Hepatic Fibrosis, Congenital

128th Annual Boston Marathon is Monday, April 15th NEW YORK, April 12, 2024 /PRNewswire/ -- The American Liver Foundation (ALF) is proud to once again be a member of the Boston Marathon Official Charity Program for the 2024 Boston Marathon®, which takes place on Monday, April 15, 2024. As the Boston Athletic Association's first official charity team, ALF has a long history in Boston. Since 1988, the American Liver Foundation Marathon Team has made a dramatic impact on the American Liver Foundation's mission. Continue Reading American Liver Foundation (ALF) runners participate in the 2024 Boston Marathon® to raise awareness of liver disease! Post this "ALF is grateful for our history with the Boston Athletic Association, our partnership has been longstanding, and we are so appreciative," said Lorraine Stiehl, Chief Executive Officer, American Liver Foundation. "Our team is made up of many runners who have been profoundly affected by liver disease. As a runner myself, I'm inspired by their stories and dedication to their loved ones, and ALF." One of those running this year is Laura Yardumian. "I'm running this year in honor of my maternal grandfather who died of liver cancer, for myself after surviving liver cancer, and for all those currently living with this challenging disease," said Laura. This will be Laura's first time running the Boston Marathon®. As she starts in Hopkinton, and treks Heartbreak Hill, she will be joined by two special members of the ALF team, her sons Lorenzo Perez and Varandt Khodaverdian. "It is not so much about the time it takes to finish the race; it is about getting to the finish line in good health, educating people about liver disease during this five-month journey, and raising funds to support a very worthy organization." The American Liver Foundation team is made up of novice to experienced runners, most of whom have a connection to liver disease. Some of the 50-member team include: Molly Dillion: Running her first marathon for her dad who received his liver transplant in 2022. Corey Fugere: Running his first Boston Marathon for his eldest son, who was diagnosed with primary sclerosing cholangitis in 2022. Michael Kim: Celebrating his 50th birthday and 50 years of living with liver disease by running again with ALF. His last Boston Marathon® was in 2014. Fred and Kristin Kostecki: Joining Team ALF for the third year running for their daughter, who was diagnosed with congenital hepatic fibrosis with portal hypertension at age four. Read about the Kostecki family here: Sophie Long: Running again with ALF for her living liver donation recipient, Caitlin. Watch the video about their story here: Felix Lung: Running his first marathon with ALF in memory of his dad, who died of liver cancer. Jessica Moran: Running her first marathon in honor of her dad, who died of liver cancer. Gordon Scott: Running in honor of his two daughters who both have liver disease. He will be joined by his son-in-law, Steven Balla. Sarah and Emily Tully, Sydney Hsu: Running in honor of Keith Tully, Sarah and Emily's dad and "Coach Keith" to Sydney. After living with primary sclerosing cholangitis Keith received a liver transplant in July 2023. Learn more about ALF's 2024 Boston Marathon® team and support their 26.2 mile run at . About the American Liver Foundation American Liver Foundation (ALF) is a national community of patients, caregivers and medical professionals dedicated to helping people improve their liver health. Providing guidance and life-saving resources, we are a beacon for the 100 million Americans affected by liver disease. We advocate for patients and families, fund medical research and educate the public about liver wellness and disease prevention. We bring people together through our educational programs and events and create a network of support that lasts a lifetime. ALF is the largest organization focused on all liver diseases and the trusted voice for patients and families living with liver disease. For more information visit or call: 1 800 GO LIVER (800-465-4837). Contact: Julie Kimbrough [email protected] Direct dial: 646-737-9409 Facebook Twitter LinkedIn YouTube Instagram SOURCE American Liver Foundation

2022年4月4日-- Sirnaomics Ltd.（“公司”，“Sirnaomics”，股份代号：2257.HK）是一家专注于探索及开发RNAi疗法行业领先的生物制药公司。Sirnaomics今天宣布，公司的小干扰核糖核酸（siRNA）候选药物STP707通过静脉给药用于原发硬化性胆管炎（PSC）治疗的一期临床试验在美国启动，并已为首例受试者进行系统给药。 此项单中心、随机、剂量递增、顺序队列研究旨在评估STP707的安全性、耐受性和药代药动，并在健康志愿者中以单次递增剂量静脉内给药。受试者分为四组，其中A组将通过静脉输注接受3mg剂量，B组将接受6mg，C组将接受12mg，D组将接受24mg。该研究招募了多达50名年龄在18至55岁之间的健康受试者。 Sirnaomics执行董事兼首席医疗官Michael Molyneaux博士说：“这项I期临床试验是我们治疗肝纤维化药物开发计划中非常重要的一步，它将使我们能够收集有关安全性和剂量的重要数据，这些数据将支持我们未来利用STP707治疗由于原发性硬化性胆管炎引起的肝纤维化的研究。这项研究将使我们能够扩大对这种治疗候选药物如何影响更多需要有效治疗的罕见疾病患者的了解。” Sirnaomics创始人、董事会主席、执行董事、总裁兼首席执行官陆阳博士表示：“评估STP707用于治疗原发硬化性胆管炎这一最为严重的肝纤维化类型研究，将为患有各种类型肝纤维化的庞大患者群体带来希望，且具有巨大的医疗需求。此次研究是Sirnaomics开发这种siRNA治疗产品用于慢性病治疗的静脉输注配方的关键里程碑。” --关于STP707 STP707由两个小干扰核酸（siRNAs）组成，分别靶向TGF-β1和COX-2 mRNA，并与专有的组氨酸-赖氨酸共聚肽（HKP+H）配制成纳米颗粒制剂作为给药载体。这一特殊载体与STP705产品中使用的载体不同。每个单独的siRNA都具备抑制相应靶点mRNA表达的能力，并可将这两个siRNA联合使用而产生协同效应。TGF-β1和COX-2的过度表达在肿瘤形成中起着关键的调节作用。在STP707的临床前研究中，静脉注射给药后可观察到包括肝脏和肺等器官中的TGF-β1和COX-2基因表达被敲低。此外，在临床前模型试验中，STP707显示出对多种实体肿瘤类型的抗肿瘤活性。 --关于Sirnaomics Sirnaomics是一家RNA疗法生物制药公司，公司候选产品处于临床前及临床阶段，专注于探索及开发创新药物，用于治疗存在医疗需求及庞大市场机会的适应症。Sirnaomics是在中国和美国均拥有重要市场地位的首家临床阶段RNA疗法生物制药公司，且是首家为核心产品STP705的RNAi疗法在肿瘤学领域取得积极IIa期临床结果的公司。 内容来源于网络，如有侵权，请联系删除，谢谢。

89bio公司日前宣布，其用于治疗非酒精性脂肪性肝炎（NASH）的在研疗法pegozafermin（原名BIO89-100），在一项1b/2a期概念验证临床试验中获得积极结果。在20名患者组成的患者队列中，63%的患者达到非酒精性脂肪性肝病活动评分（NAS）改善2分以上，并且肝纤维化没有恶化。在NASH症状清除和纤维化改善这两个支持注册的临床终点方面也观察到具有临床意义的改善。 Pegozafermin是一款潜在“best-in-class” 成纤维细胞生长因子21（FGF21）类似物。FGF21是一种内源性代谢激素，可调节能量消耗以及葡萄糖和脂质代谢。Pegozafermin利用该公司专有的糖基聚乙二醇化技术，可延长天然FGF21的半衰期和优化生物活性。目前，它在2b期临床试验中用于治疗NASH和严重高甘油三酯血症。 在这一单臂患者队列中，经组织活检确认的肝纤维化F2和F3期的NASH患者接受了为期20周的pegozafermin的治疗。基线时，65%的患者处于纤维化F3期。在20名注册患者中，19名患者治疗结束后进行组织活检，结果显示： 63%的患者达到NAS评分改善2分以上并且纤维化没有恶化，74%的患者达到NAS评分改善2分以上。 图片来源：89bio公司官网 此外，32%的患者达到NASH症状清除并且肝纤维化没有恶化的标准，26%的患者达到肝纤维化改善一级并且NASH症状未恶化的标准，47%的患者获得NASH症状清除或肝纤维化改善。 图片来源：89bio公司官网 此外，多项与肝纤维化、纤维化风险和NASH症状清除相关的关键性非侵入性检测获得具有临床意义的显著改善。在改善肝脏健康之外，该疗法还对血糖控制、血脂和体重产生显著积极影响。 图片来源：参考资料[2] “Pegozafermin的整体数据喜人，包括在组织学终点上具有临床意义的变化，在与总体肝脏健康相关的所有非侵入性评估上令人印象深刻的变化，以及在心血管标志物和血糖控制方面相对于基线的显著变化。”加州大学圣地亚哥分校非酒精性脂肪性肝病研究中心主任、该临床试验的主要研究者Rohit Loomba博士说，“NASH是一种复杂的疾病，在考虑我们患者的治疗选择时，解决整体肝脏健康问题以及治疗疾病的潜在驱动因素非常重要。” 参考资料： [1] Pegozafermin (BIO89-100) Phase 1b/2a NASH Histology Cohort Topline Results. Retrieved January 25, 2022, from https://ir.89bio.com/static-files/4046a1f3-c63d-40aa-ba18-af4f36711a9e[2] 89bio Reports Positive Topline Results from an Expansion Cohort of the Phase 1b/2a Trial of Pegozafermin (BIO89-100) for the Treatment of NASH. Retrieved January 25, 2022, from https://ir.89bio.com/news-releases/news-release-details/89bio-reports-positive-topline-results-expansion-cohort-phase 内容来源于网络，如有侵权，请联系删除，谢谢。