Editas Medicine公司宣布用于基因编辑的EDIT-101疗法进入临床试验！2018年11月30日，基因编辑公司Editas Medicine宣布，该公司开展使用CRISPR基因编辑技术治疗LCA10的临床试验获得FDA批准（Editas Medicine Announces FDA Acceptance of IND Application for EDIT-101）。该疗法可适用于遗传性视网膜衰退疾病Leber Congenital Amaurosis type 10（LCA10），该病症是由CEP290基因突变引起的单基因疾病。

EDIT-101是一个用于治疗LCA10疾病的基于基因编辑的实验药物，EDIT-101通过视网膜下注射给药，以将基因编辑系统直接输送到感光细胞中而达到治疗效果（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.）。

Editas Medicine 公司是在基因编辑领域处于领先的公司。Editas Medicine公司首席执行官Katrine Bosley说，FDA批准用于EDIT-101疗法的临床研究用的新药在基因编辑领域是一个重要时刻，重要的是对病人来讲是一个关键的里程碑，现在我们对于治疗LCA10更近了一步（“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,”）。

原文报道见：https://globenewswire.com/news-release/2018/11/30/1659958/0/en/Editas-Medicine-Announces-FDA-Acceptance-of-IND-Application-for-EDIT-101.html)。

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 Allergan received 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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