美国FDA批准张锋公司的基因编辑临床试验

（图片来源：MIT官方网站）

2018年11月30日，张锋创建的全球领先的基因组编辑公司Editas Medicine, Inc.（纳斯达克股票代码：EDIT）宣布，美国食品药品监督管理局（FDA）已接受该公司的EDIT-101的IND申请，允许其开展基因编辑临床试验，目标适应症为Leber先天性黑蒙10型（LCA10）眼病。

Editas Medicine总裁兼首席执行官Katrine Bosley表示：“FDA对EDIT-101 IND申请的接受是基因编辑领域的一个关键时刻，对患者来说是一个重要的里程碑，因为我们现在距离LCA10治疗更近了一步。对我们来说，这是一个真正令人兴奋的时刻！我们期待着作为一家临床阶段研发公司开启我们的下一章，利用CRISPR技术的力量来改变世界各地严重疾病患者的生活。”

随着IND的接受，Editas Medicine已经从合作伙伴Allergan获得了2500万美元的里程碑付款，作为两家公司发现和开发针对严重威胁视力的疾病的实验性眼科药物的合作的一部分。 这是一项1/2期开放标签、剂量递增临床试验，预计招募10至20名患者，以评估安全性、耐受性和 EDIT-101的疗效。

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Editas Medicine Announces FDA Acceptance of IND Application for EDIT-101

EDIT-101 set to be the first in vivo CRISPR medicine administered to people anywhere in the world

Earns $25 million milestone for IND acceptance from partner Allergan CAMBRIDGE, Mass., Nov. 30, 2018 (GLOBE NEWSWIRE) --  Editas Medicine, Inc.(NASDAQ: EDIT), a leading genome editing company, today announced the U.S. Food and Drug Administration (FDA) has accepted the Company’s Investigational New Drug (IND) application for EDIT-101, an experimental CRISPR genome editing medicine being investigated for the treatment of Leber Congenital Amaurosis type 10 (LCA10).

“The FDA’s acceptance of our IND for EDIT-101 is a significant moment in the field of genome editing, and importantly, a critical milestone for patients, as we are now one step closer to a treatment for LCA10,” said Katrine Bosley, President and Chief Executive Officer, Editas Medicine. “This moment is a truly exciting one for us, and we look forward to embarking on our next chapter as a clinical stage company, harnessing the power of CRISPR technology to transform the lives of people with serious diseases around the world.”

With the IND acceptance, Editas Medicine has earned a $25 million milestone payment from Allergan as part of the alliance between the companies to discover and develop experimental ocular medicines targeting serious, vision-threatening diseases. Editas Medicine and its partner, Allergan Pharmaceuticals International Limited (Allergan), a wholly-owned subsidiary of Allergan plc, expect to enroll 10 to 20 patients in a Phase 1/2 open label, dose escalation study to evaluate the safety, tolerability, and efficacy of EDIT-101.

About EDIT-101
EDIT-101 is a CRISPR-based experimental medicine under investigation for the treatment of Leber Congenital Amaurosis type 10 (LCA10). EDIT-101 is administered via a subretinal injection to reach and deliver the gene editing machinery directly to photoreceptor cells.

About Leber Congenital Amaurosis
Leber Congenital Amaurosis, or LCA, is a group of inherited retinal degenerative disorders caused by mutations in at least 18 different genes. It is the most common cause of inherited childhood blindness, with an incidence of two to three per 100,000 live births worldwide. Symptoms of LCA appear within the first years of life, resulting in significant vision loss and potentially blindness. The most common form of the disease, LCA10, is a monogenic disorder caused by mutations in the CEP290 gene and is the cause of disease in approximately 20‑30 percent of all LCA patients.

About The Editas Medicine-Allergan Alliance
In March 2017, Editas Medicine and Allergan Pharmaceuticals International Limited(Allergan) entered a strategic alliance and option agreement under which Allerganreceived exclusive access and the option to license up to five of Editas Medicine’s genome editing programs for ocular diseases, including EDIT-101. Under the terms of the agreement, Allergan is responsible for development and commercialization of optioned products, subject to Editas Medicine’s option to co-develop and share equally in the profits and losses of two optioned products in the United States. In August 2018, Allergan exercised its option to develop and commercialize EDIT-101 globally for the treatment of LCA10. Additionally, Editas Medicine exercised its option to co-develop and share equally in the profits and losses from EDIT-101 in the United States. Editas Medicine is also eligible to receive development and commercial milestones, as well as royalty payments on a per-program basis. The agreement covers a range of first-in-class ocular programs targeting serious, vision-threatening diseases based on Editas Medicine’s unparalleled CRISPR genome editing platform, including CRISPR/Cas9 and CRISPR/Cpf1 (also known as Cas12a).

About Editas Medicine
As a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cpf1 (also known as Cas12a) genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases.

For the latest information and scientific presentations,

please visit www.editasmedicine.com.

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