AVROBIO公司利用基因疗法治疗戈谢病

2017-03-22

临床前验证概念已实现,支持IND的活动正在进行中

美国马萨诸塞州剑桥市,2017年3月22日 - AVROBIO公司是一家临床阶段生物技术公司,开发用于治疗罕见疾病的转基因,改变生命的基因治疗药物,今天宣布将其管道扩大为戈谢病。这个后期临床前计划成为AVROBIO公司第一阶段法布里项目之后的第二个溶酶体贮积症(LSDs)治疗药物。瑞士隆德大学的Stefan Karlsson博士批准了戈谢病的方案,该方案已经证明了临床前概念验证。许可协议的条款没有披露。


AVROBIO公司总裁兼首席执行官Geoff MacKay表示:“针对LSD的基因疗法加上具有令人信服的临床前验证性证明,对于AVROBIO来说是一个极好的策略。 “我们天才的退伍军人团队使我们能够加速这个项目的发展,最终的目标是让全世界的戈谢病患者受益。目前正在进行促进IND的活动,准备进行1/2期临床研究。我们很高兴将这个计划加入到我们的产品组合中,因为我们将继续专注于建立我们的基因疗法来治疗罕见疾病。“


戈谢病是一种遗传疾病,其中称为葡糖神经酰胺的脂肪物质积聚在某些组织和器官中。这种疾病是由葡萄糖脑苷脂酶遗传性缺陷引起的,通常影响脾,肝,肺和骨骼。戈谢病是最常见的LSD之一,在总人口中多达1万人中有1万人活产。


AVROBIO研究基因疗法治疗戈谢病的目的是为患者提供持久而有意义的益处。患者的血液干细胞通过添加错误基因的全新功能拷贝而被提取并进行基因修饰。然后经修饰的细胞通过一次性输注返回到患者体内。预期内源性酶持续升高,有可能显着改善患者结果,并且消除负担酶替代疗法的沉重和昂贵的终生双周静脉内输注。


瑞典隆德大学分子医学和基因治疗学教授Stefan Karlsson博士授予Gaucher项目许可证,并将继续与其合作。 Karlsson博士因其对基因疗法和造血干细胞生物学的重大贡献而被广泛认可。 Karlsson博士曾担任1988-1996年美国国立卫生研究院分子和医学遗传学科主任,国际实验血液学会主席。 Karlsson博士经常在戈谢病会议上发言。


关于AVROBIO,Inc.

AVROBIO公司是慢病毒基因疗法领域的领导者,是一家临床阶段的公司,正在开发可能以单一剂量改变患者生命的破坏性治疗。公司重点发展法布里病和急性髓系白血病(AML)一期项目,同时积极构建治疗戈谢病等罕见病的治疗方案。 AVROBIO总部位于马萨诸塞州剑桥市,并在多伦多设有办事处。有关更多信息,请发送电子邮件至info@avrobio.com。


媒体查询:

Kirsten Dupuis

AVROBIO,Inc.

+1.617.914.8403

kirsten.dupuis@avrobio.com


原文:

Preclinical Proof-of-Concept Achieved, IND-Enabling Activities Underway

Cambridge, MA, March 22, 2017 – AVROBIO, Inc., a clinical-stage biotechnology company developing transformative, life-changing gene therapies for rare diseases, today announced the expansion of its pipeline to Gaucher disease. This late-stage preclinical program becomes AVROBIO’s second therapy for lysosomal storage disorders (LSDs), following on the heels of the Company’s Phase 1 Fabry program. The program for Gaucher disease was licensed from Dr. Stefan Karlsson at Lund University in Sweden, where preclinical proof-of-concept has been demonstrated. Terms of the license agreement were not disclosed.

“The addition of a gene therapy targeting an LSD with compelling preclinical proof-of-concept is an excellent strategic fit for AVROBIO,” said Geoff MacKay, AVROBIO’s President and Chief Executive Officer. “Our talented team of industry veterans enables us to accelerate the development of this program with the ultimate goal of benefiting Gaucher disease patients worldwide. IND-enabling activities are currently underway in preparation for a Phase 1/2 clinical study. We are excited to add this program to our portfolio as we continue to focus on building our pipeline of gene therapies to treat rare diseases.”

Gaucher disease is a genetic disorder in which a fatty substance called glucosylceramide accumulates in certain tissues and organs. The disorder is caused by a hereditary deficiency of the enzyme glucocerebrosidase, and typically impacts the spleen, liver, lungs and bones. Gaucher disease is one of the most common LSDs, occurring in up to 1 in 40,000 live births in the general population.

The aim of AVROBIO’s investigational gene therapy to treat Gaucher disease is to deliver lasting and meaningful benefits for patients. The patient’s blood stem cells are extracted and genetically modified by adding a new, fully functional copy of the faulty gene. The modified cells are then delivered back into the patient via a one-time infusion. A durable elevation of endogenous enzyme is expected, with the potential to significantly improve patient outcomes and eliminate burdensome and costly lifelong biweekly intravenous infusions of enzyme replacement therapy.

The Company licensed the Gaucher program from, and will continue to collaborate with, Stefan Karlsson, MD, PhD, Professor of Molecular Medicine and Gene Therapy at Lund University in Sweden. Dr. Karlsson is widely recognized for his significant contributions to gene therapy and hematopoietic stem cell biology. Dr. Karlsson previously served as Chief of the Molecular and Medical Genetics Section, NIH, NNDS from 1988-1996, and as President of The International Society for Experimental Hematology. Dr. Karlsson is a frequent speaker at meetings on Gaucher disease.

About AVROBIO, Inc.
AVROBIO, Inc., a leader in lentiviral-based gene therapies, is a clinical-stage company developing disruptive therapies that have the potential to transform patients’ lives with a single dose. The Company is focused on the development of its Phase 1 programs in Fabry disease and acute myeloid leukemia (AML), while actively building a pipeline of therapies to treat Gaucher disease and other rare diseases. AVROBIO is headquartered in Cambridge, MA and has offices in Toronto, ON. For additional information, email us at info@avrobio.com.

Media Inquiries:
Kirsten Dupuis
AVROBIO, Inc.
+1.617.914.8403
kirsten.dupuis@avrobio.com


翻译:文汐

原文链接:http://www.avrobio.com/hello-world-2/



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