Ridding the world of disease is on everyone’s wish list. Most don’t think it is possible. But, the world of genetic medicine has been constantly evolving, and the Nobel Prize-winning gene editing technology, CRISPR, has brought unprecedented possibilities. Along with it, many unique challenges. Ross Bundy, CEO and co-founder of CRISPR QC(a platform created to help predict and optimize gene editing results) dives into this vast “wild” frontier of gene editing with its immense potential and the serious need for quality control.
Bundy describes the field of gene editing as the "Wild West" of the life sciences, as the concept of gene editing is a relatively new and rapidly advancing landscape. With CRISPR, its founders, Jennifer Doudna and Emmanuelle Charpentier, simplified the process and made it look much more accessible.
CRISPR brought about the ability to efficiently and effectively edit genomes. Bundy explains, “Sequencing became a big thing. Researchers could sequence, customize, and control cells to do whatever they want, so people got really excited.” Researchers in the field began to hope for a world where they could eradicate genetic defects that cause disease. And with Bundy’s CRISPR QC proprietary Analytics Platform that interrogates editing activity, they can do it quicker and more concisely, thereby making it safer and more reliable.
However, Bundy emphasizes that academia tends to prioritize exploring the theoretical potential of an application over addressing its commercial viability and scalability. He doesn't question academia's role in inspiring scientific progress but notes that the criteria that push research publications are very different from those that push commercial accessibility and success.
The cutting-edge discoveries by academia are vital, but product development has different standards. “What's very boring in anything scientific, but is absolutely critical about a product, is consistency, reproducibility, doing the same thing thousands and thousands of times, and getting the same result every single time.” That is where things get complicated and where CRISPR QC and Bundy’s team make their mark.
Naturally, the capability of editing human genes has sparked significant interest in the life science community and the world. However, the methods for doing it are ripe with challenges and still constantly evolving. Bundy emphasizes the necessity of precise control and risk management. The potential for off-target effects and unintended edits raises many red flags when directly injecting CRISPR into human bodies. Bundy and his team are helping researchers minimize the red flags.
“Now we're talking about editing people, but the methods haven't been developed. A lot of what we're trying to do is say that there are methods that have been successful in other industries, taking very complicated scientific breakthroughs like semiconductors, building nuclear power, and all these other things. And those industries are very reliable and very safe, and they've done it in certain ways that don't exist in life science. But that's what we're, CRISPR QC, trying to bring to the table. And to say, ‘yeah, now that it's possible… if we're going to actually make this a real industry… now is the time.’”
The FDA has been hesitant to support progress in gene editing. Bundy says their caution stems from the fact that these sciences are extremely novel and being used in new areas with little knowledge. What weighs most heavily is likely a trial from 20 years ago that had disastrous consequences. “People were saying we can take these genes and insert them in people's DNA and cure people of this disease.” After successful animal and human trials, they moved forward. “But, at the time, they weren't doing gene editing. They were actually using a virus to deliver a certain genome, a certain sequence, into people's DNA. And the challenge with many genetic diseases is that these are kid diseases. The trial ended with some of its patients, children, dying when they were inserted with genetic material.”
Bundy adds that the FDA is coming back around, but they are reluctant to make another mistake. The FDA sees gene editing, in a sense, as another form of gene therapy, thereby the hesitancy and the greater need for Bundy’s CRISPR CQ. “There is zero room for error. You can’t make a mistake.”
There is excellent promise surrounding the potential of gene editing, especially with CRISPR QC’s platform, providing researchers the ability to predict and optimize the process. Still, one of the biggest hindrances in gene editing is its affordability and, thereby, its accessibility.
Bundy stresses that making these therapies economically accessible has to be at the forefront of its evolution. Some gene therapies, like curing sickle cell anemia, cost $2.5M per patient, thereby severely limiting access to those who need it most.
Bundy says that the key to making new therapies affordable and ultimately accessible is control and emphasizes the potential benefits of practices like Lean Six Sigma to efficiently improve quality, reduce costs, and scale gene editing technologies.
“My view is, can we deliver that same value but for one 20th the price and get it to something insurance could afford and make it accessible to the people you are trying to help? And as much as we all like to say there’s no cost to people’s health, there is. There is a cost, and people won’t pay for it if it is too much. So, it’s got to be affordable in order for it to be truly effective.”
With CRISPR QC, the potential to wrangle gene editing–making it safer, more affordable, and more accessible–is on the horizon. CRISPR QC’s quality control metrics and essential data is taming the wild west of gene editing and revolutionizing advancements in therapeutic medicine and sustainable agriculture.
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