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公司正在寻求开发一种新颖的机器学习驱动的数字工具，为组织提供寡核苷酸表征和杂质预测的用户界面。Intellegens将与过程创新中心（CPI）合作，建立一个广泛的相关结构和智能查找逻辑数据库，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（NATs）是一种主要的新兴药物类别，具有治疗一系列常见和罕见疾病的巨大潜力，其开发时间通常比其他类别的药物更短，可以更快。此外，NATs提供了根据个体患者的基因组序列定制治疗的机会。然而，NATs的广泛临床应用可能受到当前制造和递送方法的限制。寡核苷酸分为反义、核酶、适体、miRNA、CPG/免疫刺激和RNAi等几类。目前许多治疗领域和适应症都是肿瘤学的寡核苷酸疗法的目标%), 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.