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Allergan, Editas Medicine to develop genomic medicines to treat eye diseases

Allergan and Editas Medicine, a leading genome editing company, have joined forces to develop and commercialize EDIT-101 globally for the treatment of Leber Congenital Amaurosis, or LCA, a group of inherited retinal degenerative disorders caused by mutations in at least 18 different genes.

LCA 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% of all LCA patients.

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a tool that can be programmed to target specific stretches of genetic code and edit DNA at precise locations in the human genome. The technology allows researchers to permanently modify genes.

"CRISPR-based medicines have the potential to be game-changers for patients with both genetically-defined and genetically-treatable diseases of the eye," Allergan chief research and development officer David Nicholson said, in a statement."The Allergan team is excited to work with colleagues at Editas Medicine to develop EDIT-101 and potentially deliver a transformative medicine for LCA10 patients."

"Today marks a significant milestone in our collaboration with Allergan and in our work to develop genomic medicines to treat eye diseases," Editas Medicine president and CEO Katrine Bosley said, in a statement. "Allergan is a long-time innovator in ophthalmology, and their deep experience in developing, manufacturing, and commercializing medicines globally will meaningfully advance the EDIT-101 program and maximize our ability to bring this transformative medicine to people living with LCA10."
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