Allergan Inc. (NASDAQ:AGN) and Editas Medicine Inc. (NASDAQ:EDIT) have announced the execution of an exclusive licensing transaction in which Allergan receives an option on up to five of Editas’ CRISPR ocular research programmes, including Editas’ lead programme for a product candidate to treat Lebers congenital amaurosis (LCA10). Under the terms of the deal Editas will receive $90M in an upfront payment from Allergan and will also be eligible for undisclosed milestone and royalty payments on each opportunity. Editas will deliver a preclinical data package on each programme, over which Allergan may opt to develop and commercialise while Editas are understood to retain an option to co-develop and co-commercialise two specific programmes in the US market.
CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a “big” development within biotechnology. The core opportunity has been developed by a number of key players, chiefly the founding scientists currently associated with several start-up companies including Crispr Therapeutics (Basel, Switzerland), Caribou Biosciences, Inc. (Berkeley, California), Intellia Therapeutics (Cambridge, Massachusetts), ERS Genomics (Dublin, Ireland) and Editas Medicine (Cambridge, Massachusetts), The gene editing technology derives from observations made in the repetitive sequences isolated from a number of prokaryotic and archaebacteria, first identified in 1987 by Yoshizumi Ishino and Atsuo Nakata, then at Japan’s Research Institute for Microbial Diseases, Osaka University. Following on from the original observations three papers in 2005 reported that spacer sequences separating individual repetitive sequences appeared to have a phage associated origin. Coupled to this were separate observations that viruses were unable to infect cells that carried spacer sequences corresponding to their own genomes. In essence, the system as a whole appeared to represent an un-expected and sophisticated immune system for prokaryotes, essentially a new mechanism that provided an immune memory of previous phage infections and facilitated rapid clearing of subsequent phage invasions that had previously infected the cell. By 2012 a paper published in Science by Jinek and colleagues at the University of California, Berkeley, in collaboration with the University of Vienna and Umeå University in Sweden, harnessed the technology into a system that is “efficient, versatile, and programmable” with “considerable potential for gene-targeting and genome-editing applications.” Further iterations and development showed how relatively easy the CRISPR system could be used as an RNA-guided platform for specific control of gene expression. When compared to other editing tools, such as zinc finger proteins or TALENs, CRISPR has proved to be remarkably cheaper and less time consuming to work with. Since 2012 hundreds of millions of dollars have been invested in CRISPR R&D in both the academic and commercial arena and the latest transaction between Allergan and Editas is a continuation of the drive to market for this novel gene editing technology.
Commenting on the CRISPR deal, David Nicholson, Chief Research and Development Officer, Allergan stated, “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. 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.”