Physiologically relevant human iPSC-derived cell models and specialist services

Human iPSC-derived models

Cell provision and specialist services

• Predictive, highly validated disease and safety models
• Scalable differentiation and manufacture of cryopreserved iPSC-derived cells, shipped globally
• Comprehensive discovery and preclinical specialist services, including small molecule, ASO and AAV testing
• Model development, pilot and assay development expertise, including complex co-culture systems
• iPSC production and quality control, reprogramming and gene editing

Extensive patient and unaffected donor iPSC line portfolio

• Library of >100 iPSC lines from unaffected and patient donors representing over twenty indications with particular focus on neurodegenerative disorders and ophthalmology
• Collaboration with patient advocacy groups and PRISM ALS consortia
• Extensive quality control documentation and full commercial use ethical consents

Supporting NAMs (New Approach Methodologies)

• iPSC-derived cells validated on multiple MPS and advanced in vitro analysis platforms
• Support for tech transfer to CROs and in-house facilities

Neuroscience

Human iPSC‑derived models for neurodegeneration, neuroinflammation & preclinical drug discovery

Huntington’s disease
iPSC lines from five HD patients and one asymptomatic carrier. CENSOi019-B line (HTT: 14/127 CAG, now CAG143) displays instability in culture and is associated with accelerated disease onset. Striatal neurons derived from this iPSC line provide a model for longitudinal studies of repeat expansion and neurodegeneration and recapitulate key HD phenotypes: CAG instability, neurite pathology, and functional deficits.

ALS/MND
Mono- and co-culture models encompassing motor neurons and microglia from multiple ALS genotypes, including C9orf72, SOD1, and TDP‑43, consistently recapitulate key disease phenotypes such as neuronal hyperexcitability, altered firing, morphological changes and impaired microglial phagocytosis.

The PRISM ALS consortia is extending access to iPSC-derived cells from 14 sporadic disease lines to accelerate preclinical discovery.

Alzheimer’s disease
A portfolio of sporadic Alzheimer’s disease iPSC lines with APOE genotypes (E2/E3, E3/E3, E3/E4, E4/E4) differentiated into cortical neurons, astrocytes and microglia for complex cell model development and patient‑stratification studies.

Candidate drug testing across multiple iPSC models and neurotoxicity assays
Drug candidate, viral delivery vectors and novel mechanism drug toxicity and activity screening using multi-electrode array (MEA), flow cytometry, cellular imaging, plate-based assays and ‘omics.

iPSC lines from patients and unaffected donors
Library of fully quality-controlled iPSC lines from unaffected and patient donors, including ALS, Alzheimer’s disease, Huntington’s disease, Parkinson’s disease, frontotemporal dementia, Friedreich’s ataxia, spinocerebellar ataxia, Nasu-Hakola disease and more.

iPSC-derived neurons and neuroinflammatory cells for advanced in vitro modeling
Cryopreserved functional cortical excitatory neurons, striatal neurons, cortical inhibitory interneurons, microglia, astrocytes and motor neurons shipped globally. Disease‑relevant phenotypes and mono‑culture, co‑culture and complex model formats.

Specialist services
Services include reprogramming from patient cohort material and gene-editing. Custom differentiation of our iPSC lines, or client lines to multiple neuronal and glial cell types. Preclinical drug discovery services, assay development, target validation, mode-of-action and safety.

Ophthalmology

Retinal disease modeling & safety assessment

Dry age-related macular degeneration (AMD)
Dry AMD model using iPSC-derived RPE cells exposed to chronic low-dose N-retinylidene-N-retinylethanolamine (A2E) and blue light to simulate lipofuscin photoactivation. The model recapitulates key features of dry AMD pathology, including RPE atrophy, oxidative stress, complement activation, and inflammatory signaling.

Comprehensive high content screening services for assessment of RPE health and function, including growth factor secretion (VEGF, PEDF), barrier integrity (TEER), phagocytosis, pigmentation and maturity markers, oxidative stress, complement activation, cell viability and apoptosis.

Automated 384‑well dry AMD screening platform enabling robust and reproducible screening of 6,000 compounds per run for preclinical drug discovery.

RPE-microglia co-culture model
iPSC-derived RPE cells and iPSC-derived microglia co-culture for modeling retinal inflammation and the impact of AMD-related stressors. RPE cells displayed increased sensitivity to A2E- and blue light–induced stress when co-cultured with microglia compared to monoculture conditions including microglial proliferation and elevated secretion of inflammatory cytokines.

Retinal organoids for discovery and retinal toxicity
Compound efficacy and toxicity testing using human iPSC‑derived retinal organoids, enabling assessment of therapeutic response, mechanism of action. Optimised retinal organoid-based screening systems for retinal toxicity with cell viability (ATP-based assays), morphological assessment, and short- and long-term treatment regimes.

iPSC lines from patients and unaffected donors
Extensive human iPSC line library including multiple retinopathies, Leber congenital amaurosis and Ushers syndrome type 1.

Human iPSC-derived retinal-related cell models for advanced in vitro modeling
Functional and well characterized retinal pigment epithelium (RPE) cells, retinal organoids (d60, d150, d180 and custom maturation) and microglia. Provision of scalable custom manufacturing from iPSC library, client lines or post-gene edit.

Specialist services
Services include reprogramming from patient cohort material and gene-editing. Custom differentiation of our iPSC lines, or client lines for retinal and ophthalmic research models, including retinal organoid generation. Preclinical drug discovery services, assay development, target validation, mode of action and safety.

Cardiovascular

Cardiovascular disease modeling and cardiac safety assessment

Cardiac safety and disease‑relevant models

Extensively characterised iPSC‑derived ventricular and atrial cardiomyocyte models capture key cardiac functional endpoints with consistent readouts, including action potential dynamics, conduction velocity, contractility, and chamber‑specific pharmacology. Ventricular cardiomyocytes are CiPA‑validated models supporting consistent assessment of pro‑arrhythmic risk and regulatory‑relevant cardiac safety profiling. These systems support NAMs (New Approach Methodologies) for more predictive, physiologically relevant testing.

Cardiac model optimisation and maturation

For applications requiring enhanced maturity, we provide MyoMax™ cardiac maturation media, which improves functional and structural characteristics of human iPSC‑derived ventricular cardiomyocytes, including reduced spontaneous beat rate, improved conduction velocity, shortened action potential duration, increased expression of maturity‑associated cardiac genes, and improved sarcomere alignment and expression of cardiac maturity markers.

In vitro cardiovascular model & assay development with platform integration

Services include complex model development of advanced cardiac models in monoculture and co-culture, custom protocol design and optimization, model characterization, phenotypic analysis, treatment titration and endpoint assays, integration with MPS and other platforms and collaborative scale-up for transfer to higher throughput platforms and CRO, or in-house workflows.

Cardiotoxicology and candidate drug testing in human iPSC‑derived models

Candidate drug testing and cardiac safety assessment using human iPSC‑derived cardiomyocyte models. Services and capabilities include evaluation of drug effects on cardiac electrophysiology, contractility, ion channel function, and chamber‑specific pharmacological responses.

iPSC lines from patients and unaffected donors

Fully quality-controlled iPSC lines from male and female donors and we are actively building diversity and additional disease-relevance into the collection.

Chamber- and sex-specific iPSC-derived cardiomyocytes for advanced in vitro modeling

Functionally and phenotypically characterised iPSC‑derived ventricular, left ventricular, and atrial cardiomyocytes exhibiting distinct chamber‑specific ion‑channel expression, contractile behaviour, and pharmacological responses. Models support mono‑culture formats and scalable assay systems for cardiac safety and efficacy screening.

Specialist services

Services include manufacturing‑scale differentiation of iPSCs to cardiac cells, quality control, cryopreservation, media optimisation, and custom workflows using our, client‑supplied, or third‑party iPSC lines.

Dermatology

Human iPSC‑derived skin‑relevant models for active compound testing and research

Sensory neurons

Utilized in models of pain and sensation including co-culture, microfluidics devices, organ-on-chip platforms and for in vitro disease modeling research. Exhibit functional relevance across multiple assays, including capsaicin treatment.

Acne and hyperseborrhea

Human iPSC‑derived sebocyte models recapitulate key sebocyte functions, including lipid synthesis, androgen responsiveness, inflammation, and response to C. acnes, arachidonic acid, and cannabinoids. Available in 2D and reconstructed 3D sebaceous gland formats.

Pigmentation, tanning, and lightening

Human iPSC‑derived melanocytes from multiple donor phototypes, support assays for melanogenesis, depigmentation, tanning, toxicity, and proliferation, with demonstrated melanosome production and transfer.

Candidate compound testing

Outsourced efficacy and toxicity testing using sebocyte, melanocyte, and sensory neuron models, with flexible assay design and physiologically relevant endpoints.

High‑content screening

Multiparametric readouts including lipid synthesis, pigmentation, cytokine release, receptor activation, neurite outgrowth, cell viability, and morphology across 96‑well and scalable screening formats.

iPSC lines from patients and unaffected donors

A diverse, well‑characterized human iPSC line library enabling scalable, reproducible manufacturing of dermatology‑relevant cell types. Lines support research across cosmetics, dermatology, pigmentation, acne, and neuro‑cosmetics.

iPSC-derived skin models for advanced in vitro modeling

Functionally validated sebocytes, melanocytes, and sensory neurons, suitable for monolayer, 3D, co‑culture, and microfluidic model formats.

Specialist services

Services include manufacturing‑scale differentiation of iPSCs, quality control, cryopreservation, media optimisation, and custom workflows using our, client‑supplied, or third‑party iPSC lines. Automated, reproducible high‑throughput screening platforms for dermatology and cosmetic ingredient testing, supporting multi‑condition and multi‑endpoint studies with consistent physiologically relevant biology.

iPSC line collection

A  library of high-quality iPSC-derived lines from unaffected and patient donors, the portfolio is strengthened through strategic collaborations including with StrataStem to deliver stratified sporadic Alzheimer’s disease models, the PRISM ALS consortia with LifeArc and ALS TDI to democratise access to diverse, patient-derived ALS models for researchers worldwide and other academic research institutes and patient advocacy groups.

axoCells™: physiologically relevant human iPSC-derived cells and kits