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How iPSCs Are Used in ALS Research: Insights from TDP-43 and SOD1 Models

kfaulkner9a16998ec4 — Tue, 21 Jul 2026 14:32:51 +0000

How iPSCs Are Used in ALS Research: Insights from TDP-43 and SOD1 Models

Induced pluripotent stem cells (iPSCs) are helping researchers study amyotrophic lateral sclerosis (ALS) in more human-relevant, patient-specific ways. By differentiating patient-derived iPSCs into motor neurons and other relevant cell types, scientists can investigate disease-associated phenotypes, examine mutation-specific effects, and build experimental models for early therapeutic research. In this article, we look at how iPSC models have advanced ALS research through studies involving TDP-43- and SOD1-associated disease biology.

Post Summary

What are iPSCs used for in ALS research?
iPSCs are used to generate patient-specific motor neuron models that help researchers study ALS-associated pathology, investigate disease phenotypes, and test mechanistic hypotheses in human cells.²˒³

Why are iPSCs valuable for ALS research?
They allow scientists to examine disease-relevant phenotypes in the cell types mostly affected in ALS, while preserving the patient’s genetic background.¹˒²˒³

What have iPSC models revealed about ALS?
Studies using iPSC-derived motor neurons have helped researchers investigate disease-associated phenotypes linked to TDP-43 and SOD1, including mutation-specific effects and broader cellular dysfunction relevant to overall neurodegeneration.²˒³

Why do these models matter for translational research?
iPSC-based ALS models provide a human-relevant framework for mechanistic research and early therapeutic evaluation, though findings still need to be supported by broader preclinical and clinical validation.¹˒³

ALS is a progressive neurodegenerative disease that affects upper and lower motor neurons, leading to worsening muscle weakness, loss of function, and ultimately respiratory failure. Although the clinical picture of ALS has been studied for decades, understanding its biology at the cellular level remains challenging. One reason is simple: the cell type most affected in ALS, the motor neuron, is not easily available for direct study in living patients.¹

Induced pluripotent stem cells, or iPSCs, have become an important tool for addressing that challenge. By reprogramming adult patient cells into a pluripotent state and differentiating them into motor neurons, researchers can study ALS-associated cellular changes in a more disease-relevant human system.²˒³

In this article, we explore how iPSCs are being used in ALS research, what these models have revealed about disease biology, and why they continue to matter in translational neuroscience.

The Role of iPSCs in ALS Research

ALS is not a single-pathway disease. It involves a complex mix of genetic, cellular and enviromental factors. Some of the cellular processes affected by ALS include RNA processing, protein mislocalization, oxidative stress, mitochondrial dysfunction, disrupted axonal transport, and endoplasmic reticulum stress.¹˒³ This complexity exacerbates the need for a disease model that can faithfully recapitulate human biology.

iPSC technology gives researchers a way to model ALS using patient-derived cells that retain disease-associated genetic context. Once differentiated into motor neurons, these cells can be used to investigate how specific mutations contribute to ALS progression.²˒³

This has been especially useful in studies of genetically defined (familial) ALS. Rather than relying on in silico or animal models, researchers can examine cellular phenotypes in iPSC-derived motor neurons derived from patients with identified ALS risk variants. That makes it easier to ask targeted questions about mechanism, compare mutant and control lines, and build more informative experimental workflows.²˒³

Just as importantly, iPSC-derived ALS models help bridge discovery and translational research. They provide a controlled framework for examining disease-associated biology in vitro and, in some cases, for evaluating whether potential interventions influence those phenotypes.²˒³

What iPSC Models Have Revealed About ALS Biology

One of the major strengths of human iPSC-derived ALS models is that they more faithfully recapitulate selected features of disease biology compared to animal models. In iPSC-derived motor neurons, these features may include altered protein localization, cellular stress responses, transcriptional changes, or increased neuronal vulnerability.²˒³

This does not mean that every iPSC-derived model captures the full complexity of ALS. But these systems can reveal biologically meaningful aspects of disease that are impossible to study in living patients. In that sense, their value lies not in reproducing every dimension of ALS, but in allowing researchers to isolate and study specific mechanisms in a human cellular context.¹˒²˒³

In ALS research, two especially important discoveries involve TDP-43- and SOD1-associated pathologies. Together, these examples show how iPSC-derived motor neurons can support both mechanistic insights and early-stage therapeutic investigation.²˒³

Case Study 1: TDP-43-Associated Pathology in ALS

Abnormal TDP-43 function is one of the most widely recognized pathological features in ALS. In many forms of the disease, TDP-43 shifts from its normal nuclear role and becomes abnormally localized in the cytoplasm, where it is associated with pathological inclusions and broader cellular dysfunction.¹˒²

To study this process in a more disease-relevant system, researchers have generated iPSCs from ALS patients and differentiated them into motor neurons. In the Bilican et al. study, patient-derived lines recapitulated aspects of TDP-43 proteinopathy and revealed cell-type-specific vulnerability, helping demonstrate that human iPSC-derived motor neurons could capture meaningful disease-associated phenotypes in vitro.²

The significance of this kind of model is not only that it reflects a known feature of ALS pathology. It is that it gives researchers a way to examine how TDP-43-associated dysfunction may influence motor neuron health in a human system that is experimentally accessible. That can support studies of neuronal vulnerability, disease-associated stress responses, and mutation-linked cellular effects.²

These models are also useful because they create a platform for downstream research. Once a disease-relevant phenotype is established in human motor neurons, researchers can use that system to ask more refined mechanistic questions and, where appropriate, explore whether experimental interventions affect the phenotype.²

Case Study 2: SOD1-Mutant ALS and Mutation-Specific Modeling

Mutations in the SOD1 gene are associated with certain familial forms of ALS and have been studied extensively in both animal and human-based systems. Using iPSC-derived approaches, SOD1-mutant motor neuron models have helped researchers investigate how a defined genetic mutation can disrupt cellular pathways linked to neurodegeneration.³

The Kiskinis et al. study is especially important because it paired patient-derived SOD1-mutant motor neurons with genetically corrected isogenic controls. That design helped researchers identify transcriptional and functional changes tied more directly to the SOD1 mutation itself, including signatures linked to oxidative stress, mitochondrial dysfunction, altered subcellular transport, and ER stress..³

This type of study illustrates one of the most powerful uses of iPSC technology in disease research: the ability to compare highly similar cell lines that differ only in a singular genetic locus. That helps reduce background noise and strengthens interpretation when researchers are trying to connect genotype to phenotype.³

For ALS research, that matters because it moves the field beyond broad disease description and toward more precise questions about mechanism. SOD1-mutant iPSC-derived motor neuron models have therefore become an important tool for understanding how specific mutations influence cellular behavior and for building more targeted experimental systems for therapeutic research.³

Why These Models Matter in ALS Research

Taken together, TDP-43- and SOD1-based studies show why iPSCs have become so valuable in ALS research. They allow investigators to study disease-associated phenotypes in human motor neurons, preserve meaningful aspects of patient genetic background, and building models tailored to answer specific biological questions.²˒³

These systems are especially useful in a disease like ALS, where direct access to affected neural tissue is limited and the underlying biology is highly heterogeneous. iPSC-derived models do not eliminate that complexity, but they do provide a more flexible and human-relevant platform for studying it.¹˒²˒³

They also fit well within a broader translational workflow. iPSC-based ALS models can support target discovery, mechanistic investigation, and early therapeutic testing, helping researchers generate data that may guide more advanced preclinical work. At the same time, findings from these models still need to be interpreted carefully and validated through additional systems and, ultimately, clinical research.¹˒³

Looking Ahead

As iPSC workflows continue to improve, their role in ALS research will likely keep expanding. Advances in differentiation, genome editing, assay design, and phenotypic analysis are making it easier to build more refined motor neuron models and ask more precise questions about disease biology.²˒³

For ALS researchers, that progress is important because the disease is biologically complex and clinically heterogeneous. Tools that help clarify mechanism in a patient-specific, human-relevant framework can contribute meaningfully to both discovery and translational science.¹˒³

While iPSCs are not a complete answer to every challenge in ALS research, they have clearly strengthened the field’s ability to study disease-associated cellular dysfunction in vitro. That makes them an increasingly important part of the research toolkit for understanding ALS and evaluating new therapeutic ideas.²˒³

Conclusion

iPSCs have become an important tool in ALS research by enabling patient-specific motor neuron models that support the study of disease-associated phenotypes in human cells. Through work involving TDP-43- and SOD1-associated pathology, these models have helped researchers investigate cellular mechanisms, examine mutation-specific effects, and strengthen early translational research.²˒³

Their value lies in their ability to bring greater biological relevance to preclinical studies. By giving scientists access to human motor neurons derived from patients, iPSC-based models help connect clinical genetics to cellular phenotype in ways that can inform both mechanistic understanding and therapeutic exploration.²˒³

As ALS research continues to evolve, iPSCs are likely to remain an important platform for studying how neurodegeneration unfolds and how more targeted research strategies can be built around human-relevant disease models.¹˒²˒³

Contributing Author:

Jorge Urresti, PhD
Scientific Lead
iXCells Biotechnologies

References

Masrori, P., & Van Damme, P. (2020). Amyotrophic lateral sclerosis: a clinical review. European Journal of Neurology, 27(10), 1918-1929.
Bilican, B., Serio, A., Barmada, S. J., Nishimura, A. L., Sullivan, G. J., Carrasco, M., … & Chandran, S. (2012). Mutant induced pluripotent stem cell lines recapitulate aspects of TDP-43 proteinopathies and reveal cell-specific vulnerability. Proceedings of the National Academy of Sciences, 109(15), 5803-5808.
Kiskinis, E., Sandoe, J., Williams, L. A., Boulting, G. L., Moccia, R., Wainger, B. J., … & Eggan, K. (2014). Pathways disrupted in human ALS motor neurons identified through genetic correction of mutant SOD1. Cell Stem Cell, 14(6), 781-795.

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With over a decade of experience in cellular biology and preclinical research, iXCells delivers human-relevant models that help researchers improve predictability and move new therapies forward.

The post How iPSCs Are Used in ALS Research: Insights from TDP-43 and SOD1 Models appeared first on iXCells.

NAM-Only INDs Are Moving From Theory to Strategy

kfaulkner9a16998ec4 — Mon, 06 Jul 2026 16:00:25 +0000

NAM-Only INDs Are Moving From Theory to Strategy

A recent LinkedIn article by Stefano Gaburro, PhD, raises an important question for drug developers: under what conditions could a NAM-supported, or even NAM-only, IND be accepted?

The answer is not as simple as “animal studies are no longer needed.” The more useful takeaway is this: regulatory expectations are changing, and human-relevant models are becoming a more strategic part of early development planning.

For sponsors working with modalities that already have established safety knowledge, this shift could be especially meaningful.

The opportunity is in the evidence gap

Many development programs are not starting from zero. A sponsor may be optimizing an AAV capsid, modifying a known therapeutic platform, evaluating a structurally related compound, or developing a combination therapy where the individual components already have established safety profiles.

In these cases, the key regulatory question may not be: “Can we prove everything again from the beginning?”

It may be: “Can we show that this modification, combination, or platform application does not introduce a new safety signal?”

That is where NAMs can become highly valuable.

As Christy Holt, VP of Marketing and Sales at iXCells, notes, combination therapies are a strong example. When individual components already have established safety profiles, NAM data showing that the combination does not create new safety concerns may help support an IND strategy without requiring a full set of new animal studies.

The same logic may apply to other platform-based approaches. For example, AAV capsid optimization or other modality improvements may be supported by existing class or platform knowledge, while human biological models help evaluate what has changed.

Human-relevant models can help sponsors move faster

For development teams, the impact could be significant.

A stronger NAM package may help shorten development timelines, reduce cost, and support faster IND acceptance when the data are scientifically justified and aligned with regulatory expectations.

This is not about replacing scientific rigor. It is about generating more relevant evidence earlier.

Human iPSC-derived models, organoids, gene-edited disease models, and other advanced in vitro systems can help sponsors evaluate biology in a context that is closer to the patient. These models can be used to assess mechanism, target engagement, toxicity risk, off-target effects, and therapeutic response before advancing into more expensive and time-sensitive stages of development.

For programs where prior animal and human safety data already exist, NAMs can help fill specific gaps rather than repeat work that may not add meaningful value.

The next step is strategic model selection

The regulatory path is still case-specific. NAM data must be matched to the right context of use, the right biological question, and the right endpoint. A model that is useful for one safety question may not be appropriate for another.

That makes model selection critical.

Drug developers need human-relevant systems that are well characterized, reproducible, and aligned with the scientific question they are trying to answer. For some programs, that may mean scalable iPSC-derived cell models. For others, it may mean CRISPR-edited isogenic controls, patient-derived disease models, organoids, or assay-ready cells designed for screening and translational studies.

The opportunity is not simply to use NAMs. It is to use the right NAMs for the right question.

A practical shift for preclinical development

The movement toward NAM-supported INDs represents a practical shift in how development teams can think about preclinical evidence.

For platform technologies, optimized modalities, and combination therapies, sponsors may have an opportunity to build a more focused data package around human biology, existing safety knowledge, and clearly defined risk questions.

That could mean shorter development timelines, lower costs, and a more efficient path toward IND-enabling decisions.

At iXCells, we see this as an important moment for human-relevant preclinical research. As regulatory frameworks continue to evolve, the ability to generate reliable, disease-relevant human model data will become increasingly valuable for teams working to move therapies forward.

Author:

Christy Holt
VP of Marketing and Sales
iXCells Biotechnologies

References

Stefano Gaburro, PhD — “The NAM-Only IND: Where We Actually Stand in the US and Europe”
LinkedIn, February 2026
U.S. Food & Drug Administration — “FDA Announces Plan to Phase Out Animal Testing Requirement for Monoclonal Antibodies and Other Drugs”
FDA Press Announcement, April 10, 2025
U.S. Food & Drug Administration — “Roadmap to Reducing Animal Testing in Preclinical Safety Studies”
FDA Scientific Roadmap, April 2025
U.S. Food & Drug Administration — “General Considerations for the Use of New Approach Methodologies in Drug Development”
FDA Draft Guidance for Industry, March 2026
U.S. Food & Drug Administration — “New Approach Methodologies (NAMs)”
FDA Science & Research Topic Page
European Medicines Agency — “Regulatory Acceptance of New Approach Methodologies (NAMs) to Reduce Animal Use in Testing”
EMA Regulatory Guidance Page

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With over a decade of experience in cellular biology and preclinical research, iXCells delivers human-relevant models that help researchers improve predictability and move new therapies forward.

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Case Study Highlights: Targeted Applications of iPSC Models in Rare Disease Research

kfaulkner9a16998ec4 — Thu, 11 Jun 2026 15:58:00 +0000

Case Study Highlights: Targeted Applications of iPSC Models in Rare Disease Research

Induced pluripotent stem cell-derived models are helping researchers study rare diseases in more human-relevant, patient-specific ways. In this article, we look at how iPSC-based disease models have been used in ALS and Duchenne muscular dystrophy research to investigate disease mechanisms, study mutation-specific effects, and support early-stage research in vitro.

What are iPSC models used for in rare disease research?

iPSC models are used to create patient-specific cellular systems that help researchers study disease mechanisms, mutation-specific effects, and early research questions in vitro.

Why are iPSC-derived models valuable for rare disease research?

They allow scientists to investigate rare diseases in human cells that more closely reflect the genetic background of disease than many traditional model systems.

What does this article cover?

This article highlights case studies in ALS and Duchenne muscular dystrophy to show how iPSC-derived models support rare disease research, mechanistic studies, and early preclinical investigation.

Rare diseases present some of the most difficult challenges in biomedical research. Many are driven by specific genetic abnormalities, involve complex cellular pathology, and affect tissues that are not easily accessible for direct study. In many cases, researchers also face a lack of representative model systems that fully capture the biology of disease in a human context.

Induced pluripotent stem cells, or iPSCs, have become an important tool in this area of research because they can be used to generate patient-specific disease models in the lab. In this context, the focus is not on using iPSCs as a replacement therapy, but on using iPSC-derived cells as research models that help scientists study rare disease biology in vitro.¹˒²

For researchers working in rare disease biology, this matters for a simple reason: the closer a model comes to reflecting the disease as it appears in human cells, the more useful it can be for studying mechanisms, evaluating hypotheses, and informing early preclinical research.

In this article, we highlight several targeted applications of iPSC-derived models in rare disease research. Through case studies in amyotrophic lateral sclerosis (ALS) and Duchenne muscular dystrophy (DMD), we explore how iPSC-based models have helped researchers investigate disease-associated phenotypes, better understand mechanisms of pathology, and support the evaluation of early research strategies in vitro.¹˒²

What makes iPSC-derived models valuable in rare disease research?

Rare diseases often involve highly specific genetic drivers, which makes patient-derived models especially valuable. iPSC technology allows researchers to start with adult cells collected from a patient, reprogram them into a pluripotent state, and then differentiate them into disease-relevant cell types for in vitro study.¹˒²

This approach offers several important advantages for rare disease research. First, iPSC-derived models provide a more human-relevant system than many traditional animal models, allowing researchers to study disease biology in human cells that retain the patient’s genetic background. This makes it possible to examine how specific mutations or variants may influence cellular behavior in a disease-relevant context.

Second, iPSC-derived models can provide access to cell types that may otherwise be difficult or impossible to obtain directly from patients, such as motor neurons, astrocytes, cardiomyocytes, or muscle cells. This is especially important in rare diseases that affect tissues that are not easily accessible for direct study.

Third, iPSC-derived models are highly scalable. As differentiation, assay design, and automation workflows continue to improve, these models can support larger-scale studies, including high-content imaging and screening approaches used in early drug discovery and development.

iPSC-derived models also create opportunities to study more complex biology through co-culture systems, organ-on-chip platforms, and other multi-cell-type models. Because different disease-relevant cell types can be generated from the same iPSC disease background, researchers can investigate interactions between cells that may contribute to disease progression, including non-cell-autonomous events where one cell type influences the function or survival of another.

Over the past decade, iPSC-derived models have become increasingly important in rare disease research because they support a more direct investigation of pathology in human cells. As with any model system, they have limitations and must be interpreted within the broader context of preclinical research. But for many rare diseases, they offer an important platform for studying biology in a way that is human-relevant, patient-specific, scalable, and experimentally tractable.¹

Case study: Modeling TDP-43 pathology in ALS

Amyotrophic lateral sclerosis is a progressive neurodegenerative disease marked by the loss of motor neurons. In many forms of ALS, one of the most important pathological features is abnormal behavior of the RNA-binding protein TDP-43 in affected cells.³

To better understand how this pathology develops, researchers have generated iPSCs from ALS patients and differentiated them into motor neurons. In one notable study, these patient-derived motor neurons reproduced aspects of TDP-43-associated pathology observed in disease, helping researchers examine cellular vulnerability in a more disease-relevant setting.³

This type of model is significant because it allows investigators to study pathogenic features directly in human motor neurons carrying disease-associated genetic backgrounds. Instead of inferring disease mechanisms solely from nonhuman systems or non-neuronal cell types, researchers can observe how pathology emerges in cells that are more directly connected to the affected tissue.³

The broader value of this work lies in what the model makes possible. iPSC-derived motor neurons can help researchers investigate how TDP-43 dysfunction contributes to neurodegeneration, identify cellular phenotypes associated with disease progression, and establish a platform for evaluating early research hypotheses in vitro.³

For rare neurodegenerative diseases, that kind of human-relevant model can provide a more informative starting point for mechanistic research and early-stage preclinical exploration.

Case study: SOD1-associated ALS, C9orf72 mutation lines, and mutation-specific modeling

A second important application of iPSC-derived models in ALS research involves mutation-specific modeling. In one study, researchers focused on mutations in the SOD1 gene, which are associated with certain familial forms of ALS. The same study also included C9orf72 mutation lines, adding another important genetic context for examining ALS biology.⁴

SOD1 mutations have been linked to motor neuron toxicity and disrupted cellular pathways, making them an important focus in efforts to better understand ALS disease mechanisms. Using iPSCs derived from patients with SOD1 mutations, researchers developed motor neuron models that captured features of disease-associated dysfunction in vitro. These models helped investigators study how SOD1-related pathology affects neuronal health and how specific molecular pathways may contribute to degeneration.⁴

One of the major strengths of this research is the ability to examine mutation-specific effects in a controlled setting. In studies using genetically corrected comparison lines, researchers have been able to distinguish disease-related phenotypes from background variation more clearly. This kind of design strengthens mechanistic interpretation by allowing scientists to connect a specific mutation more directly to an observed cellular outcome.⁴

In practical terms, SOD1 iPSC models have contributed to a better understanding of how disease-associated genetic alterations influence motor neuron biology. The inclusion of C9orf72 mutation lines also underscores the broader value of iPSC-derived models for studying ALS across different genetic backgrounds. Together, these approaches support research into genotype-to-phenotype relationships in a system that is more closely aligned with human disease than many traditional preclinical models.⁴

As iPSC workflows continue to improve, models like these are becoming increasingly useful for studying mutation-specific biology in rare neurodegenerative disorders.

Case study: Duchenne muscular dystrophy and iPSC-based disease modeling

Duchenne muscular dystrophy is a rare genetic disorder caused by mutations in the dystrophin gene. The disease leads to progressive muscle degeneration and has long been a focus of research aimed at understanding how loss of dystrophin affects muscle structure and function.

iPSC technology has provided researchers with a way to model DMD using patient-derived cells that can be differentiated into muscle-relevant cell types. These models make it possible to study disease-associated phenotypes in a human genetic context and to assess how cellular function changes when dystrophin is absent or disrupted.⁵

This has been particularly valuable in research exploring gene-editing strategies. In preclinical studies, investigators have used CRISPR-Cas9-based approaches in hiPSC-derived muscle cells to evaluate whether targeted correction of dystrophin-related mutations can restore key aspects of normal cellular function.⁵

iPSC-derived DMD models have also been used to evaluate other mutation-directed approaches, including exon-skipping strategies and base-editing methods designed to restore or improve dystrophin expression in patient-derived cells.⁶˒⁷

The importance of this work is not only in demonstrating proof of concept, but in showing how iPSC-derived disease models can support therapeutic testing earlier in development. By giving researchers access to patient-specific cellular systems, these models can help refine experimental questions, evaluate editing strategies, and generate data that may inform the next stages of preclinical research.

Why these case studies matter

Taken together, these examples illustrate why iPSC-derived models have become such an important tool in rare disease research. Their value is not simply that they can generate disease-relevant cell types in the lab. It is that they give researchers a way to study pathology in models that retain meaningful aspects of the patient’s genetic context.¹˒²

In ALS, iPSC-derived motor neurons have helped researchers study disease-associated protein pathology and mutation-linked dysfunction in a more targeted way.³˒⁴ In DMD, iPSC-derived muscle models have supported the study of dystrophin-related defects and the evaluation of gene-editing approaches in human cells.⁵

These kinds of applications are especially important in rare disease research, where limited patient access and limited model availability have historically slowed progress. iPSC-derived models do not eliminate those challenges entirely, but they do provide a more flexible and biologically relevant platform for addressing them.¹

Just as importantly, they fit well within a broader translational research workflow. iPSC models can contribute to early mechanistic studies, support target validation, and help researchers evaluate experimental concepts before moving into more advanced stages of development.¹˒²

Looking ahead

The role of iPSC-derived models in rare disease research will likely continue to grow as reprogramming, differentiation, genome editing, and assay design become more refined. As these workflows improve, researchers will have even greater ability to generate disease-relevant models that support more precise and informative studies.¹˒²

For rare diseases in particular, that progress matters. These conditions often require highly tailored approaches to understanding pathology and evaluating potential interventions. iPSC-derived disease models are well suited to that challenge because they enable the development of patient-specific cellular systems that can illuminate disease biology in a more human-relevant framework.¹

While no single model system can answer every question, iPSC-derived models have clearly expanded what is possible in rare disease research. They are helping researchers move closer to models that are not only more personalized, but also more useful for connecting mechanism, phenotype, and research strategy.¹˒²

Conclusion

iPSC-derived models have become a valuable part of rare disease research by enabling patient-specific systems that can capture important aspects of disease biology in vitro. Through applications in ALS, DMD, and other genetically defined disorders, they have helped researchers study pathology more directly, investigate mutation-specific effects, and support early preclinical research.¹˒³˒⁴˒⁵

Their growing importance lies in their ability to bring greater biological relevance to rare disease research. By making it possible to study disease-associated genetic backgrounds in human cells, iPSC models help researchers ask better questions and generate more informative data.¹˒²

As the field continues to evolve, iPSC-derived disease models will remain an important tool for advancing the study of rare diseases and strengthening the connection between early discovery and translational research.¹˒²

Author:

Dr. Xinyu Kong, PhD
Director of iPSC Generation and Gene Editing
iXCells Biotechnologies

References

Shi, Y., Inoue, H., Wu, J. C., & Yamanaka, S. (2017). Induced pluripotent stem cell technology: a decade of progress. Nature Reviews Drug Discovery, 16(2), 115–130.
Takahashi, K., & Yamanaka, S. (2016). A decade of transcription factor-mediated reprogramming to pluripotency. Nature Reviews Molecular Cell Biology, 17(3), 183–193.
Bilican, B., et al. (2012). Mutant induced pluripotent stem cell lines recapitulate aspects of TDP-43 proteinopathies and reveal cell-specific vulnerability. Proceedings of the National Academy of Sciences, 109(15), 5803–5808.
Kiskinis, E., et al. (2014). Pathways disrupted in human ALS motor neurons identified through genetic correction of mutant SOD1. Cell Stem Cell, 14(6), 781–795.
Young, C. S., et al. (2016). A Single CRISPR-Cas9 Deletion Strategy That Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells. Cell Stem Cell, 18(4), 533–540.
Li, H. L., Fujimoto, N., Sasakawa, N., Shirai, S., Ohkame, T., Sakuma, T., Tanaka, M., Amano, N., Watanabe, A., Sakurai, H., Yamamoto, T., & Hotta, A. (2015). Precise correction of the dystrophin gene in duchenne muscular dystrophy patient induced pluripotent stem cells by TALEN and CRISPR-Cas9. Stem Cell Reports, 4(1), 143–154. https://doi.org/10.1016/j.stemcr.2014.10.013
Wang, P., Li, J., Wang, Y., Huang, S., Chen, L., Liu, J., Yang, X., Xu, H., Wang, X., & Wang, Y. (2023). Correction of DMD in human iPSC-derived cardiomyocytes by base-editing-induced exon skipping. Molecular Therapy: Methods & Clinical Development, 28, 40–50. https://doi.org/10.1016/j.omtm.2022.11.010

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Introduction to the Transformative Role of iPSCs in Modeling Complex Diseases

kfaulkner9a16998ec4 — Thu, 14 May 2026 02:30:06 +0000

Introduction to the Transformative Role of iPSCs in Modeling Complex Diseases

Induced pluripotent stem cells, or iPSCs, have reshaped how researchers study human disease. By enabling scientists to generate patient-specific cell models in the lab, iPSCs offer a powerful way to investigate disease biology in systems that are often more relevant than traditional animal or immortalized cell models.

Over the last two decades, iPSC technology has moved from a breakthrough discovery to a foundational tool in disease modeling, drug discovery, and translational research. Because iPSCs can be generated from adult cells and then directed into a wide range of specialized cell types, they give researchers a flexible platform for studying complex diseases in a more human-relevant context.

What are iPSCs?

iPSCs are adult cells that have been reprogrammed into a pluripotent state, allowing them to become many different cell types used in research.

Why are iPSCs important for disease modeling?

They allow scientists to create patient-specific cell models that reflect the genetic background of disease more closely than many traditional systems.

How are iPSCs used in research?

Researchers use iPSCs to study disease mechanisms, compare healthy and disease-associated cell behavior, support drug discovery, and explore gene-editing strategies. In some areas of research, iPSCs are also being investigated as a potential source for cell-based therapies, including cell replacement approaches.

In this article, we explore what iPSCs are, why they have become so important in biomedical research, and how they are helping bridge the gap between early discovery and future therapeutic development.

Understanding iPSCs and Their Versatility

iPSCs were first introduced in 2006 through the pioneering work of Shinya Yamanaka and colleagues, who demonstrated that adult somatic cells could be reprogrammed into a pluripotent state using defined transcription factors. This discovery fundamentally changed stem cell biology by showing that differentiated cells could be reset to a state with the capacity to become many functionally relevant cell types.¹˒²

In practical terms, cells collected from a patient, such as skin fibroblasts or blood cells, can be reprogrammed into iPSCs and then differentiated into disease-relevant cell types for study. Researchers can use these cells to model aspects of neurological, cardiac, muscular, hepatic, and other disorders in vitro. This patient-specific approach has made iPSCs especially valuable for studying diseases that are difficult to access directly in living patients.

What makes iPSCs so impactful is not just their pluripotency, but their relevance. Instead of relying solely on proxy systems, researchers can investigate disease in cells that carry the patient’s own genetic background. That opens the door to studying how specific mutations, risk variants, or cellular pathways contribute to disease onset and progression.

From Theory to Therapy: Why iPSCs Matter in Disease Modeling

One of the defining strengths of iPSC technology is its versatility. Once established, iPSC lines can be differentiated into specialized cells such as neurons, cardiomyocytes, and hepatocyte-like cells, allowing researchers to build models tailored to specific disease areas.³

Just as importantly, iPSCs can reproduce key features of disease biology in vitro. In many cases, they allow researchers to examine pathogenic mechanisms directly in human cells rather than inferring them from nonhuman models.⁴ For complex diseases, this can provide a more precise view of how cellular dysfunction emerges and evolves. In addition, these models are widely used in drug screening and therapeutic discovery, enabling researchers to evaluate compound effects, identify potential treatment strategies, and assess safety in a more human-relevant system.

Rare disease research has been one of the most compelling areas of iPSC application. In amyotrophic lateral sclerosis (ALS), for example, iPSC-derived motor neuron models have been used to study disease-associated phenotypes linked to mutations in genes such as TDP-43 and SOD1. These models have helped researchers investigate pathological protein aggregation, cellular stress, and motor neuron vulnerability in a patient-relevant setting. ⁵˒⁶

iPSCs have also played an important role in Duchenne muscular dystrophy (DMD) research. Patient-derived iPSC models have enabled the study of dystrophin-related dysfunction and supported work exploring gene-editing strategies designed to correct disease-causing mutations. These types of models give researchers an experimentally tractable system for testing therapeutic hypotheses before moving into more advanced development stages.

Together, these examples show why iPSCs are more than a theoretical advance. They have become a practical tool for studying disease biology in ways that are more personalized, mechanistic, and clinically relevant.

Bridging Research and Clinical Application

As iPSC technology has matured, its value has expanded beyond disease modeling alone. Today, iPSCs are increasingly used in combination with advanced genome engineering tools, including CRISPR-Cas9, to build more refined and informative experimental systems.

One major advantage of this combination is the ability to create precise mutation corrections or introduce defined variants into otherwise matched cell lines. This makes it possible to generate isogenic controls—cell lines that differ only at a specific genetic locus—so researchers can isolate the biological effects of a mutation with greater confidence.⁷˒⁸

Researchers can also derive iPSC lines directly from patients whose cells carry disease-associated mutations. Those lines can then be differentiated into target cell types for downstream characterization, pathway analysis, and therapeutic testing.⁹ This workflow helps preserve the genetic context of disease while enabling more controlled experimentation in vitro.

The implications for translational research are significant. iPSC models have contributed to mechanistic insights in areas such as neurodegeneration, cardiac disease, and inherited disorders by helping researchers connect genotype to cellular phenotype.³˒¹⁰˒¹¹ In some settings, iPSC-derived cells are also being used to evaluate drug responses and safety signals in a more human-relevant framework than some traditional preclinical systems.

This does not mean iPSCs eliminate the need for other model systems. Rather, they add an important layer of biological relevance that can complement existing tools and help researchers make more informed decisions earlier in development. As workflows continue to improve, iPSCs are strengthening their role in preclinical research by providing higher-confidence, human-relevant data within disease-specific genetic contexts—enhancing predictability without replacing the need for clinical validation.

Looking Ahead

The impact of iPSCs on disease modeling is already substantial, but the field is still evolving. Improvements in reprogramming, differentiation, genome editing, and assay development continue to expand what researchers can do with these models.

For complex diseases in particular, iPSCs offer something especially valuable: the ability to study human biology in a system that is both experimentally accessible and genetically relevant. That combination is helping researchers better understand disease mechanisms, test hypotheses with greater precision, and move closer to more predictive models of therapeutic response.

As the technology continues to mature, iPSCs are likely to play an even greater role in connecting early-stage discovery with translational research. For scientists working to understand difficult diseases, that makes them not just a useful tool, but a transformative one.

Conclusion

iPSCs have changed the landscape of disease modeling by giving researchers access to patient-specific, versatile cell models that can illuminate the biology of complex disorders. From rare disease research to gene editing and translational studies, their influence continues to grow across biomedical science.

Their true value lies in how they help connect questions of mechanism to opportunities for intervention. By enabling more relevant human cell models, iPSCs support a deeper understanding of disease and a more informed path toward therapeutic development.

As research continues to advance, iPSCs will remain central to efforts aimed at making preclinical studies more predictive, more personalized, and more biologically meaningful.

Author:

Dr. Xinyu Kong, PhD
Director of iPSC Generation and Gene Editing
iXCells Biotechnologies

References

Yamanaka, S. (2006). Induction of Pluripotent Stem Cells from Mouse Embryonic and Adult Fibroblast Cultures by Defined Factors. Cell, 126(4), 663-676.
Takahashi, K., & Yamanaka, S. (2006). Induction of Pluripotent Stem Cells from Mouse Embryonic and Adult Fibroblast Cultures by Defined Factors. Cell, 126(4), 663-676.
Shi, Y., Inoue, H., Wu, J. C., & Yamanaka, S. (2017). Induced pluripotent stem cell technology: a decade of progress. Nature Reviews Drug Discovery, 16(2), 115-130.
Zhang, Y., et al. (2013). Derivation of human pluripotent stem cells from patients with amyotrophic lateral sclerosis. Cell Stem Cell, 8(3), 305-317.
Bilican, B., et al. (2012). Mutant induced pluripotent stem cell lines recapitulate aspects of TDP-43 proteinopathies and reveal cell-specific vulnerability. Proceedings of the National Academy of Sciences, 109(15), 5803-5808.
Kiskinis, E., et al. (2014). Pathways disrupted in human ALS motor neurons identified through genetic correction of mutant SOD1. Cell Stem Cell, 14(6), 781-795.
Doudna, J. A., & Charpentier, E. (2014). The new frontier of genome engineering with CRISPR-Cas9. Science, 346(6213), 1258096.
Li, Y., et al. (2018). Generation of Isogenic Human Pluripotent Stem Cell Pairs for Functional Genomic Screening. Nature Protocols, 13(1), 14-27.
Takahashi, K., & Yamanaka, S. (2016). A decade of transcription factor-mediated reprogramming to pluripotency. Nature Reviews Molecular Cell Biology, 17(3), 183-193.
Yagi, T., et al. (2011). Modeling familial Alzheimer’s disease with induced pluripotent stem cells. Human Molecular Genetics, 20(23), 4530-4539.
Moretti, A., et al. (2010). Patient-specific induced pluripotent stem-cell models for long-QT syndrome. New England Journal of Medicine, 363(15), 1397-1409.
Young, C. S., et al. (2016). A Single CRISPR-Cas9 Deletion Strategy That Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells. Cell Stem Cell, 18(4), 533-540.
Shoji, E., et al. (2015). Modeling splicing factor SRSF2-mediated gene expression regulation in splicing deficiency using iPS cells. Stem Cell Reports, 3(4), 635-648.

PARTNER WITH US

With over a decade of experience in cellular biology and preclinical research, iXCells delivers human-relevant models that help researchers improve predictability and move new therapies forward.

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iXCells Biotechnologies Joins International Collaboration to Evaluate Chemotherapeutic Toxicity in Human Organoids

kfaulkner9a16998ec4 — Tue, 24 Mar 2026 12:00:28 +0000

iXCells Biotechnologies Joins International Collaboration to Evaluate Chemotherapeutic Toxicity in Human Organoids

San Diego, CA, 24 March 2026: iXCells Biotechnologies (“iXCells”), a leading provider of human cell-based solutions and custom iPSC services, today announced it has entered an international collaboration with Rosebud Biosciences (“Rosebud”), Kantify, and Incite to evaluate off-target chemotherapeutic toxicities in patient-specific organoids. Building on iXCells’ recent partnership with Rosebud¹ and funded by Incite, the project will initially focus on predicting toxicity in heart, liver, and kidney tissue models, to develop personalized, multi-organ toxicity profiles for novel chemotherapies.

Development of patient-specific human organoids models for heart, liver, and kidney tissues

During the collaboration, iXCells will reprogram peripheral blood mononuclear cells (PBMCs) isolated from the blood of a patient with liposarcoma, a rare, malignant cancer that originates in fatty tissues, into human induced pluripotent stem cells (hiPSCs). Rosebud, an innovator in organoid development and complex 3D biology, will then differentiate these hiPSCs into heart, liver, and kidney organoids using its industrialized organoid platform and perform the drug toxicity studies on investigational chemotherapeutic compounds supplied by Kantify, a Belgian company using a novel AI discovery engine called Sapian to discover novel targets and drugs for underserved diseases.

Toxicity will be quantified through longitudinal imaging and machine learning driven analysis, cardiac activity measurements, live/dead viability assays, tissue-specific immunocytochemistry, and multiparametric readouts of structural and functional organ injury.

Unforeseen off-target toxicity in critical organs remains one of the most significant risks for cancer patients receiving chemotherapy, with injury to the heart, liver, or kidneys being a leading cause of clinical trial failures.² By assessing chemotherapeutic toxicities directly in organoids developed from a patient’s own cells, the collaboration will generate personalized toxicity profiles to guide therapeutic decision making.

Steve Smith, Chief Executive Officer of iXCells Biotechnologies, commented: “We are excited to be collaborating with Rosebud, Kantify, and Incite on this joint initiative to demonstrate how hiPSC technologies and organoid systems can transform precision toxicology. By starting from a patient’s own cells, we gain unprecedented insight into off-target toxicity, one of the biggest challenges in developing safer and more effective cancer therapeutics.”

Kitch Wilson, MD, PhD, CEO and Co-Founder of Rosebud Biosciences added: “This project is addressing one of the most urgent questions in oncology, determining whether a patient’s vital organs can tolerate lifesaving therapies. By combining our scalable organoid technology with iXCells’ expertise in cellular reprogramming and Kantify’s AI drug discovery platform, we are creating a patient specific testing system for novel chemotherapy drugs that reflects exactly the type of new approach methodologies the FDA is encouraging.”

Visit iXCells’ human cell-based solutions and custom iPSC services page for more information.

References

About iXCells Biotechnologies

iXCells Biotechnologies (“iXCells”) is a leading San Diego, California-based cell technology company focused on generating a wide variety of predictive human disease models. In addition, iXCells is pursuing custom and patient-specific services leveraging its expertise in the preparation, handling, engineering and differentiation of induced pluripotent stem cells (iPSCs) and in the isolation of primary cells along with associated services for descriptive and functional cell characterization, compound screening and toxicology testing.

The company’s mission is to supply a diverse range of human disease models, derived from ethically sourced patient and healthy donor tissues, to support academic, pharmaceutical, and clinical researchers globally. By providing predictive human cell models, we aim to deepen the understanding of the molecular mechanisms underlying disease onset and progression as much as slow-down or reversion of disease, and accelerate the discovery and development of new medicines and innovative therapies to further advance precision medicine.

Media Contacts

Christy Holt
VP of Marketing and Sales
christy.holt@ixcellsbiotech.com
iXCellsBiotech.com

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and iXCells on YouTube.

COMPANY NEWS

Joint initiative with Rosebud Biosciences, Kantify, and funded by Incite will identify and quantify off-target toxicities in chemotherapy patients

iXCells joins global collaboration to assess chemotherapy toxicity using patient-specific human organoids and iPSC technology, advancing precision toxicology.

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iXCells Offers High-Throughput iPSC Production Built as an Extension of Your Lab

kfaulkner9a16998ec4 — Wed, 11 Mar 2026 00:29:48 +0000

iXCells Offers High-Throughput iPSC Production Built as an Extension of Your Lab

iXCells Biotechnologies USA, Inc. (“iXCells”), a leading provider of human cell-based solutions and custom iPSC services, today announced the delivery of over 500 characterized induced pluripotent stem cell (iPSC) lines as part of a multi-year collaboration with a global biotechnology company. The collaboration leverages iXCells’ iPSCore platform, which enables the large-scale generation of well-characterized patient-derived iPSC lines, to support the discovery and development of novel therapeutics.

The platform supports drug discovery by enabling the creation, characterization and delivery of validated iPSC lines derived from diverse patient cohorts. iPSCore is delivering nearly 200 lines per month, with a reprogramming success rate exceeding 98%, and built-in cell banking at each stage to ensure continuity and mitigate risk. This scale allows researchers to study large patient cohorts, capturing population-level genetic diversity and minimizing background variance, which is critical for understanding complex diseases such as neurodegenerative disorders, oncology, cardiac disorders, or toxicology screening.

iPSCore platform delivered over 500 characterized iPSC lines during first year of collaboration

To date, iXCells has delivered more than 500 characterized patient-derived iPSC lines under the collaboration, with more than 700 additional lines in development, demonstrating the scalability and efficiency of the iPSCore platform. The collaboration demonstrates iXCells’ ability to deliver iPSC reprogramming at industrial scale, enabling high-throughput production of large volumes of patient-derived lines while maintaining quality, reproducibility, and consistency. Further projects as part of the collaboration will expand the scale of the platform, including for differentiated cell types and disease-relevant models.

The generation of validated iPSC lines using iPSCore is particularly meaningful in complex therapeutic areas where understanding patient diversity and disease biology is crucial. By enabling scalable access to high-quality, cohort-based iPSC models, iXCells is supporting researchers to generate comprehensive, population-relevant data sets, accelerating target identification and optimizing therapeutic strategies

Scaling Patient-Derived iPSC Models for Better Drug Discovery

Steve Smith, CEO at iXCells Biotechnologies, said: “Reliable, large-scale access to patient-relevant cell models is revolutionizing our approach to disease research. By reaching this milestone in our collaboration, we are demonstrating that the right expertise and infrastructure can empower discovery teams to leverage diverse data from the very start, enabling delivering iPSC models at scale and dramatically improving the likelihood of successful drug development. Ongoing expansion of our iPSCore platform capabilities will enable large-scale access to high quality patient-derived human cell models to drive breakthroughs in drug discovery.”

Alex Gulotta, Board of Directors, iXCells Biotechnologies commented: ” iXCells’ commitment to industrialization has fundamentally shifted the paradigm in drug discovery. Embedding patient-derived human cell models as foundational infrastructure throughout the drug development process enables hypothesis testing that truly reflects patient biology. What iXCells has built in iPSCore, and demonstrated through this collaboration, is exactly the kind of validated, scalable, revenue-generating platform that the industry needs and acquirers are looking for. This supports smarter, earlier decision-making, reducing costly late-stage failures and mitigating risk for stakeholders and partners.”

About iXCells Biotechnologies

Media Contacts

Christy Holt
VP, Marketing and Sales
christy.holt@ixcellsbiotech.com
iXCellsBiotech.com

To request more information visit
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and iXCells on YouTube.

COMPANY NEWS

iXCells Reaches iPSC Line Delivery Milestone

iXCells Biotechnologies announces delivery of over 500 characterized patient-derived iPSC lines through its iPSCore platform, enabling large-scale human disease modeling for drug discovery.

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iXCells Biotechnologies and Rosebud Biosciences Partner to Advance Organoid-Based Models for Rare Diseases

kfaulkner9a16998ec4 — Tue, 27 Jan 2026 09:00:24 +0000

iXCells Biotechnologies and Rosebud Biosciences Partner to Advance Organoid-Based Models for Rare Diseases

San Diego, CA, 27 January 2026: iXCells Biotechnologies (“iXCells”), a leading provider of human cell-based solutions and custom iPSC services, and Rosebud Biosciences, an innovator in organoid development and complex 3D biology, today announced a partnership to develop a personalized, human-based approach for predicting drug safety and informing translational decision-making in rare diseases. As iXCells enters its next phase of growth, the collaboration expands the Company’s custom integrated platform and strengthens the suite of modeling technologies available to customers seeking advanced predictive and physiologically relevant systems.

Expansion of iXCells’ iPSCore platform with integration of 3D human organoid expertise will enable evaluation of drug toxicity and tissue-specific responses

Through this partnership, iXCells will integrate Rosebud’s organoid generation and characterization expertise into its existing iPSC solutions, enabling access to complex 3D human systems that better represent tissue-level biology and simulate therapeutic response. The combined workflow brings together iXCells’ scalable, modular iPSC platform, iPSCore, with Rosebud’s AI-driven organoid platform, designed to generate scalable, reproducible human tissue data for drug discovery and safety assessment. Together, the technologies offer researchers more complete and biologically representative model systems for understanding disease mechanisms and drug safety.

Responds to growing demand for scalable, patient-relevant human models to support translational decision-making in drug development

The expansion of organoid capabilities reflects iXCells’ continued investment in next-generation modeling technologies that meet growing customer demand for human-relevant and predictive systems. Organoids are an essential component of translational research strategies, particularly for evaluating drug toxicity and tissue-specific responses. This integrated offering delivers the consistency, scalability, and reproducibility required for screening, engineering, multi-omics profiling, and mechanism-of-action studies within a unified, end-to-end solution.

Steve Smith, Chief Executive Officer of iXCells Biotechnologies, commented: “Our focus has always been on anticipating what our partners will need next, and Rosebud brings impressive depth in organoid science that aligns perfectly with that vision. This collaboration enhances our end-to-end platform and expands our ability to deliver next-generation models that reflect human biology with greater depth and accuracy. In areas such as rare diseases, where predicting human-specific safety and response earlier can significantly impact development timelines and patient outcomes, these integrated systems provide critical insight. This partnership reflects the strategic direction we are taking as iXCells scales its platform to support more complex, translationally focused programs for our partners.”

Kitch Wilson, Chief Executive Officer and Co-Founder, Rosebud Biosciences, added: “Our partnership with iXCells allows us to make advanced organoid systems more accessible to researchers who need models that better represent human biology. By combining our complementary strengths, we can support deeper exploration of disease biology and help drive the next generation of therapeutic discovery.”

About Rosebud Biosciences

Rosebud Biosciences builds industrialized, AI-powered human organoid platforms for drug discovery, toxicology, and precision medicine. The company integrates automated organoid differentiation, scalable robotics-driven workflows, and advanced machine learning analysis to generate large volumes of standardized, clinically meaningful human data. Capable of growing 10k organoids simultaneously with plans for multiples of 10k, Rosebud’s automation- and AI-first approach enables reproducible, high throughput evaluation of drug safety and efficacy across multiple human organ systems. The platform delivers the predictive, patient specific insights envisioned by FDA aligned NAMs, providing faster and more accurate assessments of how human tissues respond to therapeutic compounds. The company was founded by experts in stem cell biology, organoid engineering, and AI-driven phenotyping, bringing deep scientific pedigree to the creation of next generation human-based models.

About iXCells Biotechnologies

Media Contacts

Christy Holt
Director of Marketing
christy.holt@ixcellsbiotech.com
iXCellsBiotech.com

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iXCells on Linkedin,
and iXCells on YouTube.

COMPANY NEWS

iXCells Biotechnologies and Rosebud Biosciences Partner to Advance Organoid-Based Models for Rare Diseases

iXCells Biotechnologies and Rosebud Biosciences announce a partnership to integrate iPSC and organoid technologies, expanding human-relevant models for drug safety, toxicity, and rare disease research.

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iXCells Biotechnologies Appoints Steve Smith as Chief Executive Officer

Tom Faulkner — Mon, 12 Jan 2026 13:23:20 +0000

iXCells Biotechnologies Appoints Steve Smith as Chief Executive Officer

San Diego, CA, 12 January 2026: iXCells Biotechnologies, (“iXCells”) a leading provider of human cell-based solutions and custom iPSC services, today announced the appointment of Steve Smith as CEO. Previously CCO, Steve will lead the Company to advance execution and delivery of iXCells’ bespoke human-derived iPSC preclinical services as demand increases for scalable, high-quality, and human relevant preclinical models.

Steve has over 25 years’ experience in the life sciences industry, with a career spanning senior commercial leadership roles across global life science tools and service organizations. Holding leadership positions at Thermo Fisher and Abcam, including North American Sales Leader and Vice President of Cell Lines, Steve most recently served as CCO at seqWell, a provider of genomic library and multiplexing workflow solutions. Steve’s appointment as CEO marks a significant milestone for iXCells as the Company transitions into its next phase of growth, supporting larger, more complex preclinical programs and expanding multi-study collaborations with biotech, pharmaceutical, academic, and CRO partners.

Two Major Initiatives are Shaping the Next Phase of Growth

During Steve’s time as CCO, iXCells has introduced two major initiatives shaping the Company’s next phase of growth. The launch of iPSCore, iXCells’ discovery support platform designed for precision drug development in rare diseases, delivers comprehensive project management and assay development capabilities to accelerate the translation of complex research into actionable results. Steve also leads Project Mosaic, a pilot project developing patient-specific models of sporadic ALS by adapting methods from cancer research.

“Steve’s appointment marks a pivotal moment for iXCells,” said Alex Gulotta, Principal at Great Point Partners. “His strategic vision and deep expertise in iPSC technologies and human cell-based research are exactly what we need to accelerate our mission. With initiatives like iPSCore and Project Mosaic already gaining traction under his leadership, I’m confident that Steve will guide iXCells to new heights of scientific innovation and impact.”

Empowering Scientists With Human-relevant Disease Models

Steve Smith, CEO, iXCells Biotechnologies said: “iXCells’ mission is to empower scientists with high-quality, human-relevant disease models and tools to support personalized solutions that enable breakthrough discoveries. I am excited to lead iXCells through this next stage of growth, expanding our iPSCore end to end services, delivering actionable results in ongoing scientific partnerships, and providing customers with the consistency, scale, and collaboration required to advance complex discovery programs to enable the development of novel therapeutics for rare and complex diseases”

The Company Remains Dedicated to Advancing Scientific Discovery

This appointment strengthens the leadership team as iXCells focuses on expanding its portfolio of custom-tailored iPSC products and delivery of enhanced preclinical services to advance scientific discovery.

About iXCells Biotechnologies

Media Contacts

Christy Holt
Director of Marketing
christy.holt@ixcellsbiotech.com
iXCellsBiotech.com

To request more information visit
iXCells on Linkedin,
and iXCells on YouTube.

COMPANY NEWS

iXCells Biotechnologies appoints Steve Smith as CEO

This leadership transition supports the company’s next phase of strategic growth, aimed at expanding its portfolio of custom-tailored iPSC products and delivery of enhanced preclinical services.

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iXCells Biotechnologies Selected to Perform the First Pilot in Patient-Led ALS Precision Medicine Effort

Tom Faulkner — Tue, 26 Aug 2025 15:00:31 +0000

iXCells Biotechnologies Selected to Perform the First Pilot in Patient-Led ALS Precision Medicine Effort

The lab will run the inaugural study for Project Mosaic, a groundbreaking initiative to develop patient-specific models of sporadic ALS and fix a pipeline where 99% of treatments fail.

Mosaic Neuroscience and iXCells Biotechnologies USA, Inc. (“iXCells”), a leading cell technology company specializing in the generation of predictive human disease models, announced a groundbreaking pilot project aimed at making it easier to develop precision drugs for ALS, a notoriously complex and deadly disease.

Launched as part of “Project Mosaic” – an ambitious effort to transform how drugs are developed for ALS by adapting methods from cancer research – the pilot is designed to advance industrial methods of creating patient-specific models of sporadic ALS (sALS), which accounts for 90% of ALS cases. If successful, Mosaic Neuroscience, the public benefit company organizing Project Mosaic, hopes to use the patient-specific models, multiomics data, and advanced AI to help drug makers develop precision medicines for sALS.

“Disease models are one of the most overlooked bottlenecks in ALS drug development,” said Bernie Zipprich, Mosaic’s founder and CEO. “In cancer, testing drugs in patient-derived cells is standard before human trials. Since you can’t biopsy an ALS patient’s brain, Project Mosaic is creating ‘neurobiopsies’ – lab-grown neurons that replicate an individual’s disease. These models will give us a path to develop precision therapies for the majority of patients. We’re thrilled to be partnering with iXCells to take the first steps in this critical journey.”

iXCells Biotechnologies was chosen through a competitive process by the Mosaic Neuroscience team, with input from the project’s scientific advisors, based on the company’s experience in ALS modeling, its R&D capacity, biomanufacturing abilities, scientific leadership, and commitment to innovation for ALS. Its new iPSCore platform, designed for precision drug development in rare diseases, made iXCells a natural partner for Project Mosaic.

“What excites us about Project Mosaic is not just the urgency – it’s the rigor,” said Nianwei Lin, PhD, President and CSO of iXCells. “This isn’t just another iPSC study. It’s a systematic effort to make sALS disease models a new preclinical standard.”

Turning Adversity into Innovation

Diagnosed with ALS in 2020 at the age of 32, Zipprich – a Harvard and Wharton-trained healthcare entrepreneur – launched Mosaic in 2024 with the goal of addressing the high rate of sALS clinical trial failures by modernizing the way ALS drugs are tested before human trials.

ALS often progresses differently in different patients and can vary biologically at the cellular level as well. But this diversity is not reflected in the cell and animal models used by most researchers to develop drugs. In fact, most ALS research is based on rare familial forms which affect fewer than 10% of patients. Mosaic contends that this is a key reason why 99% of treatments trialed for sALS fail.

“The vast majority of ALS patients have the sporadic form of the disease, but nearly all of our preclinical tools are based on the familial forms,” said Eugene Brandon, PhD, Mosaic’s CSO and a neuronal stem cell industry veteran. “It’s like trying to develop drugs for liver cancer using only HER2-positive breast cancer cells.”

A New Model for Precision Neurology

Project Mosaic builds on a growing body of academic work suggesting that, like cancer, ALS is not a single disease – but rather a “mosaic” of overlapping subtypes sharing common characteristics. More than 50 genes have been identified as important ALS triggers in a subset of cases, indicating diverse and distinct causes. Multiple labs have found strong evidence of sALS subtypes in data derived from hundreds of autopsied patients. And other researchers, including USC’s Justin Ichida, PhD, a project advisor, have found evidence that genetic differences across sALS patients may contribute to differences in how patients respond to drugs.

The pilot with iXCells is a landmark first attempt to industrialize disease models that reflect this diversity. To do this, the pilot will use patient-derived stem cell lines from Answer ALS – previously profiled by Johns Hopkins researchers Jeff Rothstein, PhD, and Alyssa Coyne, PhD – to generate transcriptomic signatures indicative of sALS. The goal is to show that these signatures are unique and consistent for each patient. Later work will investigate how these patterns can help match patients to drugs.

Working with iXCells serves another key Project Mosaic goal: shortcutting the time it usually takes for academic advances to achieve industry adoption and patient impact. Following the pilot, iXCells plans to make sALS models with standardized phenotypes available to researchers and drug makers. At the same time, Project Mosaic will advance to stage two: inviting additional academic and industrial labs to cross-validate and build on the pilot results.

“Project Mosaic is not just about cutting-edge research,” said Zipprich. “It’s about fixing the broken ALS pipeline – at the speed this disease requires. This can only happen if ALS nonprofits, academic researchers, and industry work together in new ways. Someone new is diagnosed every 90 minutes and people are dying every day. We have no time to waste.”

About Mosaic Neuroscience

Mosaic Neuroscience is a public benefit biotechnology company founded to develop precision medicine tools for sporadic ALS and other neurodegenerative diseases. Its flagship initiative, Project Mosaic, brings together leading academic and industrial labs to create and validate patient-specific ALS models.

About iXCells Biotechnologies

Media Contacts

Tushar Patel PhD
Chief Business Officer
tushar.patel@projectmosaic.org
ProjectMosaic.org

Christy Holt
Director of Marketing
christy.holt@ixcellsbiotech.com
iXCellsBiotech.com

To request more information visit
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iXCells on X.com,
and iXCells on YouTube.

COMPANY NEWS

iXCells Biotechnologies was chosen through a competitive process by the Mosaic Neuroscience team, with input from the project's scientific advisors.

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iXCells Biotechnologies Launches iPSCore™ Platform for Generating Highly Characterized iPSC-derived Human Cell Models

Tom Faulkner — Wed, 11 Jun 2025 17:31:18 +0000

iXCells Biotechnologies Launches iPSCoreTM Platform for Generating Highly Characterized iPSC-derived Human Cell Models

iXCells Biotechnologies focuses on creating a broad portfolio of iPSC-derived off-the-shelf and custom cell models from healthy and patient donors to make “clinical-trials-in-a-plate” a reality.

iXCells Biotechnologies USA, Inc. (“iXCells”), a leading cell technology company specializing in the generation of predictive human disease models is pleased to announce the launch of its modular iPSCore^TM platform.

iXCells’ mission is to empower life science researchers and patient foundations to advance personalized therapies and accelerate discovery of better medicines for a healthier future.

iXCells’ cell engineering platform iPSCore is a unified and dedicated system where tools, skills, experiences and expertise are structured together to design, develop and build iPSC-derived cell products from ethically sourced biospecimens, and cell fractions or cells (e.g. PBMCs or fibroblasts) derived from healthy or patient donors. Cells can be reprogrammed, edited and differentiated into a wide variety of iPSC-derived cell types and 3-dimensional human cell models. iPSCore incorporates extensive characterization, descriptively and functionally, which can be used in an assortment of downstream assays tailored to customer needs. Currently the platform processes >1,000 samples per year with routinely >96% success rate and is expected to multiply the output of patient-derived iPSC-derived cells by 2026.

Partnering Opportunities

“Numerous conversations with our clients reveal that relevant human cell models are not readily available, often requiring lengthy design and engineering processes essential for creating the desired genotype and/or phenotype from demographically defined cohorts,” stated Steve Smith, Chief Commercial Officer. He continued, “Our iPSCore platform leverages our capabilities, long-term expertise and passion in cell engineering, enabling iXCells to generate hundreds of iPSC lines and differentiating them into numerous cell types that are characterized for confidence and ultimately provide our customers with access as either off-the-shelf items or tailored made-to-order cells.”

Such iPSC-derived disease models continue to support the pharmaceutical and biotechnology industry’s shift away from animal models as recognized by FDA Modernization Act 2.0 which allows drug developers to submit non-animal data to demonstrate safety and efficacy of investigational drugs prior to conducting clinical trials. Human cell models and specifically patient-derived cell models have shown to be more predictable than animal models and can help find solutions for significant unmet medical needs faster.”

Helge Bastian, PhD, CEO of iXCells expressed his enthusiasm by saying, “The development and launch of our iPSCore platform marks a significant milestone in our company’s history and demonstrates our commitment to unfold the huge potential of one of the most disruptive innovations in the life sciences for developing novel medicines and therapies. Game-changing iPSC-technology combined with continued advancements in subsequent processes to edit and differentiate iPSCs into essentially any cell type of the human body together with steady elevations to characterize the true biological state of such cells will enable researchers to build highly predictive in-vitro cell or microtissue models mimicking human biology as closely as ever seen before.” Dr. Bastian added, “The integration of these serial technology advancements into an optimized and standardized workflow will allow screening drugs on an endless variety of human genotypes in-vitro. Leveraging this capability in combination with AI technology to identify disease-relevant patterns and phenotypes is paving the way for making the concept of ‘clinical-trials-in-a-plate’ a reality.”

Partnering Opportunities

Contact iXCells and build strategic partnerships to accelerate patient-specific drug and therapy developments.

About iXCells Biotechnologies

Contacts

To request more information visit ixcellsbiotech.com/ipscore-lp/.

COMPANY NEWS

iXCells Biotechnologies Launches iPSCore Platform for Generating Highly Characterized iPSC-derived Human Cell Models

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