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Allergan Licensing Crispr for Inherited Eye Disease

Editing DNA

(LaCasadeGoethe, Pixabay)

7 August 2018. Drug maker Allergan is acquiring the rights from Editas Medicine to develop an application of the genome-editing technology Crispr for the inherited eye disease Leber congenital amaurosis type 10, or LCA10. In March 2017, Allergan, headquartered in Dublin, Ireland, received access in an option deal to learn more about the Editas Medicine technology for LCA10. In the new agreement, Allergan is exercising that option to develop a Crispr-based therapy, which could bring Editas another $40 million.

Editas Medicine Inc. was founded in 2013 in Cambridge, Massachusetts by geneticist Feng Zhang and colleagues from Broad Institute, a medical research center affiliated with Harvard University and MIT, to commercialize research from their labs on genome editing known as Crispr, short for clustered regularly interspaced short palindromic repeats. Crispr is based on bacterial defense mechanisms that use ribonucleic acid or RNA to identify and monitor precise locations in DNA. The actual editing is done by enzymes such as Cas9 or Cpf1.

Editas Medicine’s lead program is a Crispr-based treatment program for LCA-10 code-named EDIT-101. Leber congenital amaurosis is an inherited disorder that results from mutations in at least 18 genes. The disease affects infants, occurring in 2 to 3 in 100,000 newborns, but is still considered one of the more common causes of blindness in children. Leber congenital amaurosis primarily affects the retina. causing severe visual impairment beginning in infancy. LCA10, a variation of the disease, is traced to a mutation in CEP290 gene, accounting for 20 to 30 percent of individuals with Leber congenital amaurosis.

In the new agreement, Allergan will develop and commercialize EDIT-101 worldwide, while Editas will co-develop the treatment in the U.S., sharing those costs and profits equally. Allergan is paying Editas an option fee of $15 million, with Editas eligible for an additional $25 million if an investigational new drug application for EDIT-101 — in effect, permission to conduct clinical trials — is accepted by FDA.

As reported by Science & Enterprise in March 2017, Editas Medicine can still choose to co-develop in the U.S. one more treatment program licensed by Allergan. The original agreement also gave Editas an initial fee of $90 million.

“Allergan is a long-time innovator in ophthalmology,” says Katrine Bosley, Editas’s CEO in a joint statement, “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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