CHDI oversees a portfolio of projects aimed at understanding the natural onset and progression of Huntington’s disease (HD) with the goal of identifying and accelerating bringing therapies to patients.  CHDI’s strategy is anchored on the causal Huntington disease gene and the wild type and mutant huntingtin protein.  The design and execution of experimental investigations of huntingtin protein pathogenic function and its therapeutic modulation is integral to our research strategy.  We seek an accomplished and collaborative scientific problem solver who will bring innovative thinking to strategic priorities, experimental planning and execution of genetic and biochemical investigations of mutant huntingtin protein pathogenesis and develops these findings towards therapeutic programs. The position offers involvement in an innovative model of non-profit rare disease drug discovery and development and participation in an entrepreneurial and collegial work environment with opportunities and resources to contribute significantly to the understanding and treatment of HD.

Role and Responsibilities:

• Contribute to bringing forward drug discovery candidates with a focus anchored on the wild type (HTT) and mutant Huntingtin (mHTT) protein (pathogenic) function(s)  

• Play a significant role in refining and evolving the strategic plans for experimental investigation of HTT protein function with an initial primary focus on HTT/(m)HTT Post-translational modifications (PTM), enzymes modulating PTMs and protein:protein interactions

• Manage a collaborative network of contract research and academic scientists engaged in an integrated portfolio of projects to interrogate huntingtin protein function and identify and evolve new collaborations.
• Explore (m)HTT protein function encompassing genetic and biochemical in vitro investigations with human cells and tissues, mouse and human stem cell platforms, and in vivo studies including genetically modified animal models.  
• Critically design experiments, analyze and synthesize data sets
• Identify candidate protein therapeutic targets/mechanisms and design and oversee Target validation experimental plans
• Provide biology co-leadership of integrated Med chem programs with therapeutic relevance to modulating pathogenic HTT dysfunction
• Advance experimental approaches including established and emerging technologies in cells, tissues, and animals (e.g. yeast two hybrid, antibody/aptamer based technologies, bioinformatics, genome and protein engineering, protein labeling, and mass spectroscopy).
• Contribute as a member of a multidisciplinary team to the evaluation, prioritization, and implementation of biophysical, biochemical, and proteomics platforms.

• Participate in internal and external team meetings to evaluate and advance the validation of targets, outcome measures, and models for (HD) drug discovery
• Pro-actively identify, source, and integrate capabilities and competencies for current and future programs in the areas of protein function,  new assay development, and candidate drug molecule candidates for therapeutic advancement
• High level management/oversight of external contract research organizations, biotechnology collaborators, and academic laboratories
• Participate in internal and external scientific reviews
• Fulfill necessary process, legal, and business requirements to enable our scientific efforts
• Communicate the state-of-the art opportunities protein function to accelerate an understanding of HD biology and candidates for treatments to internal and external parties

• Engage collaboratively with internal team members and external partners