aruvant 品牌介绍
品牌简介
Aruvant Sciences是Roivant家族公司的一部分，是一家临床阶段的生物制药公司，致力于开发和商业化革命性的 基因 治疗罕见疾病的疗法。我们拥有一支在基因治疗产品的开发、制造和商业化方面有着丰富经验的人才团队。我们有一个积极的研究项目，一个领先的候选产品正在开发中，用于 镰状细胞病患者 (SCD)。 Aruvant Sciences, a part of the Roivant family of companies, is a clinical-stage biopharmaceutical company committed to developing and commercializing transformative gene therapies for rare diseases. We have a talented team with extensive experience in the development, manufacturing and commercialization of gene therapy products. We have an active research program with a lead product candidate in development for individuals suffering from sickle cell disease (SCD). The company’s lead product candidate, ARU-1801, an investigational lentiviral gene therapy, is currently in a clinical trial as a potential one-time curative treatment for SCD. Preliminary clinical data from the ongoing Phase 1/2 study demonstrated durable efficacy in reducing the negative impacts of SCD. We are preparing for the launch of our pivotal clinical trial of ARU-1801 in severe SCD. Our second product, ARU-2801, is a one-time, adeno-associated virus (AAV) gene therapy designed to deliver potentially curative efficacy to patients with hypophosphatasia without the limitations of chronic administration. To support the development of our pipeline products and help build awareness of patient needs, we partner with patient advocacy groups. main products PIPELINE SICKLE CELL DISEASE CLINICAL TRIALS https://aruvant.com
该公司的主要候选产品ARU-1801是一种实验性慢病毒基因疗法，目前正在 的临床试验 中，作为一种潜在的一次性治疗SCD的药物。来自正在进行的1/2期研究的初步临床数据显示，在减少SCD的负面影响方面具有持久的疗效。我们正在准备启动ARU-1801在严重SCD中的关键临床试验。 我们的第二种产品，ARU-2801，是一种一次性的腺相关病毒(AAV)基因治疗，旨在为低磷酸钠患者提供潜在的疗效，而不受慢性给药的限制。为了支持我们 管道 产品的开发，并帮助建立对患者需求的意识，我们与患者倡导团体合作。
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