New Frontiers in Cell and Gene Therapy
Written by Jürgen Eckhardt, Head of Leaps by Bayer
What do you think of when I say the word “drug”? Chances are that you think of pills, chemical formulations that we take to treat everything from common, everyday pains to chronic diseases. Drug molecules from aspirin to statins to antibiotics have transformed human lives for the better — but too often, drugs only treat the symptoms of a disease, not their root cause, meaning that we have to take certain medicines for weeks, months, or even a lifetime.
I’m tremendously excited about new approaches to medicine that hold the potential to shift the paradigm from treating to curing disease. Many debilitating, chronic diseases like Alzheimer’s disease are caused when cells in the body start to malfunction or die off altogether, and traditional drug treatments are limited. Brain cells can’t generate the way skin or bone cells can. Recent innovations, collectively known as cell and gene therapies, take an ambitious new approach. In contrast to the inert, chemical formulations that we’re most familiar with, cell therapies are living medicines that aim to reverse, or even cure disease by regenerating lost cells with new, healthy ones. Gene therapies aim to address diseases like Hemophilia or Sickle Cell Anemia by correcting or replacing the dysfunctional, disease-causing genes, which are responsible for these conditions. Some approaches combine both cell and gene therapy.
If this sounds like something out of science fiction, that’s because cell and gene therapies rely on cutting-edge biotech. One major advance is the development of induced pluripotent stem cells (iPSCs or PSCs), a process that transforms common cells, which then can theoretically be reprogrammed to make specialized heart cells, liver cells, neurons, and more. A second key innovation is CRISPR-Cas9, which enables more precise editing of a cell’s DNA. Together, these advances make it possible to imagine breakthrough treatments that restore the cellular functions that are lost in degenerative disease.
We’ve been tracking the transformative potential in these approaches since 2015 — it’s thrilling to see how advances in reprogramming and engineering cells could unlock new therapies for a wide range of chronic diseases. In June 2021, BlueRock Therapeutics, one of our first Leaps investments, dosed their first patient in a Phase 1 study of a cell therapy for Parkinson’s disease — an exciting milestone. An estimated 10 million people worldwide live with Parkinson’s disease, which is caused when neurons in the brain stop producing a key brain chemical, dopamine. BlueRock’s therapy introduces PSC-derived, reprogrammed dopaminergic neurons into the brain in the hope of reversing this devastating disease. What’s more, their cell+gene™ platform is enabling the development of therapies for autoimmune diseases and degenerative heart disease as well, showing the immense promise of these cell and gene therapies.
Personally speaking, it has been a privilege to engage with BlueRock over the years and to see how their achievements can drive innovation at Bayer as a whole. In 2016, as cell therapies were just beginning to show their promise, Leaps teamed up with Versant Ventures to co-found BlueRock. With $225 million invested, it was one of the largest Series A financings in biotech history at the time. BlueRock’s early work was so promising that, in 2019, it was acquired by Bayer, where it continues to operate independently. BlueRock’s platform technologies now form a cornerstone of Bayer’s Cell and Gene Therapy unit, a great example of how bold investments by Leaps can position Bayer to access transformative, next-generation technologies.
Many other Leaps investments are working at the next frontiers in cell and gene therapies, part of a growing shift that holds the potential to revolutionize medicine — especially cancer treatment. For example, Century Therapeutics is developing allogeneic (or ‘universal’) iPSC-based cancer therapies that use donor cells to produce therapies that can be used in other patients who need it most. Instead of reprogramming a patient’s own cells (autologous therapies), a costly and time-consuming process, Century’s allogeneic platform is engineering cancer-fighting cells that avoid unwanted immune reactions and are ready to use off the shelf. Khloris Biosciences is developing iPSCs as cancer vaccines, using these engineered cells to teach the body to recognize and destroy cancer cells before they grow out of control. Another example is Triumvira Immunologics, which is engineering next-generation T-cell therapies for cancer.
As our portfolio has grown to over 35 companies, it is exciting to see collaborations emerge between these brilliant teams. In May 2021, Leaps portfolio company Senti Biosciences announced a collaboration with BlueRock to apply its innovative gene-circuit technologies to develop even more efficient, precise cell therapies. Senti’s gene circuits allow cells to sense environmental signals, compute decisions, and respond with precise biological outputs, paving the way for next-generation therapies.
The cell and gene therapy space holds tremendous potential, and we’re excited and humbled to take part in worldwide collaborations to advance the field. This year’s World Medical Innovation Forum focused on cell and gene therapies, and we were honored to be part of the Innovation Discovery Grants and to see the incredible range of new developments.
Through this combination of daring investments and collaborative partnerships, I hope that Leaps can continue to contribute to collectively usher in a new era of medicines that could reverse and cure disease.