Given the remarkable shared similarities between non-human primates (NHPs) and humans, NHP models of inherited retinal disease (IRD) provide insight into the pathogenic processes involved. Ace Therapeutics is pleased to design and develop NHP models of IRD to address the specific problems associated with such diseases. These models exhibit well-defined phenotypic features and provide stable and heritable genetic signatures, making them invaluable tools for ophthalmic studies. Considering our unparalleled experience with ocular disease models, your pipeline will reach the finish line in the blink of an eye!
Inherited retinal diseases (IRD), an important cause of blindness worldwide, represent a group of genetically and clinically heterogeneous disorders with more than 270 genes associated with IRDs. Mutations in genes critical to retinal function lead to the death of photoreceptor cells and/or the retinal pigment epithelium (RPE), resulting in progressive vision loss. A major limitation in developing effective therapies is the lack of animal models that fully replicate the human condition. Particularly for cone disease, rodent, canine, and feline models without a true macula have substantial limitations. In contrast, the cone-rich macula of nonhuman primates (NHP) closely resembles that of the human retina. Therefore, establishing well-defined NHP models of inherited retinal diseases, especially cone disease that is predictive of the human condition, could help advance novel therapies for patients in a more efficient manner.
Fig. 1. PDE6C associated retinopathy in non-human primates. (Moshiri A, et al., 2021)
Ace Therapeutics is an industry-leading provider of ocular disease models. Our development department for ocular disease models has always been committed to providing our global clients with a variety of different eye disease models for their research needs. Here, our team is pleased to provide you with non-human primates (NHP) models of inherited retinal diseases for advancing research.
Two major NHPs are used to construct genetic eye disorders at Ace Therapeutics. These are macaques (cynomolgus or cynomolgus) and macaques (rhesus).
Ace Therapeutics' NHP IRD models include naturally occurring models and models created with genetic methods. In recent years, naturally occurring retinitis pigmentosa and achromatopsic IRD NHP models have been identified. In addition, Ace Therapeutics replicates the pathogenic process of IRD development through genetic methods to create targeted genetic models, such as the CRISPR-Cas RP model and the achromatopsia model. These are achieved through gene editing or silencing using DNA- or RNA-based tools.
Tab.1. Ace Therapeutics' non-human primate (NHP) models of inherited retinal diseases
Mechanism of model | Model | Genotype |
---|---|---|
Naturally occurring | Retinitis pigmentosa (Macaca fascicularis) | Not identified |
Bardet–Biedl Syndrome (BBS), Retinitis Pigmentosa (Macaca mulatta) | BBS7 Spontaneous mutation in the BBS7 gene deletion |
|
Achromatopsia (Macaca mulatta) | PDE6C Homozygous R565Q missense mutation |
|
Gene knockout | Achromatopsia (Macaca fascicularis) | CNGB3 In situ CNGB3 knockout in cone photoreceptors |
Retinitis pigmentosa (Macaca mulatta) | RHO In situ RHO knockout in the retina |
Overall, our goal is to provide an ideal tool for testing your products. This tool not only enables the capture of disease pathogenesis by displaying disease genotypes and phenotypes but also allows for understanding the systemic effects of retinal treatments and the challenges associated with treatment delivery modes.
In order to accurately characterize the biological function of NHP models of IRD, and to evaluate the safety and efficacy of candidate therapies (gene therapy, cell therapy, implants, etc.), our scientists provide a variety of analytical services, including but not limited to:
Ace Therapeutics aims to provide a valuable tool to help our global customers capture the pathogenesis of inherited retinal diseases and test the efficacy of the product. If you are interested in our services or need more detailed information, please feel free to contact us. Our experienced scientists are ready to help you!
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