How far should science go to fight disease, or alter the food supply to withstand climate change? Some would say we need to push science to its limit, do anything we can, to keep humans safe and our food sources healthy and robust. At the forefront of these efforts are gene editing technologies… it’s truly an exciting time to be alive as we are among the first humans to witness gene editing moving from concept to reality. We’re now living in a world where we can alter living beings that have evolved over millions of years to meet our needs. It’s the stuff of fantasy come to life and the perfect example is gene edited mosquitoes.
Recently, biotech firm Oxitec announced results from a groundbreaking trial conducted involving genetically modified mosquitoes. Researchers from the team have completed the first open-air study of genetically engineered mosquitoes in the United States and are reporting what they call “positive” results. During the experiment, which began in April of 2021 in the Florida Keys, researchers released and monitored almost five million Aedes aegypti mosquitoes over the course of seven months… the special thing about these mosquitoes is that they were developed for a specific purpose using gene editing technology.
Mosquitoes carry diseases, and the types of mosquitoes released by Oxitec can carry some really nasty ones, like chikungunya, dengue, Zika, and yellow fever. Scientists have figured out how to engineer the males to carry a special gene that is lethal to the female offspring of the species. The gene edited males mate with wild females and instead of healthy babies, the female offspring of the pair die before they are able to reproduce. It sounds a bit macabre, but the goal is to reduce the population and this solution does the trick.
Keep It Commercial
While these mosquitoes may sound wild, this is only the tip of the iceberg, gene editing is all around us in some form or another and is becoming more commonplace by the day. So how exactly does gene editing work? Basically, our DNA is made of a combination of four letters, to the tune of three billion specific arrangements, and how these letters are arranged make up the instructions for how our bodies are made. Gene editing can find a specific stretch of DNA inside a cell, bind to it, and then precisely cut the DNA at a defined location, which can induce the desired DNA sequence changes in the cell… and the space holds massive potential. According to projections, the global genome editing market size will reach 19.06 billion by 2028 and was valued at 3.8 billion in 2021.
Climate Cows And Doctor Mice
Would you believe me if I told you that you could be eating gene edited cows? Well, it’s true. The FDA has approved gene edited cattle for meat production… or, more specially, their offspring will be used for meat production. The cattle, known as PRLR-SLICK, were engineered to be able to withstand a warming climate to make sure that we humans still have beef to eat as our world becomes hotter. You may be thinking that genetically modified organisms (GMOs) have been a part of our food supply for a while now, and you would be correct… but gene edited cattle are not the same.
Genetically modified organisms (GMO) are created by adding genetic material from one organism to another, which results in a plant or animal that would not exist in nature on its own. Gene editing, on the other hand, utilizes genes already native in a species, resulting in an organism that could occur in nature. PRLR-SLICK cattle have extremely slick, short hair to help the animals cope with hot weather more effectively, be less heat-stressed, and gain more weight easily. As long as all planned safety reviews are passed, we’ll see this meat on the shelves within about two years.
Another animal that has been gene edited and is being incorporated into our lives is a mouse that has been altered to help curb Lyme disease. These special white-footed mice, which look exactly like native white-footed mice, were created so that each of their cells would carry genetic code specially tailored for resistance to the bacteria that causes Lyme disease. White-footed mice are a key reservoir for the harmful bacteria and since mice breed quickly, the gene will be passed down to descendants quickly and with fewer mice carrying the disease, fewer ticks will latch onto them, catch it, and pass it on to humans.
Deals To Watch
While we’re not quite ready to start hacking the genes of humans, there are companies using gene editing technologies to advance treatments for humans. A great example is Graphite Bio (GRPH), which is working on gene-editing treatments for sickle cell disease, an immune system disorder and Gaucher disease. The company was able to raise $238 million with an IPO, listing shares on the Nasdaq last June. The timing was terrible . . . it’s barely a $4 stock now.
But hope springs eternal. The next wave of deals could be the ones to break through.
Boston-based HilleVax, formed by Takeda Pharmaceutical and private-equity firm Frazier Healthcare Partners, is a clinical-stage biopharma developing vaccines that is looking to trade on the Nasdaq. The company has filled for a $100 million IPO after the initial success of it norovax candidate in recent clinical trials. The vaccine, being evaluated for use for acute gastroenteritis has shown high levels of efficacy. The company has been able to attract top talent, including Julie Gerberding, M.D., former Centers for Disease Control and Prevention Director and Robert Hershberg, M.D., Ph.D., former Executive Vice President of Business Development and Global Alliances of Celgene Corporation, a pharmaceutical company that makes cancer and immunology drugs.
Intrinsic Medicine, based in Seattle, is a venture-backed, clinical-stage biotech headed by co-founder, Chairman, and CEO, Alexander Martinez working to bring important medicines efficiently and ethically to patients and is currently working on developing HiMO therapies for GBA and inflammatory disorders… basically, treatments for irritable bowel syndrome and variants. The global market for irritable bowel syndrome treatments was an estimated $1.5 billion in 2018 and is forecast to reach $3.2 billion by 2026. The company, which was founded in 2018, has filed to raise $47 million in an IPO of its common stock according to an S-1 registration statement.
Regardless of any reservations we may have about gene editing technologies, they are here and are becoming more prolific… we are entering a new frontier of possibilities that these technologies are unlocking. We are now looking at the possibility of changing human, plant, and animal genes in ways that could benefit the world that we could not have dreamed of only a few decades ago. Stay tuned to see which gene editing technology companies land on my Buy List and keep checking out future issues as we cover the evolution of these technologies.