End-to-End Huntington’s Disease Studies

No matter what stage of your HD program, if you’re validating your target, or ready to advance your lead candidate, we offer predictable translational Huntington’s disease studies to enhance your program efficacy. View here how to incorporate these services into your workflow.

  • In vitro assays to monitor mHTT regulators, target degradation and ATM inhibitors
  • In vivo models (mouse and rat) for pharmacology and efficacy studies

The Disruptors: Conversations in Science
Learn how Ignacio’s personal experiences continue to drive his research into potential treatments for Huntington’s disease.
Meet Ignacio

In Vitro Huntington’s Disease Studies

Charles River Laboratories has a broad portfolio of Huntingtin (HTT) detection assays to support your Huntington’s disease (HD) drug discovery and development. Cell-based assays specific for different HTT protein species are available on the Mesoscale platform as well as the Quanterix® platform.

High content imaging using disease relevant cell types is critical to the translational screening of small molecules and genetic modifiers. These validated high content in vitro assays can be applied to both target and phenotypic based screening. HD HSC assays have also been developed using animal derived neurons and iPSC/hESC CRISPR edited cell lines.

Tell Us How We Can Support Your Program

Illustration of neurons

Exploratory Toxicology for Neuroscience Drug Discovery
This eBook describes strategies across the early stages of drug discovery to support confidence in your lead small molecule candidate and to ensure you proceed through the drug development process with the most promising candidate.
Download eBook

In Vivo HD Animal Studies

For successful translation from in vitro to in vivo, Q175 and R6/2 Huntington's disease mouse models have been validated using various biochemical markers, behavioral endpoints, gait changes, and cognitive testing. Some of the tools that have been used to validate the Huntington’s disease mouse model include:

In addition, biochemical methods such as histology, immunohistochemistry, and qPCR are included to detect biochemical endpoints.

Enhance Your In Vivo project

CHDI and Charles River Collaboration

Charles River Labs and CHDI Foundation began working together in 2005 on drug discovery and development for Huntington’s disease. Since then, Charles River has established and validated tools and methodologies within expert teams to support CHDI’s mission of collaboratively developing therapeutics that will substantially improve the lives of those affected by the disease. In 2025, an open-ended extension of this partnership was signed, marking the 20-year anniversary.

Together, Charles River and CHDI have identified novel potential drug targets, bred and validated translational research animal models, run large screens and generated proof-of-concept molecules, and evaluated the potential efficacy of both small molecule and biologic candidates in preclinical models of HD. These cutting-edge approaches have resulted in over one hundred co-authored posters and dozens of peer-reviewed papers. The expertise developed throughout this partnership has helped more broadly facilitate increased HD research within the larger drug discovery and development community.

The Charles River team have become true scientific colleagues, and we highly value the intellectual and technical contributions that they have made across dozens of projects over the course of our two-decade collaboration. We look forward to further deepening our research collaboration to bring benefit to Huntington’s families as soon as we can.”

Robert Pacifici, Ph.D., Chief Scientific Officer, CHDI

 

Image of person looking at resources on a computer with a cup of coffee. Charles River has many resources for Huntington’s Disease research.

Huntington’s Disease Resources
Check out our Huntington’s Disease posters, publications, and other scientific resources.
Browse The Resources

Frequently Asked Questions (FAQ) for Huntington’s Disease Studies