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NORTH BRUNSWICK, N.J.--(BUSINESS WIRE)--Couragene, a genetic medicines company using novel delivery platforms, announces today that its co-founders Yong-Hui Jiang and Jiangbing Zhou of Yale University, together with Elizabeth Berry-Kravis of RUSH University have been awarded a competitive UG3/UH3 grant from the NIH’s Somatic Cell Genome Editing (SCGE) program.
北布伦瑞克,新泽西州-(商业线)-Couragene,一家使用新型交付平台的遗传药物公司,今天宣布其联合创始人姜永辉和耶鲁大学周建国,以及拉什大学的Elizabeth Berry Kravis,已获得美国国立卫生研究院体细胞基因组编辑(SCGE)计划提供的竞争性UG3/UH3资助。
This monumental grant, totaling roughly $40 million contingent on milestones, marks a momentous step forward in leveraging genome editing to combat neurogenetic diseases..
这项巨大的赠款,总计约4000万美元,取决于里程碑,标志着利用基因组编辑对抗神经遗传疾病迈出了重要一步。。
Professor Jiang is a distinguished physician scientist, who made significant contributions to Angelman syndrome (AS) research including the discovery of the UBE3A gene and the creation of the first two AS mouse models. Professor Jiang is also a leading researcher of H1-4 syndrome.
江教授是一位杰出的医师科学家,他为Angelman综合征(AS)研究做出了重大贡献,包括发现UBE3A基因和创建前两个AS小鼠模型。江教授也是H1-4综合征的主要研究者。
Professor Zhou is a leading expert in delivery systems for genetic medicine. His team developed the platform technology STEP (Stimuli-responsive Traceless Engineering Platform), a first-in-class non-viral delivery technology that accomplishes the efficient intracellular delivery of gene editors throughout the central nervous system, which has been a limiting factor for gene editing applications in patients..
周教授是遗传医学传递系统的领先专家。他的团队开发了平台技术步骤(刺激响应无痕工程平台),这是一种一流的非病毒传递技术,可实现整个中枢神经系统中基因编辑器的高效细胞内传递,这一直是基因编辑的限制因素应用于患者。。
Leveraging the STEP technology, a team led by Professors Jiang and Zhou devised a precise genome editing approach directly addressing the disease-causing genetic defects. Couragene exclusively licensed these innovative technologies and is poised to make a profound and lasting impact on the future treatment of genetic disorders.
由江教授和周教授领导的团队利用STEP技术,设计了一种精确的基因组编辑方法,可直接解决引起疾病的遗传缺陷。Couragene独家许可了这些创新技术,并有望对遗传疾病的未来治疗产生深远而持久的影响。
The SCGE grant supports the clinical translation of STEP-based genome editing for AS and H1-4 syndrome..
SCGE资助支持AS和H1-4综合征的基于步骤的基因组编辑的临床翻译。。
“Current approaches for delivery of biologic payloads have many limitations. This grant enables us to further establish the STEP technology, a novel delivery approach with promise to overcome these challenges,” said Professor Zhou.
周教授说:“目前提供生物有效载荷的方法有很多局限性,这种资助使我们能够进一步建立STEP技术,这是一种有望克服这些挑战的新型交付方法。
“As a physician I specialize in the treatment of patients with genetic disorders. Today there are no effective treatments for AS or H1-4 syndrome, leaving affected families with few options,” said Professor Jiang. “This NIH-funded research is critical to advancing technologies that could one day lead to more effective therapeutic options, and quite possibly cures.”.
江教授说:“作为一名医生,我专门治疗遗传性疾病患者,目前尚无有效的As或H1-4综合征治疗方法,使受影响的家庭几乎没有选择。”。“这项由NIH资助的研究对于推进技术至关重要,这些技术有朝一日可以带来更有效的治疗选择,并且很可能治愈。”。
Couragene’s leadership congratulates its co-founders on the achievement. The development of effective treatments for AS and H1-4 syndrome using STEP will revolutionize the delivery of genetic medicines, giving hope to patients awaiting an effective therapy.
Couragene的领导层祝贺其联合创始人的成就。使用STEP开发有效治疗AS和H1-4综合征的方法将彻底改变遗传药物的输送,为等待有效治疗的患者带来希望。
“STEP is a versatile platform technology with the capacity to deliver various payloads. I anticipate groundbreaking therapies beyond the diseases mentioned, and I take great pride in Couragene's role in their development,” said Dr. Amy Liao, CEO of Couragene.
Couragene首席执行官Amy Liao博士说:“STEP是一种多功能平台技术,能够提供各种有效载荷。我预计超越上述疾病的开创性疗法,我对Couragene在其发展中的作用感到非常自豪。
About Couragene
关于香豆素
Couragene is a biotechnology company committed to building first-in-class in vivo genetic therapies using its proprietary delivery platforms. Couragene was co-founded in 2022 by biotech veteran and CEO Dr. Amy Liao, who previously co-founded and led GENEWIZ for over 20 years through its acquisition by Azenta Life Sciences..
Couragene是一家生物技术公司,致力于使用其专有的交付平台构建一流的体内遗传疗法。Couragene于2022年由生物技术退伍军人兼首席执行官Amy Liao博士共同创立,Amy Liao博士此前通过收购Azenta Life Sciences共同创立并领导GENEWIZ已有20多年。。
To learn more visit www.couragene.com.
要了解更多信息,请访问www.couragene.com。