Allergan and Editas Medicine all set to discover and develop CRISPR genome editing medicines for eye

Alliance brings together eye care and CRISPR innovators to develop transformative medicines for patients.

Allergan and Editas Medicine have announced that Allergan's wholly-owned subsidiary, Allergan Pharmaceuticals International, and Editas Medicine have entered into a strategic R&D alliance under which Allergan will receive exclusive access and the option to license up to five of Editas Medicine's genome-editing ocular programs, including its lead program for Leber Congenital Amaurosis (LCA10), which is currently in pre-clinical development.

The agreement covers early-stage, first-in-class ocular programs targeting serious diseases based on Editas Medicine's CRISPR genome editing platform, including CRISPR/Cas9 and CRISPR/Cpf1. Editas Medicine's lead program is being developed for the potential treatment of LCA10, a rare, inherited retinal degenerative disease that appears in childhood and leads to blindness.

"The CRISPR genome editing platform holds the potential to transform the treatment of many genetic and non-genetically derived diseases, including diseases and conditions of the eye," said David Nicholson, Chief R&D Officer, Allergan. "The Allergan team is excited to work with colleagues at Editas Medicine to develop and potentially deliver game-changing treatment for retinal diseases such as LCA10. This program is highly complementary to our ongoing eye care development programs where unmet medical need exists for patients."

"Working together with Allergan through their Open Science R&D model significantly enhances our ability to develop genome editing medicines to help patients with serious eye diseases. This alliance is highly aligned with our strategy to build our company for the long-term and to realize the broad potential of our genome editing platform to treat serious diseases," said Katrine Bosley, President and CEO, Editas Medicine. 

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a dynamic, versatile 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 and has the potential to create medicines with a durable treatment effect.

Under the terms of the agreement, Editas Medicine will receive an upfront payment of $90 million for the development of five candidate programs. Editas Medicine has the potential to earn additional payments for achieving important near-term milestones specifically related to LCA10. Allergan will have the option to license up to five programs under the agreement and will be responsible for development and commercialization of the optioned products, subject to Editas' option right to co-develop and co-promote up to two optioned products in the US.  Editas Medicine will also be eligible to receive development and commercial milestones, as well as royalty payments on a per-program basis.