Allergan and Editas Begin First Human Trial of CRISPR-Based Gene Editing Therapy

Allergan and Editas Medicine announced the initiation of the first trial of a CRISPR-based genome editing drug candidate under development for the treatment of Leber congenital amaurosis 10, an inherited form of blindness caused by mutations in the CEP290 gene.

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. Leber Congenital Amaurosis type 10 (LCA10), the most common type of LCA, 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.

The clinical trial will be the world’s first in vivo study of a CRISPR-based genome editing medicine, where the editing takes place inside the human body.

“Beginning patient enrollment in the AGN-151587 clinical trial with our partners at Editas is an important step toward our goal of developing a game-changing, transformative, CRISPR-based medicine for people with LCA10,” said David Nicholson, chief research and development officer, Allergan.

The Brilliance clinical trial is a phase 1/2 study to evaluate AGN-151587 for the treatment of LCA10. The study will assess safety, tolerability, and efficacy in approximately 18 patients. Up to five cohorts across three dose levels will be enrolled in this open label clinical trial in multiple centers. Both adult and pediatric patients ages 3 to 17 years old with a range of vision will be evaluated. Patients will receive a single dose of AGN-151587 administered via subretinal injection in one eye following vitrectomy. The partners expect the first patient dosing to occur in the second half of 2019.

“Now that enrollment is underway, we are one step closer to delivering a transformative medicine to LCA10 patients,” said Charles Albright, chief scientific officer, Editas Medicine. “The team at Editas looks forward to continuing to collaborate with our partners at Allergan, patient advocacy organizations, and the inherited retinal diseases community as we develop this and other durable experimental medicines for patients with devastating ocular diseases.”

Sites in the U.S. are currently enrolling patients for the trial, including Massachusetts Eye and Ear, an international center for treatment and research and a teaching hospital of Harvard Medical School.

“Today marks an important day for the inherited retinal disease community, and specifically those affected by LCA10,” said Ben Yerxa, CEO, Foundation Fighting Blindness.

In March 2017, Editas Medicine and Allergan Pharmaceuticals entered a strategic alliance and option agreement under which Allergan received exclusive access and the option to license up to five of Editas Medicine’s genome editing programs for ocular diseases, including AGN-151587.

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 AGN-151587 globally for the treatment of LCA10. Additionally, Editas Medicine exercised its option to co-develop and share equally in the profits and losses from AGN-151587 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 CRISPR genome editing platform.

Photo: David Nicholson, chief research and development officer, Allergan

Author: Rare Daily Staff