Allergan plc, a leading global pharmaceutical company, and Editas Medicine, Inc., a leading genome editing company, has announced that Allergan’s wholly-owned subsidiary, Allergan Pharmaceuticals International Limited, and Editas Medicine have entered into a strategic research and development 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 unparalleled 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 Research and Development 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 like LCA10. This program is highly complementary to our ongoing eye care development programs where unmet medical need exists for patients.”
“Allergan has long been a leader in advancing innovative therapies to treat eye diseases,” said Katrine Bosley, President and Chief Executive Officer, Editas Medicine. “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.”
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 United States. Editas Medicine will also be eligible to receive development and commercial milestones, as well as royalty payments on a per-program basis.
About the CRISPR Genome Editing Technology
The CRISPR technology targets specific stretches of genetic code and allows editing of DNA at precise locations in the human genome. Cas9 and Cpf1 are both enzyme/guide RNA complexes that use traditional RNA/DNA base-pairing to precisely locate specific DNA sequences with the goal of modifying or ‘editing’ a disease-associated or therapeutic genomic location. By changing the composition of the guide RNA, the Cas9 or Cpf1 nuclease complex can be reprogrammed to target different DNA sequences and can be engineered to perform a wide range of genome editing functions, including ‘cutting and removing’, ‘cutting and revising’, and ‘cutting and replacing’ genomic sequences. In this way, genome editing has the potential to treat a broad range of genetically-defined and genetically-treatable diseases.
About Leber Congenital Amaurosis
Leber Congenital Amaurosis, or LCA, is a group of inherited retinal dystrophies 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 year of life, resulting in significant vision loss and blindness. The most common form of the disease, referred to as LCA10, is a monogenic disorder caused by mutations in the CEP290 gene and represents approximately 20‑30 percent of all LCA subtypes.