Big Biotech Breakthrough Opportunities in Healthcare to Watch in 2021 and Beyond
Written by Jürgen Eckhardt, Head of Leaps by Bayer
At Leaps, we are constantly scanning for potential biotech breakthroughs that hold promise to either cure millions of people from illness around the globe, or help feed a growing population amidst intensifying climate change. After the close of a challenging and historic year, we’re taking a look at the biggest biotech developments to watch in 2021 and beyond that can help us meet these goals.
This past year has put health and medical innovation at the center of public discussion. As COVID-19 ravaged the world, researchers were left to ask, how could devastation of this scale happen in the first place? The year was 2020, after all. Shouldn’t a society that eradicated polio long ago, and which has sophisticated public health research taking place in every corner of the globe, not have been caught so unaware by the coronavirus, let alone suffered so terribly from it? Many are now hungry for a paradigm shift in medicine — one that moves from treating disease to preventing it in the first place, and curing it fully if it arrives. With mRNA vaccines on the way with unprecedented speed thanks to commitment and collaboration, our community is focusing on how to establish this paradigm shift and apply it well beyond this current crisis.
Cell and gene therapy
This is an overlapping field that holds enormous promise for restoring health and curing disease. Cell therapy involves delivering whole cells to a patient through graft, injection or implantation to improve the health of tissues and organs, and ameliorate disease. Gene therapy involves modifying a cell’s genes for curative and therapeutic impact, either directly inside a patient’s body or in the lab, and then transferring those modified cells into the patient.
The field includes cutting edge work with induced pluripotent stem cells, gene editing, gene augmentation, and whole organ therapy. For example, BlueRock Therapeutics is a next-generation cellular medicine company that Leaps co-created five years ago and Bayer fully acquired in 2019. BlueRock has grown into an integral part of Bayer’s new Cell and Gene therapy unit, which was created to accelerate innovation in this area and translate it into tangible therapies. We applaud Bayer’s recent acquisition of Ask Bio, which has secured industry-leading expertise in regenerative cell and gene therapy to deliver this exciting category of medicine at scale.
Although there have been multiple challenges when trying to commercialize cell and gene therapy products, the landscape shows tremendous potential for curing intractable diseases. Restorative neurology, for example, transfers healthy cells and genes to patients with neurological disease. This holds promise to cure Parkinson’s in the future by implanting healthy cells that can replace lost tissue function.
Next generation immunotherapies
2020 put the human immune system in the limelight as COVID-19 hit populations around the world, making it a ripe time for vaccine development and other biotech innovations. Next-generation immuno-oncology is also advancing, and many new treatments that take advantage of the body’s immune system to fight cancer have recently appeared. Beyond CAR-T therapies that have already hit the market, for example, new drugs are in clinical trials to treat melanoma, lung cancers, and head and neck cancer, all with a patient’s own immune cells. In 2020, Leaps invested in Triumvira, a biopharmaceutical company developing a novel platform for engineering T-cells to attack solid tumors. Researchers are exploring several potent anti-tumor responses through a variety of immune system avenues, and the future is looking bright. The next step in the evolution of immunotherapies will be when they become deliverable at scale. Therapies that use a patient’s own cells — called autologous cell therapies — are often costly, but approaches pioneered by companies such as Century Therapeutics are able to develop cell therapies using induced pluripotent stem cells (iPSCs) — called allogeneic cell therapy. Advances like these could dramatically enhance the accessibility of these therapies.
AI for drug discovery
The next generation of healthcare solutions are cross-functional, combining AI with basic biology, chemistry, and physics. Drug discovery stands to particularly benefit from this convergence because machine learning can seek out new therapeutics far quicker, cheaper and more effectively than other methods. The way we discover drugs and test them in clinical trials is all about pattern recognition, which AI is supreme at analyzing in large data sets. Structure-based small-molecule detection is made magnitudes easier when AI is involved to automate assays of large samples and analyze immense amounts of biological data from patients in clinical trials, as well as look for drug targets and therapies for a variety of conditions. Towards this end, Leaps has invested in Recursion to support their digitally-powered drug discovery pipeline. They’re focused on addressing fibrotic diseases with previously undiscovered mechanisms and getting therapies to patients more rapidly.
Drug discovery can be carried out through a variety of methods, but there is growing excitement about how our ability to predict the 3-D structures of proteins will substantially advance this field. Proteins are complex molecular structures that act as the building blocks for all biological functions in all species. The way they get their function stems from the ways in which they’re folded. For instance, the now-iconic “spike-proteins” that shoot up on the exterior of the coronavirus are what allow it to enter human cells. mRNA COVID-19 vaccines mimic the shape of those proteins, spurring the body to produce an immune response[1]. Figuring out protein structure is therefore key to unlocking a treasure trove of knowledge about how to create potential applications, such as vaccine development and drug discovery. The latest achievement of Deep Mind’s Alphafold, which uses a neural-network based algorithm to predict the 3-D structure of proteins from amino acid sequences is a huge leap forward in this space.
From cell and gene therapies, to next-generation immunotherapies, to AI for drug discovery, healthcare is advancing at breakneck pace in several directions that we couldn’t be more excited about. At Leaps, we are passionate about restoring patient health and preventing disease from taking hold in the first place. These emerging biotech breakthroughs strengthen our confidence in our ability to do just that. Here’s to a healthier 2021, comprised of scalable solutions for those who need them, no matter where they live or what walk of life they come from.
[1] https://jamanetwork.com/journals/jama/fullarticle/2770485
