Retina Efficacy Study

Predictive efficacy testing on human retinal models

Accelerate your drug development using human retinal organoids and RPE models

Recapitulate the architecture & function of the human retina

Rapid evaluation of novel compounds’ efficacy

Accelerate drug discovery

Efficacy testing of novel therapies on predictive human in vitro retinal models

Retinal compound efficacy study: this service evaluates how a compound or viral vector affects structure and function of retinal cellular models.

The in vitro drug efficacy study allows rapid evaluation of the efficacy of your drug on high quality healthy and/or diseased human IPSC-derived retinal organoids and/or RPE models. The models are highly representative of the structure and function of human retinal tissues and can also be generated from patients’ iPSCs or gene edited iPSC lines, thus facilitating predictive advanced in vitro testing of new therapeutics and accelerating lead compound selection.

The assays can be carried out at different time points of organoid differentiation and will provide predictive data to allow for confident decision making to progress your new drug or submit an IND.

Service outputs

Rapid retinal efficacy evaluation of compounds in retinal organoids and RPE
Quantitative and qualitative analysis of key markers expression by gene expression analysis and immunofluorescence on frozen retinal organoid sections
Time points comparisons
Compound effect on PRE barrier, phagocytic integrity, viability and ultrastructure

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Retinal therapy efficacy study

96-well format

Rapid and predictive

Assays

Retina efficacy study: Cell morphology and cell viability; key markers analysis and functional readouts

Models

Human retinal organoids (RO)
Human retina pigment epithelium (RPE)
Models are derived from WT iPSCs or Patient/gene-edited iPSCs supplied by the client

Timeline

2-6 months

How to access the retinal efficacy study service?

Outsource your experiments to us to obtain robust predictive data

Take advantage of our expertise. With our fast and reliable service, we design the experiments jointly with you to ensure we answer your specific questions. We will use our readily available models or generate them from your patients' cells or gene edited iPSC line. The data that will be provided to you at the end of the study will define the retinal efficacy profile of your compounds and, on request, rescue strategies. Our bespoke projects are carried out in our state-of-the-art UK facilities by our team of retina experts.

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Generation of patient-derived retinal organoids and RPE as part of the service

The complete service includes the generation of retinal organoids or RPE from healthy, patient-derived or gene-edited iPSCs supplied by the client, feasibility studies, experimental design, compound testing and development of a set of data that can be used to support your regulatory submissions.

Service overview Close Open

Newcells provides retinal efficacy study service using our highly reproducible iPSC-derived human models (retinal organoids and RPE) to provide you with predictive data

Retinal compound efficacy studies are carried out on our highly reproducible iPSC-derived human models (retinal organoids and RPE) to provide clients with predictive data for clinical translation and to be included in regulatory applications.

With a streamlined manufacturing process releasing regular supply of highly reproducible tissue, projects timelines are usually short with studies generally completed within two months. The predictive data generated by our scientific experts will therefore allow you to rapidly assess the efficacy of your drugs to confidently guide key decision-making steps during lead compound identification, drug development or regulatory submissions.

An typical retinal compound efficacy study includes a set of advanced in vitro assays to assess cell morphology and cell viability, photoreceptor functionality and degeneration as well as analysis of key marker expression and localisation to test the efficacy of novel drug compounds and viral vectors such as AAV.

Example 1: Testing the effect of a novel therapeutic on retinal organoids Close Open

The effect of a compound was assessed in a retinal organoid model containing a specific mutation found in an ocular disease to determine how it affected the different cell types present in the retinal organoid model.

Quantification graphs of horizontal cells marker PROX1 following treatment with a novel compound; Statistical analysis performed by unpaired t-test (p< 0.01). Data represents mean ± SEM, n=6.

Disease model organoids had significantly higher expression of horizontal cell marker (PROX1) and rod cell marker (RHO) compared to wild type organoids at day 180 of differentiation following treatment with the novel compound. Other cell-specific expression markers for bipolar cells, amacrine, muller glial and astrocytes cells were not affected by treatment.

Example 2: Assessment of Retinal Pigment Epithelial barrier integrity upon drug treatment Close Open

The retina efficacy service on the RPE model assessed the effect of a compound on the barrier properties of RPE. The compound was tested for its ability to disrupt the epithelial barrier such as layer integrity, permeability and cell viability.

Morphological changes of the epithelium were assessed through brightfield imaging.

Representative brightfield imaging (x20 magnification) of RPE cells before and after 6 hours of treatment with 25 and 100 µg/ml of test compound. Controls include media only (untreated control) and vehicle control (PBS). All the images were acquired with the same magnification, using consistent exposures across all conditions.

No notable morphological changes were observed after 6 h of treatment. TEER measurement showed no significant differences upon treatment with the chosen compound, confirming that the tested compound does not significantly affect the RPE barrier integrity.

Models to choose from for this service

Retinal organoids

The human retinal organoids are iPSC-derived microtissue models. They recapitulate the complex structure of the human retina with laminar cell organisation mimicking embryonic development. They contain the outer photoreceptor segment of the retina that responds to light.

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Retinal Pigment Epithelium

A functional human 2D in vitro model of retinal pigment epithelial cells generated from human iPSCs recapitulating phagocytosis of photoreceptor outer segments. The RPE cells are pigmented and displays typical cobblestone morphology making them a complex human in vitro model able to produce predictive translational data.

RPE cells displaying cobblestone morphology. Cells were immunolabeled with tight-junction ZO-1 marker (shown in green) and co-stained with nuclei marker, Hoechst (shown in blue). — RPE cells displaying cobblestone morphology. Cells were immunolabeled with tight-junction marker ZO-1 (shown in green) and co-stained with nuclei marker Hoechst (shown in blue).

Disease modelling

We can develop a bespoke retinal organoids or RPE model or retinopathies starting from patient's iPSC line or gene-edited iPSC lines supplied by our clients.

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Gene therapy vector assessment

Rapid in vitro preclinical evaluation of viral vectors for retinal therapy development.

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Retinal toxicity evaluation

High throughput in vitro drug screening for retinal safety/toxicity and efficacy testing.

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