bit.bio Announces First Project Within Multi-Year Collaboration Agreement with The Michael J. Fox Foundation (MJFF) to Generate Human Cell Products for Research and Drug Discovery in Parkinson’s disease

• First project will prioritise the discovery and development of a key human cell type for Parkinson’s disease (PD) research and drug discovery.
• Collaboration agreement allows MJFF to fund specific projects under which bit.bio will develop a range of human iPSC-derived cell products for PD.
• Products created under the collaboration agreement will offer researchers a physiologically-relevant and defined source of human cells for drug screening and disease modelling, and to further the fundamental understanding of PD pathology.
• The collaboration with MJFF leverages both bit.bio’s discovery platform to identify transcription factor (TF) combinations and bit.bio’s opti-ox™ technology for the deterministic reprogramming of iPSCs into mature, functional human cells.

CAMBRIDGE, England--(BUSINESS WIRE)-- bit.bio, the company coding human cells for novel cures, today announces the first project within a multi-year collaboration agreement with The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to prioritise the development and delivery of a range of human cell products relevant to Parkinson’s disease (PD).

Under the terms of the collaboration agreement, both wild type human cells and physiologically relevant disease model cells can be generated through individual project agreements. Funding to bit.bio by MJFF will be allocated on a project-by-project basis. The first project has been agreed and will prioritise the discovery of TF combinations for a relevant human cell type in PD and the development of that cell type into a product for the PD research community.

PD occurs when dopaminergic neurons stop working or die. PD is a lifelong and progressive disease, and beyond movement disorders such as tremor, slowness, stiffness and balance problems, it can cause depression and memory loss. PD affects 1–2% of the population over the age of 65 and it is estimated that cases will exceed 12 million individuals in 2040.¹ It is a complex and heterogeneous disease affected by multiple genetic and environmental factors.

Currently, Parkinson’s disease researchers mostly rely on animal models and human iPSC-derived dopaminergic neurons, generated via directed differentiation protocols. These protocols present significant drawbacks as they are lengthy, difficult to reproduce and yield heterogeneous populations. Having access to a consistent and scalable source of physiologically relevant human cell models will help researchers understand and pinpoint the diverse pathways that lead to loss of dopaminergic neurons, and gain essential insights into specific cellular and molecular mechanisms involved in disease pathogenesis.

Nicole K. Polinski, PhD, Director of Research Resources, MJFF said:

“We are thrilled to partner with bit.bio and to leverage the company’s unique platform for the generation of human cells. Parkinson’s disease researchers urgently need accessible and well-characterised tools to improve our understanding of PD, and to help deliver therapeutic breakthroughs. The consistency and scalability of bit.bio’s human cell products will provide a very important addition to the suite of tools we know to be necessary.”

All bit.bio ioCells^TM products, including those to be developed under the partnership with MJFF, are precision reprogrammed using bit.bio’s opti-ox technology. As such they are defined human cells that have inherent experimental scalability, offer unparalleled biological consistency and are simple to handle and culture.

Mark Kotter, CEO, bit.bio said:

“This important collaboration with MJFF gives us the opportunity to accelerate the development and delivery of a series of human cell types and associated disease models to the PD research community. The work that MJFF has done to advance the understanding and treatment of PD has been groundbreaking. The team here at bit.bio is already working to deliver the first cell product under the collaboration framework and we are delighted to join with MJFF on this essential path.”

Notes to Editors

References

1. https://www.who.int/news-room/fact-sheets/detail/parkinson-disease

About bit.bio

bit.bio is a synthetic biology company focused on human cells that is advancing medicine (UN SDG9) and enabling curative treatments (UN SDG3). The company does this by industrialising the manufacture of human cells and making them more accessible. The company was spun out of the University of Cambridge in 2016 and has since raised approximately $200m from investors such as Arch Venture, Foresite Capital, Milky Way, Charles River Laboratories, National Resilience, Tencent, Verition Fund and Puhua Capital.

bit.bio’s opti-ox™ precision cell programming and manufacturing technology enables conversion of induced pluripotent stem cells (iPSCs) into any desired human cell type in a single step. This can be achieved within days and at industrial scale, while maintaining exceptional purity and unparalleled consistency.

Our discovery platform extends this approach to any desired cell type by identifying the transcription factor combinations that define cell states (including identity, cell subtype identity, maturity) using high throughput screens and advanced data analysis. We believe that opti-ox can revolutionise regenerative medicine similarly to how CRISPR is unlocking gene therapy.

bit.bio’s cell therapy pipeline, based on txCells™, is focused on serious diseases that lack effective treatments. Our current therapeutic development areas include metabolism and endocrinology, immunology and neurology. Our lead candidate, bbHEP01 based on txHepatocytes, is in development as a treatment for patients suffering from acute liver failure (ALF) and acute-on-chronic liver failure (ACLF). Complementing our internal pipeline, we have a collaboration with BlueRock Therapeutics (a wholly owned independently operated subsidiary of Bayer AG) focused on regulatory T cell (Treg) based cell therapies.

In addition, our extensive ioCells™ research cell product portfolio, which includes ioWild Type Cells™, ioDisease Model Cells™ and ioCRISPR-Ready Cells™, is opening up new possibilities for studying human biology and developing new medicines in both research and high-throughput and high-content drug discovery.

For more information, please visit www.bit.bio

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