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RARE DISEASE

Patient-derived iPSC models and research solutions designed to advance rare disease understanding and accelerate therapeutic development. Built on validated platforms that support reproducible, high-quality discovery workflows.

Science Built for Diseases Too Rare to Wait.

iPSC-derived 2D models are the backbone of our platform, scalable, reproducible, and validated for the drug discovery workflows that move programs forward. For most applications, they’re the right tool.

But some questions require more.

When tissue architecture drives the phenotype, when cell-cell interactions are central to your mechanism, or when 2D data raises questions a monolayer can’t resolve, organoids are the next step.

Learn more about our capabilties
Talk with our team

Tell us your story
and how we can
support you.

23 rare disease
partnerships in 2025

Already 18 in 2026
and growing.

Why iXCells

  • We understand "rare". Rare disease programs don't fit standard CRO models. Small patient populations, limited published data, and non-existent commercial cell lines demand a partner willing to build from the ground up. That's where we work best.
  • Speed without shortcuts. We move with urgency because we understand the stakes for patients and families behind every program
  • Built for your biology. Every engagement starts with understanding your disease — not fitting it into a standard menu of services.
  • A growing community. With 23 rare disease partnerships last year and 18 already underway this year, we bring cross-disease perspective that benefits every program we support.
  • Accessible expertise. From academic labs and patient advocacy groups to emerging biotechs, we structure collaborations that work within real-world budgets and timelines.

Our current partnerships span:

  • Epilepsy & seizure disorders — including channelopathies, myoclonic-atonic epilepsy, and PACS2-related epileptic encephalopathy
  • Neurodevelopmental & intellectual disability — including X-linked and autosomal rare gene disorders affecting cognition and development
  • Neuromuscular disease & muscular dystrophy — including congenital muscular dystrophies and rare myopathies
  • Neurological & movement disorders — including multisystem proteinopathies and alternating hemiplegia of childhood
  • Mitochondrial disease — including DNM1L-related disorders and other mitochondrial dysfunction syndromes
  • RASopathies & developmental syndromes — including cardio-facio-cutaneous syndrome, Myhre syndrome, and chromosomal deletion disorders
  • Retinal & vision disease — including inherited retinal dystrophies with no current treatment options
  • Repeat expansion & myotonic disorders — including myotonic dystrophy type 1
  • Ultra-rare & N-of-1 disease — single-patient and ultra-rare conditions where no commercial path exists and every data point matters

Don’t see your disease listed?
Most of our partnerships start with a conversation.
Reach out to our team because we’re built for exactly this.

What We Bring to Your Program

Disease-Relevant Cell Models We build and validate human cell models that faithfully recapitulate your disease. This starts at patient-derived iPSCs to primary cells and engineered lines carrying your disease’s specific mutations. Your biology, modeled accurately from day one.

Custom Assay Development & Screening We design and execute assays built around your specific disease biology, giving you the right readouts to evaluate candidate compounds and validate therapeutic hypotheses. iXCells has no off-the-shelf guesswork.

Collaborative Research Support We work as a true scientific partner: advising on study design, executing preclinical studies, and delivering the data you need to attract funding and move toward clinical translation.