This is why genome editing is the real turning point in the healthcare sector
Healthcare is a sector that long-term investors cannot ignore and genome editing represents a major revolution. Commentary by Rich Wolf, equity portfolio manager at Capital Group
We've heard of computer hacking before; now the new thing is genetic hacking. In an era of significant opportunities for innovation in healthcare, scientists are tapping into human DNA to identify new ways to treat disease. And this is how the laboratory launched a treatment for sickle cell anemia into the real world, a pathology capable of compromising life expectancy; This is the first approval based on a revolutionary genome editing technology known as the CRISPR system.
Whether it's biotechnology or medical devices, there has always been a major breakthrough that shifts investor sentiment around new technologies or therapies, which can turn out to be a huge success or series of successes. This is exactly what is happening in the healthcare sector right now.
Weight loss drugs from Novo Nordisk and Eli Lilly, originally developed to treat diabetes, are a prime example. The drugs, sold under the names Ozempic, Wegovy and Zepbound, could reshape industries other than healthcare.
Meanwhile, cell and gene therapy companies are forging their own paths. These therapies can modify, replace, activate and deactivate genes. Rather than radically modify human DNA, some companies are working on ways to moderate or refine how genes are expressed. There will be other developments, but the progression will not be linear. The goal is to be able to exploit these technologies in the treatment of pathologies that affect a larger population of patients.
The healthcare sector is a sector that long-term investors cannot ignore. According to the Centers for Medicare and Medicaid Services, healthcare spending in the United States reached $4.5 trillion in 2022, equivalent to 17.3% of US GDP. Healthcare sector ratings have improved. Since the beginning of 2024, investors have returned to healthcare stocks. And if interest rates were to fall, this could encourage continued capital flows into the sector.
However, being at the cutting edge of science always involves failures. Significant barriers remain to the widespread adoption of cell and gene therapies, and investing in healthcare is a decades-long endeavor.
The biotechnology sector is advancing
Companies in the healthcare sector compete to define the methods of treating pathologies. Cell and gene therapy companies, including Vertex Pharmaceuticals, Gilead Sciences and Amgen, address the same conditions that weight-loss drugs are designed for in the kidney, liver and heart, as well as tumors, autoimmune disorders and more.
In the case of cell therapy, cells are modified outside the body and then infused into patients. One specific type is commonly known as CAR-T, which has gained approval as a hematology-oncology therapy. CAR-T stands for “chimeric antigen receptor,” and T refers to a type of immune cell modified to seek out and destroy tumor cells.
Currently available CAR-T treatments use a patient's own cells and are limited by the long and complex journey patients face, manufacturing difficulties and high costs. Treatments could become more accessible and safer as scientists develop non-standardized techniques derived from cells from unrelated donors. We also believe that, over the next decade, companies will move beyond the use of T cells and incorporate other types of cells.
Another area of cell engineering focuses on modifying stem cells to replace missing or defective cells. For example, Vertex aims to cure type 1 diabetes by transplanting insulin-producing cells into the pancreas, a program currently in human clinical trials.
Another promising innovation is RNA interference (RNAi). This technology allows companies to create highly specific therapies that deactivate the production of proteins responsible for the disease. The biotechnology company Alnylam is currently developing programs in areas such as heart failure, hypertension and Alzheimer's. The idea of not irreversibly modifying DNA is compelling, but as with most healthcare innovation, safety is paramount.
This is a machine translation from Italian language of a post published on Start Magazine at the URL https://www.startmag.it/economia/ecco-perche-lediting-genomico-e-la-vera-svolta-in-ambito-sanitario/ on Sat, 22 Jun 2024 05:03:02 +0000.