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May 8, 2023
Synthetic biology author. Got it! Juliana Sohn In the world of biotechnology, few innovations have generated as much excitement and promise as gene editing. With the advent of technologies such as CRISPR/Cas9, researchers are unlocking new frontiers in widespread disciplines ranging from agriculture to medicine. One of the pioneering minds driving this field forward is Harvard University Professor David Liu, Ph.D. His newest company, Nvelop Therapeutics, joins Liu’s impressive list of nine others, each of which has exemplified the vast applications of gene editing. From addressing drug targets previously thought inaccessible to engineering healthier food choices for combating the obesity crisis, Liu’s work is truly pushing the limits of what is possible in biotechnology. The CRISPR/Cas9 system has spurred shockwaves in the field and holds great promise for significant impact in disease research and therapy. This innovative technique involves utilizing the Cas9 enzyme, which functions as molecular scissors, to make precise cuts in DNA at specific sites. The Cas9 enzyme is directed to the targeted location by a guide RNA (gRNA), a small RNA molecule designed to complement the DNA sequence at the desired site. Once the Cas9 enzyme and the gRNA have located the target site, the enzyme cuts both strands of DNA, allowing for the replacement or insertion of specific sequences at the site of the cut. Despite the success of the CRISPR/Cas9 system, it still has limitations that prevent it from achieving its full potential. While CRISPR’s ability to disrupt target genes has been beneficial for certain therapeutic applications, the large majority of genetic diseases arise from mutations that cause genes to lose their function altogether. “Most disease-causing mutations thus require precise gene correction, rather than gene disruption, to best benefit patients,” Liu stated. Staying On Target To address the off-target issues, Liu and his Harvard University team developed base editing as an add-on to the CRISPR/Cas9 system. In contrast to the traditional CRISPR/Cas9 system, base editing works to change one DNA letter to another. This is achieved without causing damage to other parts of the DNA by using a modified version of the Cas9 enzyme. This system makes base editing a valuable tool for researchers as it’s more accurate than traditional editing and doesn’t require complex repair mechanisms. The impact of base editing is underscored by the case of Alyssa, a teenage girl diagnosed with an incurable form of leukemia known as T-cell acute lymphoblastic leukemia. After several other treatments failed, doctors at the Great Ormond Street Hospital in the U.K. turned to base editing to cure her disease. Using the technology developed by Liu and his team, doctors were able to edit Alyssa’s DNA successfully such that her modified T-cells could find and destroy the cancerous T-cells, thus curing her leukemia. This breakthrough in personalized medicine has given hope to countless others with previously deemed incurable diseases. Liu’s company, Beam Therapeutics, founded in 2017, hopes to apply base editing technology to other human genetic diseases, such as Sickle Cell Anemia and Cystic Fibrosis—to treat and perhaps cure millions of patients. In January of 2022, Beam Therapeutics launched a four-year research collaboration with Pfizer Nothing Ventured Nothing Gained Liu’s companies extend beyond therapeutic development. Pairwise Plants is one of the entrepreneur’s most successful ventures and is driven by the mission that “healthy shouldn’t be a choice–it should be a craving.” The company applies its industry-leading CRISPR gene editing technology to traditional plant breeding methods, leveraging the ability to select specific favorable traits such as sweetness or color over others. Additionally, the company has taken a stance on the mounting environmental challenges surrounding agriculture. In a strategic alliance with Bayer, the two companies are working to help farmers produce better harvests, conserve resources, and protect crops from evolving threats. Since 2003, Liu has founded eight other companies using novel gene-editing tools: Ensemble Therapeutics, Permeon Biologics, Editas Medicine, Exo Therapeutics, Prime Medicine, Chroma Medicine, and Resonance Medicine. When asked about his decision to start several new companies, Liu explained that "patients’ interests are best served by recruiting a talented team who wakes up each morning and their top professional priority is to use that technology to treat patients.” In addition, he envisions that his wide range of companies will minimize expenditures of time and resources as the number of patients that can benefit from any of his therapies is maximized. Nvelop Therapeutics, Liu’s most recent venture, marks a significant milestone in his career. Dedicated to advancing innovative approaches to deliver a variety of therapeutic payloads using gene editing, the Nvelop team believes their technology has the potential to impact patients suffering from severe genetic diseases positively. Liu’s prolific career over the past two decades has won him several prestigious distinctions. In 2017, he was named one of Nature’s Top 10 researchers in the world. Additionally, his invention of base editing was marked as a historical discovery in the gene editing field, rewarding him as a Finalist for the Science 2017 Breakthrough of the Year. Other distinctions include the American Chemical Society Pure Chemistry Award and David Perlman Award. Moreover, his laboratory has published over 225 papers, and he is the inventor of over 90 issued U.S. patents. Despite advancing gene editing technology to new heights, Liu also acknowledges the ethical concerns surrounding the technology and has been vocal about the need for responsible use and regulation. It is only then that the true potential of gene editing can be obtained safely and equitably. In our interview, Liu expressed he is “optimistic that the coming years will bring much of the promise of therapeutic gene editing to patients.” It is his career’s ultimate mission that “humans have some say in the sequences of our genomes so that we are no longer so beholden to the misspellings in our DNA.” Thank you to Sohum Phadke and Jeffrey Buguliskis for additional research and reporting on this article. I’m the founder of SynBioBeta and some of the companies I write about are sponsors of the SynBioBeta conference . For more content, you can subscribe to my weekly newsletter and follow me on Twitter and LinkedIn . Follow me on Twitter or LinkedIn . Check out my website .
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