bit.bio Adds Two New Human Cell Products to Address the Translation Gap and Accelerate Research and Drug Discovery for Neurodegenerative Disease

● Cell coding company bit.bio announces an expansion to its product portfolio to accelerate research into neurodegenerative diseases.

● Frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) disease model offer a scalable and reproducible in vitro system to model neurodegenerative disease.

● Early access now available to microglial cells, which can provide the basis for research into conditions affecting the central nervous system.

Cell coding company bit.bio has announced an expansion to its product portfolio - ioGlutamatergic Neurons TDP-43^M337V disease model and early access to its ioMicroglia cell product.

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vials of cells and cell image of ioGlutamatergic Neurons TDP-43M337V (Photo: Business Wire)

Despite considerable research efforts and funding, the development of therapies for devastating diseases like Alzheimer’s disease (AD), Frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) has been challenging. Due to a lack of standardised, easy to use and readily accessible human cell models, scientists have relied on animal models and cell lines that differ considerably from human biology.

bit.bio’s latest cell products provide a scalable source of human cells and will enable scientists to study neurodegenerative diseases in a human context. With consistency across batches and a scalable supply, bit.bio’s products will significantly reduce experimental variability in non-clinical studies and improve the translatability and reproducibility of research findings. These unique product features have the potential to transform research and drug discovery.

Dr Mark Kotter, CEO and Founder of bit.bio said:

“The products we are announcing today address an area of high unmet clinical need where high failure rates in drug development are common and no effective treatments exist. I look forward to seeing how our customers will use them to develop new insights and treatments for these devastating conditions.

“This is another step towards our vision of an exciting future in which precision reprogrammed human cells will accelerate biomedical innovation and a new generation of cures. The launch of two new cell products for research and drug discovery in neurodegenerative diseases validates our cell identity coding platform’s ability to create and manufacture any human cell type consistently at scale.”

Today, AD and FTD, the leading causes of early onset dementia, have no treatment options to stop or slow their onset. Similarly, current treatment options for ALS, the most common motor neuron degenerative disease, are limited. ioGlutamatergic Neurons TDP-43^M337V, have a mutation in the TAR DNA binding protein gene that codes for the TDP-43 protein, which is known to cause both FTD and ALS. The disease model cells and the genetically matched control, ioGlutamatergic Neurons, mature rapidly, are highly reproducible between batches, and have unprecedented scalability. These key features make them ideally suited to high-throughput screening applications for early drug discovery. Being able to compare data from the physiologically-relevant disease models to those of the control offers the potential to identify and investigate the effects of the genetic mutation on the disease mechanisms of FTD and ALS.

Increasing evidence suggests that microglia contribute to the onset and progression of AD and are involved in the pathogenesis of ALS and FTD. Therefore they may represent an additional therapeutic target. However, speed, variability, and scalability continue to be major challenges with commonly used microglia. ioMicroglia, now available as part of an early access program, address these challenges allowing scientists to work with consistent, functional cells that are ready for experimentation within just 10 days.

Dr Farah Patell-Socha, VP Research Products at bit.bio, said:

“Human cells are key to disease research, drug discovery, and clinical translation. However, traditional methods of producing human cells have long, laborious, protocols that often result in heterogenous cell populations and can lead to data variability. Our latest products provide robust, standardised tools for neurodegenerative research and drug discovery, paving the way for high-throughput screening and drug target validation in human iPSC-derived models that was previously impossible, and bringing huge benefits to medicine as a result.”

The latest disease models are now available to order on the bit.bio website

Scientists can register interest in early access ioMicroglia vials, and find full physiological data on the bit.bio website

Notes to editors

About bit.bio

bit.bio is a synthetic biology company providing human cells for research, drug discovery and cell therapy. It has released six ioCells research products into the market and is currently building out its clinical pipeline.

bit.bio’s foundational opti-ox^TM precision reprogramming technology enables highly consistent and scalable manufacture of human cells. The activation of cell type-defining transcription factor programs inserted into genomic safe harbour sites allows deterministic reprogramming of induced pluripotent stem cells (iPSC) into highly defined and mature human cells. bit.bio’s discovery platform is based on a deep synthetic biology tech stack and uses large scale experimentation and machine learning to identify combinations of transcription factor genes that encode cell identity.

bit.bio has assembled a team of pioneers with world-leading expertise in stem cell and synthetic biology, manufacturing, and clinical translation. The board is chaired by serial entrepreneur Dr Hermann Hauser and includes Sir Gregory Winter (Nobel Prize for Medicine) and biotech veteran Alan Roemer (co-founder Roivant and Pharmasset).

The company was spun out of the University of Cambridge in 2016. It is at Series B stage and has raised a total of $200M capital from Arch Ventures, Foresite Capital, Milky Way, Charles River Laboratories, National Resilience, Tencent, and Puhua Capital, and others.

For more information visit bit.bio

About ioMicroglia

ioMicroglia are iPSC-derived (induced pluripotent stem cell) immune cells, which will boost research into the physiology and diseases of the central nervous system, such as Alzheimer’s disease, Parkinson’s disease and ALS.

ioMicroglia joins a growing portfolio of stem-cell derived human cells available from bit.bio, with the goal of empowering biomedical innovation towards a new generation of cures through precision reprogramming of human iPSCs. Microglia are immune cells which act as the first line of defence for the central nervous system, from both external and internal threats, and are a key research target when investigating immunological mechanisms in the brain. They are also key in supporting drug discovery into the modulation of microglia activation which can be used as a potential preventative strategy for Alzheimer’s disease.

The key advantage of ioMicroglia is that it addresses two of the major challenges encountered with commonly used human iPSC-derived microglia production protocols: namely speed and variability. ioMicroglia allows scientists to work with functional microglia that are ready for experimentation within 10 days versus 8-10 weeks with classical differentiation protocols. Moreover, ioMicroglia show high batch-to-batch consistency in morphology, key marker expression and functionality, in contrast to classical differentiation protocols.

ioMicroglia show key functional and phenotypic traits in culture including expression of TMEM119, P2RY12, and IBA1, and the ability to phagocytose invasive microorganisms, apoptotic cells or amyloid beta peptides associated with Alzheimer’s disease. Inflammatory properties are also maintained in bit.bio’s cells, further widening the potential applications of their use in translatable laboratory research. The ioMicroglia cells can also be co-cultured with bit.bio’s ioGlutamatergic Neurons whilst retaining microglial functionality, in order to recreate physiological conditions present in the central nervous system.

The new ioMicroglia cells are produced using bit.bio’s opti-ox™ platform which has once again proved its effectiveness in manufacturing physiologically relevant, consistent and reproducible cells at scale in a matter of days. Alongside bit.bio’s existing catalogue of cell types, ioMicroglia are a hugely useful resource for researchers investigating neurodegenerative disease.

About the TDP-43 disease models

Following the launch of its Huntington’s disease model earlier in 2022, ioGlutamatergic Neurons TDP-43 M337V/WT and ioGlutamatergic Neurons TDP-43 M337V/M337V are the second and third products in the company’s ioDisease Model portfolio. The ioDisease Model cells take advantage of precision cell reprogramming and CRISPR/Cas9 genetic engineering, to produce highly characterised human induced pluripotent stem cell (hiPSCs)-derived excitatory neurons with disease-relevant mutations..

Frontotemporal dementia (FTD) is the second leading cause of early onset dementia following Alzheimer's disease. It involves atrophy of the frontal and temporal regions of the brain affecting language, memory, and behaviour. Amyotrophic lateral sclerosis (ALS) is the most common motor neuron degenerative disease and is characterised by progressive degeneration of both upper and lower motor neurons.

The disease models are engineered with a disease-relevant M337V mutation in the TARDBP gene, which codes for the TAR DNA-binding protein 43 (TDP-43). This mutation in the TDP-43 protein promotes cytoplasmic mislocalisation and aggregation, which is implicated in the pathology of ALS and FTD. These disease models offer a fast and easy-to-use system for investigations into the impact of gene function on disease progression. One of the key advantages is the availability of a genetically matched control, which enables researchers to make true comparisons as they can attribute observed differences to the single genetic modification.

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