Combining coding and biology to design human cells
bit.bioa leading synthetic biology company, aims to combine coding and biology to transform human cells into a new generation of drugs using human-induced pluripotent stem cells (iPSC). Their current goal is to develop a scalable technology capable of producing consistent batches of every human cell using the principles of stem cell biology, cell reprogramming, mathematical modeling and cell therapy.
To learn more about new technologies being developed for drug discovery and cell therapy, Technology networks spoke to Dr. Farah Patell-Sochavice president of research products at bit.bio, and Furqan Iqbalsenior director of information systems at bit.bio.
Molly Campbell (MC): Can you discuss the rationale for combining the concepts of coding and biology to improve human cells used for research, drug discovery and cell therapy?
Farah Patell-Socha (FPS): At bit.bio, we consider the cell as if it were a computer, the cell nucleus containing the DNA or the hard disk. Embedded in DNA is the software that stores all genetic programs. We know that by entering a unique combination of genetic code into a cell’s DNA, we can trigger the reprogramming of a cell in any cell type – the key is to find the right code and combination unique for each desired cell type.
We apply a new alternative approach to the generation of human cells, called “precision reprogramming”. This next-generation cell reprogramming method uses the exclusive property of bit.bio opti-ox™ platform, with systematically optimized and inducible gene expression to enter the correct code into stem cells, telling them to transform into a specific human cell type. opti-ox enables large-scale manufacturing of unlimited batches of any human cell, overcoming many of the challenges faced by traditional cell reprogramming methods such as site-directed differentiation and cell reprogramming.
By taking advantage of precision reprogramming, we have already been able to produce two types of cells on an industrial scale: skeletal muscle cells and neurons, which are available for researchers to use “off the shelf”. Our cells provide a physiologically relevant response in vitro A reliable and consistent batch-to-batch drug discovery and research model, which means scientists can be more confident in their experiments.
MC: Why did you prePrevious methods to generate human iPSC-derived cells may have been insufficient or had limitations?
FPS: The ability to generate iPSC-derived human cells has been revolutionary for the scientific industry, offering a physiologically relevant model system and theoretically providing an infinite and renewable source of human cells for research, drug discovery and cell therapy. However, current methods to generate cells from human iPSCs (directed differentiation and cell reprogramming) can be hampered by long, complex protocols and lack reproducibility and scalability.
Inconsistencies and inefficiencies in current cell generation methods mean that scientists struggle with long experimental lead times and invariably struggle with variance in their datasets.
MC: How does the bit.bio platform and products enable reproducible scientific research?
FPS: In laboratories around the world, there is no standardized way to procure and experiment on human cells. This means that conditions vary greatly between academic institutions, biopharmaceutical companies and countries, and therefore results may be difficult to replicate under different conditions. Cells in different conditions, derived in different ways, can produce wildly different results, which impacts our scientific understanding of basic biological processes and can also impact pharmaceutical preclinical trials. This can lead to the publication of erroneous or inaccurate scientific data, even in the most respected scientific journals.
With cells derived from human iPSCs powered by opti-ox, we can create a universal reference standard for future biological research. Other fields of science are unified by common units, whether ‘inches’ or ‘millimeters’, and these can be easily converted from one to the other. By applying engineering principles to biology, we can create the equivalent of the “millimeter” for research in the life sciences, a universal metric by which all research can be judged.
MC: Can you discuss the applications of bit.bio cells and how they are used by customers working in particular in drug discovery and cell therapy?
FPS: bit.bio cells are already used by leading biopharmaceutical companies around the world to accelerate their research, which will help develop the treatments of tomorrow. We recently launched our ioDisease Model cell portfolio, having announced our first product – a model of Huntington’s disease – last month. By combining genetic engineering technology like CRISPR in combination with our opti-ox technology, we have been able to grow not only wild-type human cells, but also those that express the specific genetic aberrations that lead to neurodegenerative and muscular conditions. With these cells, researchers have the best tools to target debilitating and sometimes fatal diseases, providing deeper insights at the preclinical stage that have the potential to accelerate drug discovery and development over the next few years. This is just the first in our pipeline of disease models, many of which will be deployed in 2022.
The ability to produce large quantities of specific human cells also opens up enormous possibilities for cell therapies. Cell therapies involve transfusing or transplanting human cells into a patient to repair damaged tissue or boost bodily functions like immunity. Previously, cell therapies were tailor-made and expensive, tailoring individual treatments to individual patients, which takes up a lot of time and resources. A prefabricated and preprogrammed treatment of human cells democratizes access to this new generation of drugs, making it accessible to a greater number of people at affordable costs.
MC: What is bit.bio’s vision for the laboratory of the future, and how do its products and services align with it?
Furqan Iqbal (FI): It’s all about data – the future begins with operating a single platform that will provide access to all available data assets. Adopting Laboratory Information Management System (LIMS) will improve laboratory productivity and efficiency by tracking data associated with samples, experiments, workflow and instruments. A single source of truth that will enable reliable, detailed on-demand research that connects colleagues, equipment, consumables and data.
Data acquisition will be further enhanced through direct integration with labware, minimizing reliance on manual data transfer. With the huge volumes of data expected, our lab of the future will introduce advanced analytics and modalities to generate actionable insights, enabling us to make data-driven business decisions. Our lunar goal is to leverage our discovery platform and opti-ox for the production of every type of human cell. If we achieve this, access to accurate and physiologically relevant cellular models will no longer represent a bottleneck in therapeutic discovery and disease research.
Farah Patell-Socha and Furqan Iqbal were talking to Molly Campbell, Senior Science Writer for Technology Networks.