CRISPR tool unlocks potential for novel treatments across different therapeutic areas

Nov 22 2015

CRISPR is one of the most exciting advances in gene-based medicine in decades. A uniquely powerful tool for editing the genome, it allows scientists to manipulate DNA with unprecedented precision by deleting unwanted or faulty genes and replacing them with different ones. Its promise in this field of medicine is immense, unlocking the potential for novel treatments across a wide range of therapeutic areas.

Douglas E. Brough, Ph.D., the Chief Scientific Officer at GenVec, Inc., foresees a strong alliance between CRISPR (along with other gene editing technologies) and another innovation he knows very well: GenVec's own AdenoVerse™ gene delivery technology platform. Engaged in the field of gene delivery for more than 20 years, Brough led the discovery teams that developed the key elements of the AdenoVerse platform. He also pioneered drug development efforts such as GenVec's regenerative medicine product candidate for hearing loss and balance disorders, and the development of the first vaccine for foot-and-mouth disease for cattle to be licensed for production on the U.S. mainland.

Under Brough's direction, the AdenoVerse platform has allowed GenVec to design, test, scale-up and manufacture its own adenovectors, which have accumulated a track record demonstrating an outstanding ability to deliver complex genes. The AdenoVerse gene delivery platform can work with a variety of strategies for therapeutics and vaccines, including gene editing tools—not only CRISPR but also TALEN, Zinc Finger, ARCUS and others that are either available now or in development.

GenVec's AdenoVerse adenovectors include specialized features that can overcome the hurdles that hampered the utility of first-generation adenovectors for certain indications, and provide superior performance that could be beneficial for gene editing technology delivery. For example, AdenoVerse vectors include multiple genome deletions of the vector backbone that not only increase gene delivery payload capacity, but also offer the unique opportunity to incorporate the nuclease, guide RNA and donor DNA sequence into a single vector construct.

AdenoVerse gene delivery technology also includes adenovectors based on rare human serotypes and new vectors of non-human origin. Further, the company's adenovectors are non-integrating and episomal on uptake into the target cell, which allows the activity of the nuclease to mediate highly specific editing without further modification from the vector. Finally, the AdenoVerse platform includes Tunable Tropism, allowing broad delivery of genes or delivery to very precisely defined cell types.

As CRISPR and other gene editing technologies continue to be refined, GenVec's AdenoVerse platform may play a key role as an effective adjunct technology.