CHDI
KineMed and CHDI Foundation collaborate to develop new biomarkers of disease progression and to evaluate potential therapeutics for Huntington’s disease
Emeryville, CA and New York, NY, KineMed, Inc. (www.kinemed.com) and CHDI Foundation, Inc. announced today a collaboration to use KineMed’s isotopic labeling and dynamic proteomics platform to investigate the turnover rates of huntingtin protein, a mutant form of which plays a central role in the pathogenesis of Huntington’s disease (HD).
There are currently no therapeutics approved that can slow the progression of HD, an inherited neurodegenerative disorder that affects about 30,000 people in the United States, with up to 150,000 additionally at risk of developing the disease. HD patients have a genetic variation that causes the production of a mutant huntingtin protein with an expanded region of polyglutamine (polyQ) amino acids. The expanded huntingtin protein leads to the degeneration of brain cells, with disease severity related to the size of the expansion. The clinical signs of disease are behavioral, cognitive, and motor impairments that, over the course of the disease, significantly reduce the individual’s quality of life and ultimately cause death within 15 to 25 years of overt motor symptom onset.
KineMed’s uniquely sensitive tandem mass spectrometric technology will be used in this collaboration to measure in vivo dynamics of the huntingtin protein, including protein half-‐life and synthesis rate. This proprietary measurement approach will allow assessment of the rate of metabolism of huntingtin protein as a function of polyQ length and whether investigational drugs are affecting its production and clearance.
“The application of KineMed’s technology will provide us with unique insights into central nervous system disease pathophysiology”, said Seung Kwak, PhD, Director, Target Biology at CHDI. “We are excited and hopeful that this collaboration will help define the dynamics of the mutant protein in disease-‐relevant tissues, and ultimately help measure therapeutic efficacy for Huntington’s disease”.
“We are delighted to continue our collaboration with CHDI in an area that leverages our capabilities to understand the biology of neurodegenerative diseases with complex phenotypes,” said Scott Turner, PhD, Executive Vice President of Research and Development at KineMed. “This deeper understanding ultimately contributes towards targeting mechanisms at the core of the disease pathway, accelerating the development of therapeutics that can provide real hope for Huntington’s patients.” KineMed’s techniques have also been applied to investigate other pathways such as microtubule dynamics, mitochondrial turnover and axonal transport which are involved in HD and other neurodegenerative diseases.
About KineMed, Inc.
KineMed, based in California, is a world-‐leader in the identification and measurement of the dynamic biochemical processes that cause disease. KineMed’s mission is to create better ways to develop medicines and to manage disease. KineMed’s deep biological expertise combined with its core, patented, dynamic proteomics technology platform, accelerates and lowers the cost of diagnostic and therapeutic R&D.
KineMed is constantly seeking to expand its portfolio of collaborations with pharmaceutical, CRO, histopathology, diagnostics, medical instruments and biotechnology partners. Established programs with premier pharmaceutical collaborators address critical challenges facing drug discovery:
• Focus on causes rather than symptoms: Generating pivotal knowledge for developing blockbuster drugs, by targeting underlying biochemical causes
• Systems biology approach: Insight into intact living systems, rather than simplified models, ensures that drug effects are understood in their intended biological context
• Reduce late-stage attrition: Early, decision‐relevant metrics of drug activity separate winners from losers and reduce later failures to improve the NPV of R&D spend
• Powerful assays of disease state: Custom‐developed assays create companion diagnostic tests for personalized medicine
About CHDI Foundation, Inc.
CHDI Foundation, Inc. is a privately funded, not‐for-profit, biomedical research organization that is exclusively dedicated to rapidly discovering and developing therapies that slow the progression of Huntington’s disease (HD). Our scientists work closely with a network of more than 600 researchers in academic and industrial laboratories around the world in the pursuit of these novel therapies, providing strategic scientific direction to ensure that our common goals remain in focus. This helps bridge the translational gap that often exists between academic and industrial research pursuits and that adds costly delays to therapy development. In its role as a collaborative enabler, CHDI seeks to bring the right partners together to identify and address critical scientific issues and move drug candidates to clinical evaluation as rapidly as possible. Our activities extend from exploratory biology to the identification and validation of therapeutic targets, and from drug discovery and development to clinical studies and trials. More information about CHDI can be found at www.chdifoundation.org.