Adverum Biotechnologies, Inc. (NASDAQ:ADVM), previously Avalanche Biotechnologies Inc., and Editas Medicine, Inc. (NASDAQ:EDIT) have announced a collaboration to employ gene editing technology (CRISPR) in the treatment of up to five inherited retinal disorders. The companies will aim to use their complimentary strengths in delivery and gene-editing to tackle major opportunities in the gene therapy field. Advernum pitches itself as the leader in next generation adeno-associated vector (AAV) deivery while Editas claims leadership in the CRISPR space. The transaction will be of interest on several levels, not least of all in how it values the challenging intellectual property landscape around the relatively new CRISPR assets.
CRISPR gene editing technology is currently generating significant interest in the biomedical community, not least of all due to its low cost, its incredible ease of use and its versatility across a broad range of applications. Referred to as “clustered regularly interspaced short palindromic repeats” or “CRISPR/Cas9”, the technology has emerged from research into prokaryotic immune defence systems. However, the mechanisms at play have recently been shown to correct several gene defects including congenital cataract in mammalian models, with many additional applications in the pipeline. Remarkably, this very ancient bacterial technology is being rapidly applied to several areas of 21st century medicine and biotechnology. The impact is additionally been felt beyond the university walls with enormous sums of venture capital invested to date in a handful of start-up companies alone. In 2013, researchers at the Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, used CRISPR/Cas9 technology to correct a dominant mutation in the mouse crystallin gamma C gene (Crygc), mutations of which may cause significant cataracts. Injection of Cas9 mRNA and a single-guide RNA (sgRNA) targeting the mutant Crygc allele, into animal zygotes, facilitated gene correction via homology-directed repair based on either a supplied oligo or the wild type allele. The researchers reported minimal off-target modifications and confirmed that treated animals transmitted the corrected allele to their progeny.
With similar proof-of-concept studies appearing at a ferocious pace, the real practical challenge is delivery. Delivery of gene-editing tools to the required cells and tissues will determine whether or not the technology can work and such realities will act as the drivers behind collaborations such as that announced by Advernum and Editas. Commenting on the commercial milestone, Paul Cleveland, CEO of Adverum stated, “we are pleased to bring together our gene therapy capabilities with Editas’ CRISPR based approach to genome editing. Our innovative vectors have the potential to deliver Editas’ genome editing components efficiently to the retina. This collaboration expands our opportunities to capitalize on our science, ophthalmology expertise and vector development know-how.”