Allergan plans to develop and commercialize Editas Medicine’s CRISPR genome editing treatment candidate EDIT-101 globally for Leber Congenital Amaurosis type 10 (LCA10), a development effort that could generate up to $40 million for Editas.

Editas has already received $15 million after a wholly-owned Allergan subsidiary, Allergan Pharmaceuticals, exercised its option to develop and commercialize EDIT-101—an option held under a strategic alliance and option agreement between the companies inked last year.

Editas is also eligible for an additional $25 million from Allergan upon FDA acceptance of an investigational new drug (IND) application for EDIT-101.

EDIT-101 is designed to eliminate a mutation in the CEP290 gene by using CRISPR to cut out that nucleotide and surrounding DNA, thus restoring normal protein expression and function of remaining photoreceptor cells. Editas reasons that therapeutic approaches aimed at restoring function of remaining photoreceptor cells could arrest further loss of vision for patients with LCA10, the most common cause of inherited childhood blindness, for which no treatment options now exist.

“CRISPR-based medicines have the potential to be game-changers for patients with both genetically-defined and genetically-treatable diseases of the eye,” David Nicholson, Ph.D., Allergan’s chief R&D officer, 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.”

EDIT-101 was one of up to five Editas early-stage CRISPR genome-editing programs targeting eye diseases for which Allergan held a licensing option when the companies launched their R&D partnership in March 2017, with Allergan paying Editas $90 million upfront.

Under that agreement, Allergan agreed to oversee development and commercialization of optioned products, subject to Editas’ option to co-develop and share equally in the profits and losses of two optioned products in the U.S.

Profit-Sharing Option

The companies said today that Editas has exercised its option to co-develop and share equally in U.S. profits and losses from EDIT-101.

Editas is also eligible for payments tied to achieving development and commercial milestones, as well as royalties on a per-program basis where the parties are not sharing profits and losses.

The companies’ agreement covers a variety of first-in-class ocular programs targeting serious, vision-threatening diseases based on Editas’ CRISPR genome editing platform, which uses a protein-RNA complex composed of either the protein Cas9 or Cpf1.

Each of these proteins binds to a guide RNA (gRNA) molecule that has been designed to recognize a particular DNA sequence. Each protein allows for different types of cuts—including cut and revise and cut and remove, designed to disrupt a problematic gene or eliminate a mutation. These approaches complete the edit by applying the cell's natural DNA repair mechanisms known as nonhomologous end joining (NHEJ).

EDIT-101 uses NHEJ, as does the other eye treatment in Editas’ pipeline, indicated for genetic and infectious diseases of the eye—including herpes simplex virus type 1 (HSV-1), whose complications can include keratitis; and Usher syndrome 2a, which  includes progressive vision loss that begins in adolescence or adulthood as well as hearing loss from birth.

“Today marks a significant milestone in our collaboration with Allergan and in our work to develop genomic medicines to treat eye diseases,” added Editas president and CEO Katrine Bosley. “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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