The rapid advances in cell therapy are among the most exciting avenues for the future of biotech. As I have written before, an emerging field of cell therapies harnesses the incredible powers of induced pluripotent stem cells (iPSCs) to regenerate tissues lost and damaged in conditions like Parkinson’s disease, heart disease, retinal disease, diabetes, and beyond. These living medicines, built from live cells that can carry out complex regenerative functions, provide glimpses of a possible future, one in which Leap 02 / Provide sustainable organ and tissue replacement becomes a reality.
A handful of cell therapies have been approved by the FDA and are beginning to reach patients, with hundreds more in clinical trials. But this new paradigm requires bold new approaches to foundational problems of manufacturing. Making a cell therapy is a very different process from synthesizing a small-molecule drug, due to the complex growth requirements of iPSCs. Currently, cultivating and maintaining iPSCs is a challenging, time-consuming process that relies on the work of highly skilled scientists. Solving the challenge of manufacturing cell therapies could significantly expand patient access and even the scope of addressable diseases.
In particular, scaling up cell manufacturing could unlock the potential of a promising class of treatments known as autologous cell therapies. Many cell therapies currently under development are allogenic, or “universal” cell therapies, which use a single source of iPSCs to treat patients. Since these allogenic therapies can be used to treat many patients, they can help lower the cost of cell therapies, but the risk of inducing potentially life-threatening immune responses must be navigated. In contrast, autologous therapies are derived from a patient’s own cells, which are much less likely to be rejected by the immune system. A key barrier to making these personalized treatments a reality has been the laborious process of cultivating a patient’s own cells.
Leaps recently invested in Cellino Biotech, a company combining stem cell biology with AI and laser physics to tackle the problem of manufacturing cell therapies at scale. Cellino’s platform could help solve this bottleneck through a fully autonomous process that removes the manual-intensive steps, resulting in a higher-quality, scalable, standardized, and lower cost process. This type of technology could enable a whole new class of exciting cell therapies to reach patients — therapies that would be otherwise limited by costs and challenges of the manufacturing process. The company’s groundbreaking approach aims to build early-stage Good Manufacturing Practice (GMP) capabilities for cell therapies, a crucial step towards the company’s goal of developing the world’s first autonomous human cell foundry in 2025. The foundry aims to use high-throughput machines to process human samples and turn cells into autologous therapies. The company’s innovative platform combines software and hardware advances in label-free imaging, high-speed laser editing, and machine learning and could increase efficiency and lower costs by orders of magnitude, making potentially lifesaving cell therapies accessible to patients at scale. To learn more about the potential of autologous cell therapies, I recommend this great TED talk by Dr. Nabiha Saklayen, CEO and co-founder of Cellino and a recipient of the Tory Burch Fellowship at the Innovative Genomics Institute.
Cellino joins an inspiring set of Leaps portfolio companies that are taking groundbreaking approaches to unlocking the potential of iPSCs. One of our first investments, BlueRock Therapeutics, is conducting a Phase 1 clinical trial in the U.S. and Canada of a cell therapy to treat Parkinson’s disease and continues to advance their powerful cell+gene platform, which reprograms mature, differentiated cells back to iPSCs and applies advanced engineering to enhance the cells’ therapeutic capabilities. BlueRock is now a cornerstone of Bayer’s Cell and Gene Therapy platform. Century Therapeutics and Khloris Biosciences are developing allogenic and autologous iPSC-based therapies to fight cancer, and Senti Biosciences is collaborating with BlueRock Therapeutics to use approaches from synthetic biology to develop smarter, more precise cell therapies that can respond to environmental cues. With these partnerships, I hope that Leaps can help usher in a new generation of cell therapies that shift the paradigm from treating to curing disease.