bit.bio Wins Landmark Patent Defence for Groundbreaking opti-ox™ Cell Programming Technology

• The European Patent Office (EPO) upholds bit.bio's patent for its dual genomic safe harbour technology, opti-ox™, securing the broadest possible claims for programming of pluripotent stem cells (PSCs) into any cell type. Much like how CRISPR transformed genome editing, opti-ox establishes a new paradigm for the safe, consistent, and scalable manufacture of iPSC-derived cells.
• The successful EPO ruling follows the granting of patents in the U.S., China, and South Korea, with additional applications pending in key markets, including Australia and Japan. This comprehensive global patent coverage solidifies bit.bio’s leadership position for the manufacture of iPSC-derived cells via transcription factor (TF) mediated forward programming.
• bit.bio is creating highly differentiated human cell products for research, drug discovery, and cell-based therapies, with the intention of addressing critical unmet needs in human health. opti-ox holds transformative potential for reducing cell therapy costs of goods sold by an estimated two orders of magnitude.

bit.bio, the company coding human cells for novel cures and a pioneer in synthetic biology, today announces the successful defence of its foundational European patent for opti-ox, a breakthrough technology enabling deterministic forward programming of pluripotent stem cells (PSCs), including iPSCs.

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Following an anonymous opposition filed on 5 January 2023, the European Patent Office (EPO) upheld bit.bio's European Patent (EP3545079), securing the broadest possible claims and protections in the field of transcription factor mediated forward programming of PSCs. Following a public hearing on 25 September 2024, the EPO’s Opposition Division confirmed the patent's validity after a rigorous scientific and legal review, maintaining its broad claims and affirming its novelty and inventive step.

The EPO ruling reinforces bit.bio's position as the global market leader in scalable and consistent iPSC-derived cell manufacturing and demonstrates the strength of its intellectual property portfolio. This decision builds on three key patents granted in 2023 across major markets, including United States Patent 11697823, Chinese Patent CN110249045 and South Korean Patent KR102500322. With patents now secured in four major markets and applications pending in countries including Australia and Japan, bit.bio is strategically positioned to lead the next wave of innovation in iPSC-derived cells for R&D applications and cell-based therapies.

"This successful defence of our European patent is a significant milestone for bit.bio,” said Mark Kotter, CEO of bit.bio. “Our unique ability to conduct multiplexed screens of over 2,300 transcription factors, combined with our AI platforms that rapidly predict and read out transcription factor combinations that define cell types, is driving the expansion of our cell type portfolio and IP. Our team’s dedication and expertise were instrumental in firmly establishing bit.bio as the global leader in cell programming technology.”

bit.bio’s opti-ox technology enables a paradigm shift in the manufacture of human cells from pluripotent stem cells, establishing iPSC-derived cell therapies as an emerging modality in medicine, much like the transformative impact CRISPR had on genome editing. Using a dual genomic safe harbour (GSH) approach, opti-ox deterministically controls gene expression, allowing for the rapid and consistent conversion of induced pluripotent stem cells (iPSCs) into specific human cell types at an industrial scale. The achieved consistency and scalability is unprecedented and is critical to industrialise human cells for both research and therapeutic applications in a cost-effective manner.

"opti-ox has the potential to revolutionise research and cell therapies on a global scale,” said Thomas Südhof, Nobel Laureate, Director of Stanford University School of Medicine's Center for Molecular Neuroscience in Health and Disease, and a pioneer in TF-mediated cell programming. “The opti-ox dual genomic safe harbour approach is currently the only way to ensure safe, consistent, and scalable forward programming of PSCs, because, unexpectedly, one GSH is not sufficient. We are only at the beginning of understanding the transformational potential of this new paradigm. In contrast to the general expectations around biology, it provides a deterministic approach for controlling cell fate.”

bit.bio's opti-ox technology is already playing a crucial role in providing off-the-shelf human cell models at scale for a variety of diseases, including Alzheimer's and Parkinson's. The company's ioCells^TM for research and drug discovery are recognised for their functionality, consistency, and scalability.

According to stem cell research firm BioInformant, the global market for iPSC-derived biomedical research tools is currently valued at approximately $1.3 billion, though it remains in its early stages, limited by product breadth and quality. bit.bio projects the addressable market for its ioCells to exceed $10 billion, considering significant opportunities in the $3 billion primary cells market (Precision Business Insights, Zion Market Research), the $25 billion high throughput screening market (Global Market Insights, Grand View Research, Markets and Markets), and the $25 billion in vitro toxicology market (Allied Market Research, Global Market Insights, Grand View Research, Markets and Markets, Precedence Research).

“Being at the forefront of cell programming with IP that protects indispensable technology gives bit.bio an opportunity to create a truly transformative business,” said Hermann Hauser, Chairman of the Board of bit.bio and co-founder of Arm and Amadeus Capital Partners. “The current market size for R&D grade cells is valued in the billions, but our opportunity reaches far beyond that. Given the transformational potential of cell therapies, the market for iPSC-derived therapeutics is likely to exceed that of biologics. This opens up unprecedented opportunities for growth and innovation in personalised medicine and drug discovery."

About bit.bio

bit.bio, a synthetic biology company, is democratising human cells to advance biomedical research (SDG9) and enable transformative treatments (SDG3). With Nobel Prize-winning science underpinning our understanding of cell identity as defined by unique combinations of transcription factors, our deterministic cell programming technology, opti-ox, enables conversion of induced pluripotent stem cells (iPSCs) into any desired human cell type in a single step. This is achieved within days and at industrial scale, while maintaining exceptional purity and consistency.

Since spinning out from the University of Cambridge and launching in 2016, we have raised approximately $200m from leading investors, including Arch Venture, Blueyard Capital, Charles River Laboratories, Foresite Capital, Milky Way, National Resilience, and Tencent.

For more information, please visit: www.bit.bio

For information on bit.bio’s trademarks, visit www.bit.bio/trademarks

LinkedIn: https://www.linkedin.com/company/bitbioltd/

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