Scientific advances in human genome editing have proven that somatic cell genome editing holds great potential to treat or maybe even cure genetic diseases. During the panel, Scientific Advances in Genome Editing: Where Are We Today?, panelists shared insights on how recent advances are accelerating innovation, some of the challenges that stand in the way and how genome editing will become a mainstay in the future.
Biotech company CRIPSR Therapeutics is developing gene-editing therapy in treating rare blood diseases beta thalassemia and sickle cell disease using its proprietary CRISPR/Cas9 platform. It also hopes to use the technology to potentially treat cancer patients. The CRISPR-Cas9 gene editing tool is a breakthrough technology that gives researchers the ability to change an organism’s DNA, so that they are able to remove or alter certain genetic material.
Caribou Biosciences was also represented in the discussion by chief scientific officer Steve Kanner. The company uses proprietary technologies to develop an internal pipeline of off-the-shelf CAR-T cell therapies, other gene-edited cell therapies and engineered gut microbes.
The panelists spoke of a sense of urgency to cure diseases but also a sense of responsibility to lead the patient down the right path. Sandy Macrae, CEO of Sangamo Therapeutics, said it’s important on the path of innovation to identify areas where “the benefit is clear and the risk is as small as possible.”
The panelists agreed that developing any medicine has a specific set of challenges tied to pharmacology, biology, engineering, manufacturing, and so on, but the bigger picture is: can we then apply those tools in the right way to the right biology to treat rare disease and other diseases?
Some of the more pressing challenges that genome editing innovators currently grapple with is delivery, or the sending of the technology to the organ of interest in the body, which depends on the modality you’re using and what organ you’re going to. It also becomes a challenge to determine how high the bar for editing should be. For some diseases, it’s five percent, whereas other diseases may require 30 or 40 percent editing. Safety, of course, is also of utmost importance.
The panelists reaffirmed that the era of genome editing has arrived, and innovation will continue to accelerate. Someday in the future, these groundbreaking innovations will become a mainstay.