Stanford University

TAIPEI, Taiwan, Sept. 30, 2023 /PRNewswire/ -- Foresee Pharmaceuticals (TPEx: 6576), ("Foresee") announced today that the company will present the clinical design of the FP-045 Phase 1/2 clinical Fanconi anemia clinical study (FuschiA study) currently being initiated, at the Fanconi Anemia Research Fund (FARF) 2023 Scientific Symposium taking place in Vancouver, BC, Canada from September 28-October 1, 2023. The oral presentation entitled: "A Clinical Trial Evaluating the Safety & Biological Activity of FP-045, an ALDH2 Activator, in Fanconi Anemia Patients" details the clinical design of the FuschiA Phase 1/2 study that will be conducted in collaboration with clinical experts in the area of Fanconi anemia (FA) from Stanford University, Memorial Sloan Kettering Cancer Center and the University of Minnesota. Dr. Yisheng Lee, Foresee's Chief Medical Officer presenting the study, stated: "The FuschiA study is a first-in-patient clinical trial to test the safety and tolerability of FP-045 and to obtain data on preliminary biological activity in FA patients. We are very hopeful in the therapeutic promise of ALDH2 activation for FA patients and are confident the information from this study will help guide future registrational efforts for FP-045 in this highly unmet patient population." "We are strong supporters of FARF's mission and are working closely with all stakeholders and patients in the FA community, as we are committed to bringing forward FP-045, a novel oral therapy which we are hopeful one day may become a valuable part of the FA patient treatment standard of care.", stated Dr. Ben Chien, Foresee's Chairman and CEO. Details of the oral presentation are as follows: Title: A Clinical Trial Evaluating the Safety & Biological Activity of FP-045, an ALDH2 Activator, in Fanconi Anemia Patients Date: Saturday, September 30, 2023 T ime: 11:10 - 11:25 PST Location: Sheraton Vancouver in Grand Ballroom AB The presentation slides will become available on the Foresee website after the presentation. More information about the FuschiA trial is available at: About FP-045 in Fanconi anemia FP-045 is a first-in-class, potent oral aldehyde dehydrogenase 2 (ALDH2) activator. A dominant-negative allele of ALDH2 in Fanconi anemia (FA) patients is associated with accelerated progression of bone marrow failure, suggesting ALDH2 activation might be a potential therapeutic approach in FA. Laboratory studies demonstrate that FP-045 decreases aldehyde-induced toxicity in lymphoblastoid cell lines from FA patients. Preclinical animal experiments also demonstrated that sustained activation of ALDH2 can prevent Hematopoietic stem cell (HSC) loss in FA, resulting in improved HSC numbers and functions. To allow for clinical testing in FA patients, single ascending dose (SAD), multiple ascending doses (MAD) 7 days studies, and a 28 days dosing study of FP-045 have been conducted in healthy human subjects that showed safety and tolerability of FP-045 following oral administration. Dose-proportionality and time-stability of the pharmacokinetics of FP-045 in humans were also established. Based on these favorable results from healthy subjects and further discussions with regulators, Foresee is initiating the FuschiA study, a first-in-patient Phase 1/2 clinical trial to test the safety and tolerability of FP-045 and to obtain data on preliminary biological activity in FA patients. About ALDH2 ALDH2 (Aldehyde Dehydrogenase 2) is a mitochondrial matrix enzyme and key regulator of mitochondrial quality control systems/health and regulator of reactive aldehydes/carbonyls, oxidative stress, inflammation, and fibrosis. Activation of ALDH2 is a compelling therapeutic strategy for improving mitochondrial quality and regulatory mechanisms for treating the treatment of rare/orphan diseases and severe diseases of aging. The key role of ALDH2 in disease is supported by strong genetic/GWAS evidence related to a dominant-negative ALDH2*2 polymorphism. About Foresee Pharmaceuticals Co., Ltd. Foresee is a Taiwan and US-based biopharmaceutical company listed on the Taipei Exchange (TPEx: 6576). Foresee's R&D efforts are focused in two key areas, namely its unique Stabilized Injectable Formulation (SIF) long-acting injectable technology with derived drug products targeting specialty markets and secondly, its transformative preclinical and clinical first-in-class NCE programs targeting rare and severe disease areas with high unmet needs. Foresee's product portfolio includes late and early-stage programs. CAMCEVI® 42 mg, for the treatment of advanced prostate cancer, is now approved in the U.S., Canada, EU and Taiwan and launched in the U.S. in April 2022. Additionally, U.S. and EU regulatory submissions are under preparation for CAMCEVI® 21 mg. The second indication of CAMCEVI® 42 mg – central precocious puberty (CPP), the Casppian phase 3 clinical study, has been initiated. FP-025 – a highly selective oral MMP-12 inhibitor targeting inflammatory and fibrotic diseases, a Phase 2 proof-of-concept study in allergic asthmatic patients. The study has been completed with positive outcomes, with future development in rare immune-fibrotic diseases. FP-045 – a highly selective oral small molecule allosteric activator of ALDH2, a mitochondrial enzyme, for which the FuschiA Phase 1b/2 Fanconi Anemia study is currently being initiated, and a P2 study in pulmonary hypertension-interstitial lung disease (PH-ILD) patients is in planning. SOURCE Foresee Pharmaceuticals Co., Ltd.

SALT LAKE CITY, Sept. 29, 2023 /PRNewswire/ -- ARUP Laboratories today announced the formation of the new ARUP Institute for Research and Innovation in Diagnostic and Precision Medicine™ (R&I Institute). The institute will improve the lives of patients by advancing groundbreaking diagnostic and prognostic technologies and innovative tests. "This institute will enable us to unite the exceptional expertise and operational capabilities at ARUP with industry partners to push the boundaries of diagnostic medicine," said Tracy George, MD, ARUP chief scientific officer (CSO) and president of ARUP's newly formed Innovation Business Unit, of which the R&I Institute is part. "We are at the cusp of remarkable, landscape-altering technologies, and by creating this institute, we are in a position to grasp that future and bring it to the present." George, who has been instrumental in the advancement of innovation at ARUP since joining the company in 2018, will steer the new institute's efforts with the goal of maximizing patient impact. During her time as the executive director of PharmaDx, George brought her expertise in research, pathology, and clinical trials work to that division, developing novel companion diagnostic tests such as AAV5 DetectCDx™ via industry partnerships. Now, with a broad vision as CSO and president of the Innovation Business Unit, her attention has turned to the R&I Institute. As one of the largest single-site reference laboratories in the nation, with a close affiliation with the University of Utah, ARUP has long engaged in research and innovation. Many ARUP-developed technologies and assays have revolutionized the diagnostic industry. Notable successes include the LightCycler, developed early in ARUP's history and the predecessor of the now-standard polymerase chain reaction technology; the world's first artificial intelligence (AI)-augmented ova and parasite assay, deployed in 2019; and AAV5 DetectCDx™, the first FDA-approved companion diagnostic immunoassay. The establishment of an institute dedicated to innovation signals a new era as ARUP moves to intensify its efforts to accelerate invention and discovery. "ARUP is known for its excellence in quality, across the board and across the entire range of diagnostics. Now, we're witnessing the conglomeration of the right experts, the right community, and the right operational capabilities to translate that excellence into groundbreaking innovation," said Robert S. Ohgami, MD, PhD, FCAP. "We've seen the need and the opportunity, and now we're here to fearlessly seek out the future of laboratory medicine." Ohgami, who has spent his career dedicated to translational research, will oversee the institute as vice president and chief medical director. He received his medical degree from Harvard Medical School before completing his residency in anatomic pathology, a clinical fellowship in hematopathology, and a postdoctoral fellowship in immunology and oncology research at Stanford University School of Medicine. While at Stanford, Ohgami made significant contributions to the development of next generation sequencing technologies, particularly targeted sequencing for myeloid neoplasms. At the University of California, San Francisco, Ohgami was central to operationalizing digital pathology in hematopathology and expanded novel applications of next generation sequencing to the diagnosis of lymphomas and leukemias. Ohgami joined ARUP in 2022 and has continued his work in research and new assay development. Joining the institute as its director is Erica Clyde, who started in laboratory medicine as a bench technologist more than 15 years ago and whose career has since expanded to include critical positions at ARUP. Recently, Clyde served as group manager in ARUP's Clinical Trials department, which experienced rapid growth to become a multimillion-dollar client portfolio under her leadership. Clyde also played an instrumental role in ARUP's research efforts during the COVID-19 pandemic, notably in the Utah Health and Economic Recovery Outreach (HERO) project that monitored the spread and impact of COVID-19 and mitigation efforts, and she helped to significantly increase ARUP's operating margin for COVID-19-related testing. "This will not be cookie-cutter research; instead, we are providing highly customized solutions to our industry partners," Clyde said. "The institute will serve as a channel not only to the expertise of our 100-plus medical directors, but also to our extensive experience in scaling and optimizing laboratory operations and translating discoveries from research environments into production." Clyde's in-depth understanding of the technical, regulatory, and operational aspects of laboratory research will be instrumental in driving the R&I Institute forward. Institute leadership will also include experts such as David P. Ng, MD, a hematopathologist who has developed transformational technology in flow cytometry and who will lead efforts in new applications of applied artificial intelligence (AI), machine learning, and digital pathology. Salika M. Shakir, PhD, D(ABMM), another institute leader, is medical director of diagnostic technology evaluations and has conducted more than 20 in vitro diagnostic studies on the implementation of novel molecular assays for pathogen detection. Shakir will continue to collaborate with industry partners to develop and validate new testing technology. "Innovation is essential to create the high-quality tests that physicians and patients rely on and to continue to support delivery of the best quality of care. Ultimately, our goal is to improve outcomes for the millions of patients we already serve each year," Ohgami said. For more information and to submit an inquiry, visit the R&I Institute website at . About ARUP Laboratories Founded in 1984, ARUP Laboratories is a leading national reference laboratory and a nonprofit enterprise of the University of Utah and its Department of Pathology. ARUP offers more than 3,000 tests and test combinations, ranging from routine screening tests to esoteric molecular and genetic assays. ARUP serves clients across the United States, including many of the nation's top university teaching hospitals and children's hospitals, as well as multihospital groups, major commercial laboratories, group purchasing organizations, military and other government facilities, and major clinics. In addition, ARUP is a worldwide leader in innovative laboratory research and development, led by the efforts of the ARUP Institute for Clinical and Experimental Pathology® and the ARUP Institute for Research and Innovation in Diagnostic and Precision Medicine™. ARUP is ISO 15189 CAP accredited. ARUP Media Contact Lisa Carricaburu, [email protected], 801-583-2787 ext. 3842 SOURCE ARUP Laboratories

▎药明康德内容团队编辑每年的10月是诺贝尔奖公布的时间，在这一年一度的科学盛典举行前，预测最终大奖花落谁家一直是领域内外的观众都乐此不疲的事情。不久后，我们将迎来2023年诺贝尔奖的揭晓。那么，哪些杰出科学家有望荣膺这一殊荣呢？图片来源：诺贝尔奖官网在今天的这篇文章中，药明康德内容团队将根据各类诺奖风向标奖项的最近评选结果、历年获奖的可能规律以及来自ChatGPT的预测结果，为大家介绍几位备受瞩目的“种子选手”，他们在生理学或医学奖、化学奖的角逐中拥有很高的竞争力。诺贝尔生理学或医学奖的获奖成果预测（排名不分先后）获奖成果预测1：光遗传学工具的开发（根据ChatGPT推荐预测）Karl Deisseroth教授图片来源：Stanford University官网Karl Deisseroth博士是斯坦福大学生物工程、精神病学和行为科学的D.H. Chen教授，也是霍华德休斯医学研究所（Howard Hughes Medical Institute，HHMI）的研究员。2004年，他在斯坦福大学接受博士后训练期间，与当时还是博士研究生的Edward Boyden教授共同开发出了现代光遗传学技术的雏形。Deisseroth教授解决了在神经元中稳定表达从绿藻中发现的光敏感通道-2（ChR2）蛋白的难题。该蛋白能在光的作用下，精确地控制离子的进出。Edward Boyden教授图片来源：MIT Media Lab官网Edward Boyden博士是麻省理工学院媒体实验室（MIT Media Lab）和麻省理工学院麦戈文研究所（MIT McGovern Institute）的生物工程和大脑与认知科学教授。2004年，他在光遗传工具的开发过程中找到了通过光线激活或抑制神经活动的方法，能够对光进行精准的控制。他与Deisseroth教授开发的光遗传学新工具使在多种不同的动物体内研究神经元如何控制行为成为了可能，加快了神经科学领域的发展。在过去的几年里，光遗传学技术曾先后斩获科学突破奖、加拿大盖尔德纳奖、京都奖、沃伦·阿尔珀特奖、以及拉斯克奖等重磅荣誉。获奖成果预测2：雷帕霉素（rapamycin）靶点mTOR的发现（根据历年获奖的可能规律预测）Michael Hall教授图片来源：University of Basel官网Michael Hall教授是瑞士巴塞尔大学生物中心的副主任。雷帕霉素是一种从土壤里的吸水链霉菌（Streptomyces hygroscopicus）中发现的小分子，不仅具有很强抗真菌效力，并具高度的免疫抑制和癌细胞生长抑制的活性，但其具体作用机制自发现以来并不清楚。Michael Hall教授发现了雷帕霉素的靶点mTOR（target of rapamycin），并揭示了其在细胞生长代谢调控中的关键作用。该靶点的发现带来了大量mTOR通路抑制剂，让我们有望治疗癌症、糖尿病、肥胖症、心脏病、神经退行性疾病和衰老相关疾病。Hall教授曾在2017年获得了拉斯克临床医学研究奖。获奖成果预测3：T细胞和B细胞的发现（根据近期诺奖风向标预测）Max D. Cooper教授图片来源：Emory University官网Max D. Cooper教授是佐治亚研究联盟杰出学者，他是埃默里大学医学院埃默里疫苗中心和艾滋病研究中心的成员。1965年-1966年间，他通过研究鸡的胸腺和法氏囊提出了适应性免疫的双通路模型，解决了经典的免疫反应单通路模型中存在的悖论，并在确定B细胞的起源中做出了关键贡献。Jacques Miller教授 图片来源：The Walter and Eliza Hall Institute of Medical Research官网Jacques Miller教授曾在沃尔特和伊丽莎·霍尔医学研究所（The Walter and Eliza Hall Institute of Medical Research）工作，现已退休。1960年代，Miller教授的实验室确定了B细胞和T细胞这两种类型的免疫细胞，以及它们在特定免疫反应中是如何协同工作的。Miller教授和Cooper教授的开创性工作为现代免疫学的发展奠定了坚实的基础。2019年，两位已是耄耋之年的科学家获得了拉斯克临床医学研究奖。诺贝尔化学奖的获奖成果预测（排名不分先后）获奖成果预测4：多孔材料的开发（根据ChatGPT推荐预测）Omar Yaghi教授图片来源：UC Berkeley官网Omar Yaghi教授是加州大学伯克利分校化学学院的教授，同时是伯克利全球科学研究所的创始所长。Yaghi教授在一种被称为金属有机骨架（MOF）的纳米级多孔材料的理论、设计、制备、应用等方面做出了一系列的开创性工作，并掀起了MOF的研究热潮。这类材料具有极大的表面积、孔隙率高、化学稳定性强，因而具有极强的吸附能力。孔道结构及大小可根据需要进行调节，可选择性吸附特定的分子，在气体储存及分离、清洁水的生产及输送中具有重要的应用价值。Edith Flanigen女士 图片来源：National Science and Technology Medals Foundation官网Edith Flanigen女士曾是美国UOP工艺技术国际公司的研究化学家，致力于分子筛的开发，并于1994年退休。她和她领导的团队在天然沸石的基础上，发明了200多种不同的合成沸石，用于不同颗粒的筛选。自此，沸石的应用被大大拓宽，可用于石油提炼，取代洗衣粉中对环境有害的磷酸盐，甚至还可用于帮助过滤核电站周围水中的放射性物质。2012年，她获得了美国国家技术奖。获奖成果预测5：蛋白质折叠机制的发现（根据历年获奖的可能规律预测）Franz-Ulrich Hartl教授图片来源：Max Planck Institute of Biochemistry官网Franz-Ulrich Hartl教授是马克斯·普朗克生物化学研究所的细胞生物化学主任。蛋白质的折叠过程一直被认为是自发进行，直到20世纪80年代末，Hartl教授与耶鲁大学的Arthur L Horwich教授的合作首次证明了细胞内的蛋白质折叠需要其它蛋白（如伴侣蛋白）的协助，而伴侣蛋白在蛋白质折叠方面可能有重要作用。1992年，Hartl教授建立了重要的伴侣蛋白接力概念，这意味着不同的伴侣蛋白在蛋白质折叠过程的各个阶段中担负着不同的功能。Arthur L Horwich教授图片来源：Yale School of Medicine官网Arthur L Horwich教授是耶鲁大学医学院的遗传学兼儿科教授，他的工作最初涉及将蛋白质导入线粒体，并因此发现了线粒体内的一种伴侣蛋白——热休克蛋白60（Hsp60）。此后，Horwich教授利用遗传学、生物化学和生物物理工具成功解析出了伴侣蛋白的作用机制。Horwich教授近年来的研究方向是肌萎缩性侧索硬化（ALS）中蛋白折叠异常的影响。获奖成果预测6：揭示RNA和蛋白质的功能和病理性功能障碍，并创造了以新的方式利用这些生物聚合物的能力来改善人类疾病的策略（根据近期诺奖风向标预测）何川教授图片来源：Wolf Foundation官网何川教授是芝加哥大学的教授以及HHMI的研究员。何川教授是表观转录组学领域的权威学者，因发现了可逆的RNA甲基化及其在基因表达调控中的作用而获得2023年的沃尔夫化学奖（素有“诺奖风向标”之称）。他的实验室发现了第一个RNA去甲基化酶，这是一种从N6-甲基腺苷中去除甲基的酶（腺苷N6-甲基化是真核生物中最普遍的mRNA修饰）。Hiroaki Suga教授图片来源：Wolf Foundation官网Hiroaki Suga博士是日本东京大学化学系的教授，并是现任日本化学会会长。Suga教授因“开发基于RNA的催化剂，彻底改变了生物活性肽的发现”而获得了2023年的沃尔夫化学奖。他发明了一种基于RNA的催化剂flexizyme，它超越了自然机制，极大地拓展了可与核糖体机制结合的氨基酸范围。Suga教授的策略能够快速构建和筛选巨大的环状肽库，他的独特发现为药物化学建立了一种新的方法，并为药物发现创造了新的工具。Jeffery W Kelly教授图片来源：Wolf Foundation官网Jeffery W Kelly博士是斯克里普斯研究所（Scripps Research Institute）的化学教授。蛋白质稳态失调与一系列人类疾病有关。Kelly教授的开创性贡献揭示了分子水平上蛋白质稳态的基本特征，包括蛋白质折叠、错误折叠和聚集之间的相互作用。Kelly教授的实验室利用这些见解开发了药物Vyndaqel（通用名tafamidis），用于治疗因为野生型或遗传性转甲状腺素蛋白介导的淀粉样变性引起的心肌病（ATTR-CM）。2023年，他因“制定改善病理性蛋白质聚集的临床策略”同样获得了沃尔夫化学奖。除了以上提到的这些成果，还有哪些具有重大进展的研究领域是各位读者认为有望摘得诺奖桂冠的呢？欢迎大家在底部留言区给出预测（可回复文中的成果数字编号，或提名不在文章内的成果）~不过，无论最终得奖者是谁，这些勇敢的先行者和开创者们都为人类的进步和发展做出了不可磨灭的贡献，值得我们为他们鼓掌和点赞。让我们共同期待最终结果的揭晓！参考资料（可上下滑动查看）[1] Karl Deisseroth. Retrieved September 19, 2023, from https://profiles.stanford.edu/karl-deisseroth[2] Edward Boyden. Retrieved September 19, 2023, from https://www.media.mit.edu/people/esb/overview/[3] TOR signaling and the control of cell growth. Retrieved September 19, 2023, from https://www.biozentrum.unibas.ch/research/research-groups/research-groups-a-z/overview/unit/research-group-michael-n-hall[4] Australian immunology pioneer Jacques Miller wins Lasker Award. Retrieved September 19, 2023, from https://www.wehi.edu.au/news/australian-immunology-pioneer-jacques-miller-wins-lasker-award/[5] Max D. Cooper MD. Retrieved September 19, 2023, from https://winshipcancer.emory.edu/bios/faculty/cooper-max-d.html[6] Omar Yaghi. Retrieved September 19, 2023, from https://chemistry.berkeley.edu/faculty/chem/yaghi[7] Edith Flanigen. Retrieved September 19, 2023, from https://nationalmedals.org/laureate/edith-flanigen/[8] Prof. Dr. F.-Ulrich Hartl. Retrieved September 19, 2023, from https://www.biochem.mpg.de/ulrich_hartl_cv[9] Arthur Horwich, MD. Retrieved September 19, 2023, from https://medicine.yale.edu/profile/arthur-horwich/[10]Congratulations! 2023 Wolf Prize Laureates were announced. Retrieved September 19, 2023, from https://wolffund.org.il/2023/02/07/congratulations-2023-wolf-prize-laureates-were-announced/[11]Chuan He. Retrieved Feb 7, 2023 from https://wolffund.org.il/2023/02/07/chuan-he/[12]Jeffery W. Kelly. Retrieved September 19, 2023, from https://wolffund.org.il/2023/02/07/jeffery-w-kelly/[13]Hiroaki Suga. Retrieved September 19, 2023, from https://wolffund.org.il/2023/02/07/hiroaki-suga/本文由药明康德内容团队根据公开资料整理编辑，欢迎个人转发至朋友圈。转发授权请在「药明康德」微信公众号留言联系我们。其他合作需求，请联系wuxi_media@wuxiapptec.com。免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。