There are thousands of rare diseases spanning various therapeutic areas, and many of the diseases lack therapies or even knowledge of disease drivers. Recent trends in a supportive regulatory environment and pharmaco-economic incentives are driving increased investments in rare disease drug discovery.

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Charles River has developed tools for quicker and more translational preclinical studies that will help push more clinical candidates targeting rare diseases into the clinic. This includes validating relevant models and investigating the mechanisms of action using appropriate biomarker endpoints. Most importantly, given the dearth of in vitro and in vivo models that can be used to test orphan drug compounds, we are committed to making these tools available to the drug development community.

We offer end-to-end drug discovery services from target identification to IND enabling safety/toxicology studies via integrated multi-disciplinary teams across multiple therapeutic areas. Our teams use state-of-the-art capabilities and resources to customize rare and orphan disease drug discovery across several therapeutic areas.

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Eureka Blog Posts

Read what the rare disease experts are saying about everything from identifying novel therapies with cross-functional and pre-competitive consortia to personalized medicine.

Global Genes® RARECast

Hear about the evolving nature of rare disease collaborations and how that’s changing the way researchers work.

Scientific Posters

Members of our scientific staff showcase key findings from independent research and collaborative efforts with our clients.