MARCKS

最新研究表明，蛋白质相分离（PPS）广泛存在于细胞中，并驱动着各种重要的生物学功能。在错误的地点或时间发生相分离，可能会造成与神经退行性疾病相关的分子堵塞或聚集，不良的细胞凝聚物也可能会导致癌症，并有助于解释衰老过程。鉴于人类疾病与蛋白质相分离过程的关联性，确定基于蛋白质相分离调控的潜在治疗靶点备受关注。近期，BioBAY园内企业英矽智能与剑桥大学在《美国国家科学院院刊》（PNAS）上联合发表了一篇论文，介绍了一种方法识别与蛋白质相分离相关的人类疾病靶点。值得一提的是，这是自 2021 年 9 月双方达成合作以来取得的一个重要研究里程碑。在这项研究中，研究人员将英矽智能自主研发的靶点识别引擎PandaOmics 与 FuzDrop 相结合，用于识别与蛋白质相分离易感疾病相关的蛋白。PandaOmics 是一种人工智能驱动的靶点发现工具，它整合了多种组学样本、文本数据以及人工智能生物信息学模型，以评估蛋白质作为治疗靶点的潜力。FuzDrop 是剑桥大学 Michele Vendruscolo 教授小组推出的一种开创性工具，它可以计算蛋白质自发现分离的倾向，从而帮助识别易形成液相分离凝聚物的蛋白质。利用这种方法，研究人员对人类样本数据进行了大规模的多组学研究，量化了蛋白质相分离在调控与疾病相关的各种病理过程中的影响，对具有较高 PandaOmics和FuzDrop评分的候选蛋白进行优先排序，并生成了与蛋白质相分离过程失调相关的潜在疾病靶点列表。此外，研究人员还在两个阿尔茨海默症细胞模型中验证了这种方法下预测的三个靶点（MARCKS、CAMKK2和p62）的不同相分离行为，这为这些预测靶点参与阿尔茨海默症提供了实验验证，并支持它们作为治疗靶点的进一步开发潜力。通过调节这些凝聚物的形成和行为，有望开发出新颖的干预措施，来减轻与阿尔茨海默症相关的病理过程。论文共同作者、英矽智能香港团队负责人潘颖博士表示，"我们很高兴与剑桥大学的合作取得了阶段性成果。这项研究旨在为靶向蛋白相分离易感疾病相关蛋白提供初步方向。随着研究蛋白质相分离过程的技术不断进步，以及有关其在细胞功能和功能障碍中作用的数据日益增多，我们将有机会进一步了解蛋白质相分离过程中的靶点与疾病发生的因果关系。我们期待着在不久的将来把这项临床前研究转化为新型治疗干预措施。”剑桥大学化学系错误折叠疾病中心副主任Michele Vendruscolo教授表示, “迄今为止，了解蛋白质相分离在细胞功能中的作用一直是一项挑战。更困难的是弄清它与人类疾病的确切相关性。通过与 英矽智能合作，我们开发出了一种多组学方法来系统地解决这一问题，并确定多种可能的治疗干预靶点。为研究人员探索这一复杂领域提供了指导工具。”▌文章来源：英矽智能责编：赵家帅审核：任旭推荐阅读企业资讯丨沙砾生物荣获沙利文「2023中国细胞药物创新奖」企业资讯丨博腾生物：与邦耀生物达成战略合作，加速推进基因与细胞治疗药物的商业化企业资讯丨飞依诺：全球总部暨研发生产大楼顺利封顶

Insilico, a clinical-stage generative artificial intelligence (AI)-drive drug discovery company, says recent research demonstrates that protein phase separation (PPS) is widely present in cells and drives a range of important biological functions. PPS at the wrong place or time, it says, could create clogs or aggregates of molecules linked to neurodegenerative diseases, and poorly formed cellular condensates could contribute to cancers and may explain the ageing process. An emerging link between human disease and the PPS process has led scientists to look for ways to identify potential targets for therapeutic interventions based on PPS regulation. The university and Insilico have jointly published a paper in Proceedings of the National Academy of Sciences (PNAS) ​, presenting an approach to identify therapeutic targets for human diseases associated with PPS. It recognizes a significant research milestone in their collaboration launched in September 2021, they say. Protein separation prediction In this study, researchers combined Insilico's proprietary artificial intelligence (AI)-driven target identification engine PandaOmics with the FuzDrop method for predicting protein separation to identify PPS-prone disease-associated proteins. PandaOmics integrates multiple omics and text-based AI bioinformatics models to assess the potential of proteins as therapeutic targets. The FuzDrop is a pioneering tool introduced by Michele Vendruscolo’s group at the University of Cambridge, which calculates the propensity of a protein to undergo spontaneous phase separation, aiding in the identification of proteins prone to forming liquid-liquid phase-separated condensates. googletag.cmd.push(function () { googletag.display('text-ad1'); }); “It has been challenging so far to understand the role of protein phase separation in cellular functions,” said Vendruscolo, co-director, Centre for Misfolding Diseases, Yusuf Hamied department of chemistry, University of Cambridge and lead author on the paper. “Even more difficult has been to clarify the exact nature of its association with human disease. By working with Insilico Medicine, we have developed a multi-omic approach to systematically address this problem and identify a variety of possible targets for therapeutic intervention. We have thus provided a roadmap for researchers to navigate this complex terrain.” "The method described in the paper enables target discovery for a new class of diseases that have not been fully mechanistically understood yet. Until now, it has been challenging to identify actionable targets for drug discovery companies in this space -- this provides a new tool for finding them." Large scale multi-omic study ​ Using this approach, the researchers conducted a large-scale multi-omic study of human sample data, quantified the relative impact of PPS in regulating various pathological processes associated with human disease, prioritized candidates with high PandaOmics and FuzDrop scores, and generated a list of possible therapeutic targets for human diseases linked with PPS. Researchers validated the differential phase separation behaviors of three predicted Alzheimer’s disease targets (MARCKS, CAMKK2 and p62) in two cell models of Alzheimer’s disease, which provides experimental validation for the involvement of these predicted targets in Alzheimer's disease and support their potential as therapeutic targets. By modulating the formation and behavior of these condensates, it may be possible to develop novel interventions to mitigate the pathological processes associated with Alzheimer's disease. “We are pleased to reach this milestone in our collaboration with the University of Cambridge,” said Frank Pun, head of Insilico Medicine Hong Kong, and co-author of the paper. “The study is intended to provide initial directions for targeting PPS-prone disease-associated proteins. With ongoing technical advancements in studying the PPS process, coupled with growing data about its roles in both cellular function and dysfunction, it is now possible to comprehend the causal relationship between PPS targets and diseases. We anticipate facilitating the translation of this preclinical research into novel therapeutic interventions in the near future.”

The rise in the incidence of ARDS stimulates the research and development of the drug, as it is likely to provide an appropriate environment for newer products to be profitable. Companies across the globe have shifted their focus toward the treatment of ARDS. The increase in the incidence will potentiate the treatment market for ARDS. Treatment goals, such as "permissive atelectasis", "permissive hypercapnia", and even "permissive hypoxemia", have been introduced as targets of ventilatory support. Together with the increased knowledge and understanding of how mechanical ventilation can harm the lungs, a major effort has been put into preventing or shortening the use of mechanical ventilation as much as possible and using settings that are considered to be "lung-protective." Several other supportive care options are available, but they all do not withstand the current needs of patients for this indication. LAS VEGAS, March 30, 2023 /PRNewswire/ -- DelveInsight's ' Acute Respiratory Distress Syndrome Pipeline Insight – 2023 ' report provides comprehensive global coverage of available, marketed, and pipeline acute respiratory distress syndrome therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the acute respiratory distress syndrome pipeline domain. Key Takeaways from the Acute Respiratory Distress Syndrome Pipeline Report DelveInsight's acute respiratory distress syndrome pipeline report depicts a robust space with 100+ active players working to develop 105+ pipeline therapies for acute respiratory distress syndrome treatment. Key acute respiratory distress syndrome companies such as Biocon, NRx Pharmaceuticals, Humanigen, Mesoblast, Sage Therapeutics, Evgen Pharma, Dimerix Bioscience, Vanda Pharmaceuticals, EUSA Pharma, Veru Healthcare, Foresee Pharmaceuticals, BioMarck Pharmaceuticals, Boehringer Ingelheim, Biohaven Pharmaceuticals, Biophytis, NovImmune SA, Constant Therapeutics, Windtree Therapeutics, Acticor Biotech, Direct Biologics, F4 Pharma, Apeiron Biologics, Trevena, Synact Pharma, Pluristem Therapeutics, UCB Pharma, Chiesi Farmaceutici, and others are evaluating new acute respiratory distress syndrome drugs to improve the treatment landscape. Promising acute respiratory distress syndrome pipeline therapies in various stages of development include Itolizumab, ZYESAMI, Lenzilumab, Remestemcel-L, Brexanolone, SFX-01, DMX-200, Tradipitant, Siltuximab, VERU 111, BIO 11006, FP025, Alteplase, Sarconeos Zavegepant, EB 05, USB 002, Sinapultide, Glenzocimab, DB 001, FX06, APN01, TRV 027, AP1189, PLX PAD, Zilucoplan, CHF5633, and others. In March 2023, Tetra Bio-Pharma received up to $150,000 in funding from the National Research Council of Canada Industrial Research Assistance Program (NRC IRAP) to support a research and development project to enable the development of its ARDS-003 oral formulation. Specifically, the funds will be used to optimize the formulation and perform animal non-GLP Pharmacokinetic Studies. In February 2021, Avid Bioservices and Humanigen announced that they had entered into a manufacturing services agreement to expand production capacity for lenzilumab. Under the terms of this Current Good Manufacturing Practice (cGMP) agreement, Avid would initiate technical transfer and analytical validation activities for lenzilumab with the goal of delivering cGMP drug substance batches to support Humanigen's regulatory and potential commercial activities. Request a sample and discover the recent advances in ARDS drug treatment @ Acute Respiratory Distress Syndrome Pipeline Report The acute respiratory distress syndrome pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage acute respiratory distress syndrome drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the acute respiratory distress syndrome clinical trial landscape. Acute Respiratory Distress Syndrome Overview Acute respiratory distress syndrome (ARDS) is a rapidly progressing condition that primarily affects the critically ill. The most serious complication of ARDS is fluid leaking into the lungs, making breathing difficult or impossible. The ARDS causes are classified as direct or indirect lung injuries. Direct lung injuries include pneumonia, aspiration, trauma, and others. Examples of indirect lung injuries include pancreatic inflammation, severe infection (also known as sepsis), blood transfusions, burns, and pharmaceutical responses. ARDS symptoms include shortness of breath, coughing, and fever; in some cases, high heart rates and rapid breathing have also been observed. Patients with ARDS may experience chest pain, especially during inhalation, and some may develop blue nails and lips due to drastically reduced oxygen levels in the blood. Mechanical ventilation, stress ulcer and venous thromboembolism prevention, nutritional support, and underlying damage therapy are all part of the acute respiratory distress syndrome treatment. Find out more about drugs for ARDS @ New Acute Respiratory Distress Syndrome Drugs A snapshot of the Acute Respiratory Distress Syndrome Pipeline Drugs mentioned in the report: Learn more about the emerging acute respiratory distress syndrome pipeline therapies @ ARDS Clinical Trials Acute Respiratory Distress Syndrome Therapeutics Assessment The acute respiratory distress syndrome pipeline report proffers an integral view of acute respiratory distress syndrome emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Acute Respiratory Distress Syndrome Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Oral, Parenteral, Intravenous, Subcutaneous, Topical Therapeutics Assessment By Molecule Type: Monoclonal Antibody, Peptides, Polymer, Small molecule, Gene therapy Therapeutics Assessment By Mechanism of Action: Androgen receptor antagonists, Hedgehog protein modulators, Histone deacetylase inhibitors, NF E2 related factor 2 stimulants, Wnt signalling pathway modulators, Cell replacements, Toll-like receptor 4 antagonists, Fibrinolytic agents, Plasminogen activator stimulants, Plasminogen activators, Myristoylated alanine-rich C kinase substrate inhibitors, Cell membrane permeability enhancers, Sodium channel agonists Key Acute Respiratory Distress Syndrome Companies: Biocon, NRx Pharmaceuticals, Humanigen, Mesoblast, Sage Therapeutics, Evgen Pharma, Dimerix Bioscience, Vanda Pharmaceuticals, EUSA Pharma, Veru Healthcare, Foresee Pharmaceuticals, BioMarck Pharmaceuticals, Boehringer Ingelheim, Biohaven Pharmaceuticals, Biophytis, NovImmune SA, Constant Therapeutics, Windtree Therapeutics, Acticor Biotech, Direct Biologics, F4 Pharma, Apeiron Biologics, Trevena, Synact Pharma, Pluristem Therapeutics, UCB Pharma, Chiesi Farmaceutici, and others. Key Acute Respiratory Distress Syndrome Pipeline Therapies: Itolizumab, ZYESAMI, Lenzilumab, Remestemcel-L, Brexanolone, SFX-01, DMX-200, Tradipitant, Siltuximab, VERU 111, BIO 11006, FP025, Alteplase, Sarconeos Zavegepant, EB 05, USB 002, Sinapultide, Glenzocimab, DB 001, FX06, APN01, TRV 027, AP1189, PLX PAD, Zilucoplan, CHF5633, and others. Dive deep into rich insights for new drugs for ARDS treatment; visit @ Acute Respiratory Distress Syndrome Medications Table of Contents For further information on the acute respiratory distress syndrome pipeline therapeutics, reach out @ Acute Respiratory Distress Syndrome Drug Treatment Related Reports Acute Respiratory Distress Syndrome Market Acute Respiratory Distress Syndrome Market Insights, Epidemiology, and Market Forecast – 2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key acute respiratory distress syndrome companies, including Cellular Biomedicine Group, Baylx, Histocell, Cartesian Therapeutics, Cynata Therapeutics, Immunovative Therapies, Orbsen Therapeutics, Celularity, Bonus BioGroup, Diffusion Pharmaceuticals, among others. Respiratory Distress Syndrome Market Respiratory Distress Syndrome Market Insights, Epidemiology, and Market Forecast – 2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key respiratory distress syndrome companies, including Dimerix Bioscience, Vanda Pharmaceuticals, EUSA Pharma, Veru Inc., BioMarck Pharmaceuticals, CTI BioPharma, Athersys, Foresee Pharmaceuticals, Boehringer Ingelheim, among others. Chronic Obstructive Pulmonary Disease Market Chronic Obstructive Pulmonary Disease Market Insights, Epidemiology, and Market Forecast – 2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key chronic obstructive pulmonary disease companies, including Sanofi, Chiesi Farmaceutici S.p.A., United Therapeutics Corporation, Verona Pharma, among others. Asthma Pipeline Asthma Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, including clinical and non-clinical stage products, and the key asthma companies, including GSK, AstraZeneca, Teva Pharmaceuticals, among others. Severe Asthma Pipeline Severe Asthma Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, including clinical and non-clinical stage products, and the key severe asthma companies, including AstraZeneca, Novartis, Sanofi, among others. 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