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Product › Details

EDIT-101 CRISPR-based medicinal product

Period Status 2019-08-06 development pp existent
Organisation Organisation Editas Medicine Inc. (Nasdaq: EDIT)

Editas Medicine, Inc.. (9/26/17). "Press Release: Editas Medicine Receives EMA’s Orphan Medicinal Product Designation for EDIT-101 for the Treatment of LCA10". Cambridge, MA.

Editas Medicine, Inc. (NASDAQ:EDIT), a leading genome editing company, today announced the European Medicines Agency (EMA) granted Orphan Medicinal Product designation to EDIT-101, a pre-clinical, CRISPR-based medicinal product to treat Leber Congenital Amaurosis type 10 (LCA10). LCA10 is a monogenic disorder caused by mutations in the CEP290 gene. LCA is the most common cause of inherited childhood blindness, with an incidence of two to three per 100,000 live births worldwide, and LCA10 is the most common form, accounting for 20-30 percent of all LCA patients.

EMA grants Orphan Medicinal Product designation to support development of medicines to treat life-threatening or chronically debilitating diseases where no satisfactory method of diagnosis, prevention, or treatment exists and of low prevalence (no more than five in 10,000 individuals) in the European Union (EU).

“There are currently no approved treatments for patients suffering from LCA10, a serious eye disease that results in progressive vision loss beginning from infancy and eventual blindness,” said Dr. Gerry Cox, M.D., Ph.D., Chief Medical Officer, Editas Medicine. “This designation for EDIT-101 highlights the need for treatment options for LCA10 patients, and we are extremely pleased the EMA agrees.”

Editas Medicine plans to submit an Investigational New Drug (IND) application for EDIT-101 in mid-2018. In March, Editas Medicine and Allergan Pharmaceuticals International Limited (“Allergan”) entered a strategic research and development alliance under which Allergan received an exclusive option to license up to five of Editas Medicine’s genome-editing ocular programs, including Editas Medicine’s lead program for LCA10. The agreement covers a range of first-in-class ocular programs targeting serious diseases based on Editas Medicine’s CRISPR genome editing platform, including CRISPR/Cas9 and CRISPR/Cpf1.

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 year of life, resulting in significant vision loss and 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 Editas Medicine

Editas Medicine is a leading genome editing company dedicated to treating patients with genetically-defined diseases by correcting their disease-causing genes. The Company was founded by world leaders in genome editing, and its mission is to translate the promise of genome editing science into a broad class of transformative genomic medicines to benefit the greatest number of patients.

Forward-Looking Statements

This press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words ‘‘anticipate,’’ ‘‘believe,’’ ‘‘continue,’’ ‘‘could,’’ ‘‘estimate,’’ ‘‘expect,’’ ‘‘intend,’’ ‘‘may,’’ ‘‘plan,’’ ‘‘potential,’’ ‘‘predict,’’ ‘‘project,’’ ‘‘target,’’ ‘‘should,’’ ‘‘would,’’ and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the Company’s goal of submitting of an IND for the LCA10 program by mid-2018. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company’s product candidates; availability and timing of results from preclinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption “Risk Factors” included in the Company’s most recent Quarterly Report on Form 10-Q, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.


Cristi Barnett
(617) 401-0113

Mark Mullikin
(617) 401-9083

Record changed: 2020-07-26


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