On Innovations in Gene-Editing, Addressing Blindness and More
Photo by Viktor Talashuk
Last week, CRISPR gene-editing was used for the first time to edit a person’s DNA while it was still in their body. This was part of clinical trial to remove a cause of blindness in young children. The project is a collaboration between Oregon Health & Science University (Portland, OR), Editas Medicine (Cambridge, MA) and Allergan (Dublin, Ireland)
Charles Albright, Editas Medicine chief scientific officer said, “We literally have the potential to take people who are essentially blind and make them see …. We believe that the ability to edit inside the body is going to open entire new areas of medicine and lead to a whole new class of therapies for diseases that are not treatable any other way."
Mark Pennesi, M.D, Oregon Health & Science University said “Being able to edit genes inside the human body is incredibly profound … our first treatment in this clinical trial is an important step toward bringing new and promising treatments to patients with disease-causing gene mutations."
Dr. Eric Pierce, professor of ophthalmology at Harvard Medical School said, "We're helping open, potentially, an era of gene-editing for therapeutic use that could have impact in many aspects of medicine … We're really optimistic that this has a good chance of being effective."
NOTES: 1) Editas Medicine is commercializing CRISPR/Cas9 and CRISPR/Cas12a genome editing systems to address serious diseases. 2) CRISPR is a gene editing technology for use with any living organism. 3) Leber congenital amaurosis, the disorder the clinical trial focuses on, is caused by mutations in at least 18 different genes that causes blindness or severe visual impairment in infants.
OUR TAKE
In healthcare, CRISPR research efforts are addressing needs in areas such blood, muscle and brain disorders. The opportunity set will continue to expand.
In agriculture, CRISPR techniques are already in commercial use - to improve crop yields, increase disease resistance and enhance flavor.
Other CRISPR uses under consideration (or speculated about) include increasing an athlete's performance eliminating pests such as mosquitoes, improving biofuel production, tracking environmental changes, refining pet breeding processes and more.
Regarding CRISPR economics: in some markets, innovative solutions should provide attractive (lower cost) alternatives relative to incumbent theraputic approaches. In other areas, the cost to deliver CRISPR solutions may be out of reach to many people, which could trigger social and ethical concerns.
Bottom line: CRISPR-Cas9 and other gene-editing technologies will drive significant innovation in health care, agriculture and other life sciences.