 还提供了根据个体患者的基因组序列定制治疗方案的机会。然而，NAT 更广泛的临床应用可能受到当前制造和递送方法的限制。寡核苷酸分为几类，包括反义、核酶、适体、miRNA、CPG/免疫刺激和 RNAi。目前，寡核苷酸疗法针对许多治疗领域和适应症，从肿瘤学（29%）、中枢神经系统（CNS）疾病（12%）和遗传性疾病（9%）到心血管、呼吸、眼科、胃肠道和传染病。全球寡核苷酸开发管线目前有660个处于​​临床前阶段、70个一期、94个二期、19个三期和13个已上市，其中较大比例针对患者群体>50万。 40% 的批准发生在 5 年内（例如 Inclisiran），这提供了寡核苷酸是有效治疗领域的积极信号/信心\[NICE，2021\]。这鼓励在发现端进行更多投资。专为小分子设计的杂质预测工具不够复杂，无法预测寡核苷酸的杂质。随着对机制的理解和筛选方法的改进，可以确定具有更好安全性的更有效的寡核苷酸候选药物，用于治疗越来越多的疾病和患者群体。最近批准的寡核苷酸治疗药物的激增表明了迫在眉睫的潜力，因为寡核苷酸具有中等大小，与小分子相比，对靶点的选择性大大提高，脱靶效应更少[Thakur，2022]。Intellegens 解决了市场对能够进行杂质预测和表征寡核苷酸的大而复杂性质的数字工具的未满足的需求。随后，降低开发成本并促进制造规模扩大。