美國FDA批准張鋒公司的基因編輯臨床試驗

轉化醫學網2018-12-03 09:26:14

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(圖片來源:MIT官方網站)


2018年11月30日,張鋒創建的全球領先的基因組編輯公司Editas Medicine, Inc.(納斯達克股票代碼:EDIT)宣佈,美國食品藥品監督管理局(FDA)已接受該公司的EDIT-101的IND申請,允許其開展基因編輯臨床試驗,目標適應症為Leber先天性黑蒙10型(LCA10)眼病。


Editas Medicine總裁兼首席執行官Katrine Bosley表示:“FDA對EDIT-101 IND申請的接受是基因編輯領域的一個關鍵時刻,對患者來說是一個重要的里程碑,因為我們現在距離LCA10治療更近了一步。對我們來說,這是一個真正令人興奮的時刻!我們期待著作為一家臨床階段研發公司開啟我們的下一章,利用CRISPR技術的力量來改變世界各地嚴重疾病患者的生活。”


隨著IND的接受,Editas Medicine已經從合作伙伴Allergan獲得了2500萬美元的里程碑付款,作為兩家公司發現和開發針對嚴重威脅視力的疾病的實驗性眼科藥物的合作的一部分。 這是一項1/2期開放標籤、劑量遞增臨床試驗,預計招募10至20名患者,以評估安全性、耐受性和 EDIT-101的療效。




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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. 


來源:藥時代




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