LA JOLLA, Calif.--(BUSINESS WIRE)--As people live longer than ever before, the incidence of aging-related, chronic diseases is on the rise, severely impacting quality of life and placing an unprecedented financial and care burden on overwhelmed families, government agencies and healthcare providers..

加利福尼亚州拉霍亚市——（商业新闻短讯）——随着人们的寿命比以往任何时候都长，与衰老有关的慢性疾病的发病率正在上升，严重影响了生活质量，给不堪重负的家庭、政府机构和医疗保健提供者带来了前所未有的财务和护理负担。。

To help address this growing challenge, scientists at Scripps Research and its nonprofit drug development arm, the Calibr-Skaggs Institute for Innovative Medicines, are advancing novel regenerative medicines capable of repairing tissues damaged by age-related disease. In contrast to cell therapies, these first-in-class small molecule drugs—several of which are already in preclinical and phase 1 clinical trials—act on endogenous stem cells in the body to control their fate.

为了应对这一日益严峻的挑战，斯克里普斯研究所（Scripps Research）及其非营利药物开发部门卡利布·斯卡格斯创新药物研究所（Calibr Skaggs Institute for Innovative Medicines）的科学家正在开发能够修复因年龄相关疾病受损组织的新型再生药物。与细胞疗法相反，这些一流的小分子药物-其中一些已经处于临床前和1期临床试验-作用于体内内源性干细胞以控制其命运。

They are being developed to treat osteoarthritis, inflammatory bowel disease, lung diseases, heart failure, macular degeneration and more..

它们正在被开发用于治疗骨关节炎、炎症性肠病、肺部疾病、心力衰竭、黄斑变性等。。

“Current therapies for these chronic conditions slow disease progression or treat the symptoms, but don’t fully stop or reverse the resulting damage,” says Peter Schultz, PhD, President and CEO of Scripps Research. “In contrast, the regenerative medicine pipeline at Scripps Research represents a new frontier in healthy aging, with disease-modifying therapies that repair damaged tissues and thereby restore function in patients.”.

Scripps Research总裁兼首席执行官彼得·舒尔茨（PeterSchultz）博士说：“目前针对这些慢性病的疗法可以减缓疾病进展或治疗症状，但不能完全阻止或逆转由此产生的损害。”。“相比之下，斯克里普斯研究所的再生医学管道代表了健康衰老的新前沿，疾病缓解疗法可以修复受损组织，从而恢复患者的功能。”。

Scripps Research and Calibr-Skaggs’ programs in development include:

Scripps Research和Calibr Skaggs正在开发的项目包括：

Lung: Restoring lung tissue to treat pulmonary disease - Respiratory disease is the third leading killer globally. The ability to repair damage to the lung by stimulating growth of stem cells in the lower airway, called type 2 alveolar epithelial cells (AEC2s), could reverse loss of lung function caused by lung disease.

肺：恢复肺组织以治疗肺部疾病-呼吸系统疾病是全球第三大杀手。通过刺激下呼吸道干细胞（称为2型肺泡上皮细胞（AEC2s））的生长来修复肺损伤的能力可以逆转由肺部疾病引起的肺功能丧失。

Scripps Research and Calibr-Skaggs have developed a once-weekly, inhaled drug, CMR316, that promotes AEC2 stem cell expansion and the formation of new lung cells responsible for gas exchange. CMR316 promotes lung repair in several animal models of lung damage, including emphysema, chronic obstructive pulmonary disease (COPD) and IPF.

Scripps Research和Calibr Skaggs开发了一种每周一次的吸入药物CMR316，可促进AEC2干细胞的扩增和负责气体交换的新肺细胞的形成。CMR316在几种肺损伤动物模型中促进肺修复，包括肺气肿，慢性阻塞性肺病（COPD）和IPF。

CMR316 is currently undergoing a phase 1 clinical trial in healthy volunteers and IPF patients..

。。

Heart: Regenerating cardiac tissue after a heart attack - Heart disease is the leading cause of death globally. Cardiomyocytes—the muscle cells of the heart—are unable to regenerate tissue after a heart attack, often resulting in progressive disease and scarring. Scripps Research and Calibr-Skaggs are developing a small molecule drug (CMV852) that activates a regenerative pathway through YES-associated protein 1 (YAP) to stimulate the growth of new heart cells.

心脏：心脏病发作后心脏组织再生-心脏病是全球死亡的主要原因。心肌细胞心脏病发作后，心脏的肌肉细胞无法再生组织，通常会导致进行性疾病和瘢痕形成。Scripps Research和Calibr Skaggs正在开发一种小分子药物（CMV852），该药物通过YES相关蛋白1（YAP）激活再生途径，刺激新心脏细胞的生长。

This drug is injected precisely at the site of the damage in the heart, and it has already shown the ability to induce new functional cardiac tissue and significantly improve heart function in mice and pigs after a heart attack. CMV852 is expected to initiate investigational new drug (IND)-enabling studies in 2025..

这种药物被精确地注射到心脏损伤部位，它已经显示出诱导新的功能性心脏组织的能力，并在心脏病发作后显着改善小鼠和猪的心脏功能。预计CMV852将于2025年启动研究性新药（IND）研究。。

Cartilage: Repairing cartilage to address osteoarthritis (OA) - OA, a disease involving degeneration of joint cartilage and chronic pain, affects more than 30 million individuals in the U.S. Calibr-Skaggs is developing the small molecule, KA34, to promote robust regeneration of lost cartilage in animal models of OA, resulting in new functional joint tissue and decreased pain.

软骨：修复软骨以解决骨关节炎（OA）-OA是一种涉及关节软骨退化和慢性疼痛的疾病，影响着美国3000多万人。Calibr Skaggs正在开发小分子KA34，以促进OA动物模型中丢失软骨的强力再生，从而产生新的功能性关节组织并减轻疼痛。

KA34 has undergone extensive preclinical testing and shown good safety in a phase 1b human trial. Calibr-Skaggs scientists are currently developing extended-release formulations of KA34 to enable the drug to stimulate cartilage growth over longer periods (3 to 6 months), which will reduce the frequency of injections and patient burden..

KA34经过了广泛的临床前测试，并在1b期人体试验中显示出良好的安全性。Calibr Skaggs科学家目前正在开发KA34的缓释制剂，以使该药物能够在更长的时间（3至6个月）内刺激软骨生长，从而减少注射频率和患者负担。。

Eye: Repairing the retina in macular degeneration - Age-related macular degeneration (AMD) is the most common form of vision loss associated with age, affecting nearly 13% of people 40 and older. AMD is associated with the loss of retinal pigment epithelial cells (RPEs) due to age and oxidative damage.

眼睛：修复黄斑变性中的视网膜-年龄相关性黄斑变性（AMD）是与年龄相关的最常见的视力丧失形式，影响40岁及以上人群中近13%的人。AMD与年龄和氧化损伤导致的视网膜色素上皮细胞（RPE）的丢失有关。

Scientists have identified several small molecule drugs that selectively expand RPEs to regenerate a functional RPE layer, which are being evaluated in laboratory and animal studies to determine which of several mechanisms would be most amenable as an AMD therapy. If successful, the envisioned therapy would be a one-time injection of a long-acting drug to replenish the RPE layer in people with early-stage AMD..

科学家已经确定了几种选择性扩增RPE以再生功能性RPE层的小分子药物，这些药物正在实验室和动物研究中进行评估，以确定哪种机制最适合作为AMD治疗。如果成功，设想的治疗方法将是一次性注射长效药物，以补充早期AMD患者的RPE层。。

Intestine: Restoring the intestinal barrier for inflammatory bowel disease (IBD) - IBD, including Crohn’s and ulcerative colitis, results from auto-inflammatory destruction of the gastrointestinal tract. Calibr-Skaggs has developed a long-acting, chemically modified GLP-2 mimetic that robustly promotes healing in the gut and restores the mucosal barrier function.

肠道：恢复炎症性肠病（IBD）的肠道屏障-IBD，包括克罗恩病和溃疡性结肠炎，是胃肠道自身炎症破坏的结果。Calibr Skaggs开发了一种长效，化学修饰的GLP-2模拟物，可强烈促进肠道愈合并恢复粘膜屏障功能。

This agent has undergone a successful phase 1 clinical trial in humans, demonstrating safety, a best-in-class half-life and upregulation of a key biomarker of intestinal repair. Calibr-Skaggs or its partner will be conducting a phase 2 study to evaluate the efficacy of the clinical candidate in patients with IBD and IBD-related disorders..

该药物已在人体中成功进行了1期临床试验，证明了安全性，最佳半衰期和肠道修复关键生物标志物的上调。Calibr Skaggs或其合作伙伴将进行一项2期研究，以评估临床候选人对IBD和IBD相关疾病患者的疗效。。

Michael Bollong, PhD, associate chemistry professor and the Early Career Endowed Roon Chair for Cardiovascular Research at Scripps Research, who led the discovery of several of the regenerative programs, along with Schultz, who is also a professor of Chemistry, notes that the Scripps Research/Calibr-Skaggs team’s approach focuses on the development of small molecule drugs designed to be more accessible and easily administered than stem cell therapies..

迈克尔·博隆博士（MichaelBollong）是斯克里普斯研究院（Scripps Research）的化学副教授，也是其早期职业生涯中的心血管研究主席，他与化学教授舒尔茨（Schultz）一起领导了几个再生项目的发现。他指出，斯克里普斯研究院/卡利布·斯卡格斯（Calibr Skaggs）团队的方法侧重于开发比干细胞疗法更容易获得和管理的小分子药物。。

With conventional cell-based therapies stalled by complexity, poor efficacy and high costs, these small molecules provide a path forward for more effective regenerative medicines. “Our ultimate goal,” says Bollong, “is to expand to other organs to repair disease-related injury or even to rejuvenate healthy tissues as we age.”.

由于传统的基于细胞的疗法因复杂性，疗效差和成本高而停滞不前，这些小分子为更有效的再生药物提供了前进的道路。博隆说：“我们的最终目标是扩大到其他器官，以修复与疾病相关的损伤，甚至随着年龄的增长使健康组织恢复活力。”。

Schultz adds, “If we are able to show these therapies work in a disease setting, the next question will be to ask if they reverse age-related damage in otherwise healthy aging adults.”

舒尔茨补充道：“如果我们能够证明这些疗法在疾病环境中起作用，那么下一个问题将是，它们是否可以逆转健康老年人与年龄相关的损害。”

For more information on Scripps Research’s work on aging-related diseases, please visit the Regenerative Medicine Resources Page.

有关斯克里普斯研究院（Scripps Research）在衰老相关疾病方面工作的更多信息，请访问再生医学资源页面。

About Scripps Research

关于Scripps Research

Scripps Research is an independent, nonprofit biomedical institute ranked one of the most influential in the world for its impact on innovation by Nature Index. We are advancing human health through profound discoveries that address pressing medical concerns around the globe. Our drug discovery and development division, Calibr-Skaggs, works hand-in-hand with scientists across disciplines to bring new medicines to patients as quickly and efficiently as possible, while teams at Scripps Research Translational Institute harness genomics, digital medicine and cutting-edge informatics to understand individual health and render more effective healthcare.

斯克里普斯研究院（Scripps Research）是一家独立的非营利生物医学研究所，因其对自然创新的影响指数而被评为世界上最具影响力的研究所之一。我们正在通过深刻的发现促进人类健康，这些发现解决了全球迫切的医学问题。我们的药物发现和开发部门Calibr Skaggs与跨学科的科学家携手合作，尽可能快速有效地为患者带来新药，而Scripps研究转化研究所的团队则利用基因组学、数字医学和前沿信息学来了解个人健康并提供更有效的医疗保健。

Scripps Research also trains the next generation of leading scientists at our Skaggs Graduate School, consistently named among the top 10 US programs for chemistry and biological sciences. Learn more at www.scripps.edu..

斯克里普斯研究院还在我们的斯卡格斯研究生院培养下一代顶尖科学家，该研究生院一直被评为美国化学和生物科学十大项目之一。请访问www.scripps.edu了解更多信息。。