2022 saw some incredible scientific and technological breakthroughs, as well as some major medical breakthroughs — and 2023 is poised to follow up with more paradigm-shifting advances in the making. As the Deputy Director of Communications for Leaps by Bayer, I surveyed a brain trust of scientists, investors, and CEOs to learn which areas of biotech they are most eagerly watching this year. Here’s what they said.
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2022 saw some incredible scientific and technological breakthroughs, as well as some major medical breakthroughs — and 2023 is poised to follow up with more paradigm-shifting advances in the making. As the Deputy Director of Communications for Leaps by Bayer, I surveyed a brain trust of scientists, investors, and CEOs to learn which areas of biotech they are most eagerly watching this year. Here’s what they said.
Cell and gene therapies
Cell and gene therapies encompass a wide range of approaches that attempt to treat disease at the cellular and genetic levels. With cell therapy, patients’ cells are extracted, reprogrammed, and then injected back into their bodies, often with the goal of leveraging their own immune systems to fight diseases like cancer. Of the approved cell therapies in the U.S., most focus on blood cancers because they have not yet shown success in treating solid tumors. One of the next goals in cell therapy is to improve efficiency by reprogramming patients’ cells in vivo — that is, inside their own bodies.
In gene therapy, genetic material is delivered into patients’ bodies with the objective of replacing or correcting faulty DNA. Several gene therapies recently have been approved to treat a variety of disorders: a rare eye disease called Leber congenital amaurosis, spinal muscular atrophy in infants, and hemophilia B in adults. One limitation of current gene therapies is their delivery system. They are typically delivered to patients via a viral vector, a harmless virus that carries its genetic cargo into the body, but this approach typically only affects cells in the liver. Delivering precision genetic medicine to organs and cells beyond the liver represents one of the next frontiers in gene therapy. ReCode Therapeutics, a Leaps portfolio company, is at the forefront of that effort.
“The promise of changing the ability of a cell to alter the course of disease with a simple injection is very exciting and helped tremendously by efforts and success of the Covid-19 mRNA vaccines.”Laura Shawver, CEO of Capstan Therapeutics
Synthetic biology
Synthetic biology involves the wholesale redesign or construction of biological entities to carry out desirable biotechnological processes. It’s already revolutionized the treatment of diabetes, for example. Since 1982, insulin has been produced by inserting a human gene into a common bacterium. Today, through companies like Ginkgo BioWorks, a Bayer partner, many more opportunities are arising to use synthetic biology in everything from medicine to farming to manufacturing.
