CHDI Foundation and KineMed established a collaboration to exploit the latter’s tandem mass spectrometry-based proteomics platform to analyze the in vivo dynamics of mutant huntingtin protein as a means of assessing Huntington disease progression and measuring the efficacy of therapeutic candidates. The aim is to generate data that will both provide further insights into HD pathophysiology and also a means for determining whether investigational drugs impact on protein production and clearance.
The mutant huntingtin protein responsible for HD exhibits an expanded region of polyglutamine (polyQ) amino acids, and the size of the expansion correlates with disease severity. Through the collaboration with CHDI, KineMed will use its isotopic labeling and dynamic proteomics platform to determine parameters including huntingtin protein half-life and synthesis rate as a function of polyQ length.
“The application of KineMed’s technology will provide us with unique insights into central nervous system pathophysiology,” comments Seung Kwak, Ph.D., director of 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 HD.”
KineMed’s platform leverages ultrasensitive isotopic tracer technology to reveal the dynamics of causal pathways in disease. The firm is exploiting its technologies both through disease- and biomarker-focused collaborations with pharma and biotech partnerships, and also to develop an internal drug and extensive biomarker pipeline.
The firm’s lead therapeutic program is an NIH-partnered apo-A1 mimetic (designated FX-5A), which is in development for the potential treatment/reversal of atherosclerosis and heart disease. In April KineMed was awarded a Rapid Access to Intervention Development grant by NIH to support formulation, manufacturing, and IND-enabling studies with FX-5A. KineMed’s internal drug development programs also include noscapine and noscapine analogues for the treatment of neurodegenerative diseases.