Science Director, Protein Structure: Function and Translational Biomarkers

Job Location

The position will be based in Princeton, N.J. with occasional travel to CHDI’s other offices in LA and NYC.

Job Reports To

VP of Biology

Job Description

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. Recent human genetic studies have identified genes that modify the onset and progression of Huntington disease phenotypes. These human genetic validated genes provide entry into the discovery and development of modulators of candidate therapeutic targets. We seek an accomplished and collaborative scientific problem solver who will bring innovative thinking to approaches for understanding how these targets modify HD pathogenesis.  This position requires strategic oversight of genetic, biochemical, and pharmacological investigations into how therapeutic modulation of those activities paired with translational biomarkers can result in new treatments. Successful prosecution of these human genetic targets is integral to our research priorities. 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 advancing human genetic validated genes that modify HD as candidate therapeutic targets
  • Play a significant role in refining and evolving the strategic plans for experimental investigation of DNA repair and handling proteins as candidate therapeutic targets
  • Manage a collaborative network of contract research and academic scientists engaged in an integrated portfolio of projects to interrogate modifier protein function and identify and evolve new collaborations.
  • Explore modifier protein function encompassing genetic and biochemical 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
  • Advance a comprehensive biological understanding of the mechanism of action of the modifier proteins specifically with respect to altering the pathogenic course of HD
  • Provide biology co-leadership of integrated medicinal chemistry programs with therapeutic relevance to modulating pathogenic HD
  • 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).
  • Participate in internal and external team meetings to evaluate and advance the development of target engagement and pharmacodynamics biomarkers to support therapeutic candidates and clinical trials
  • 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
  • Fulfill necessary process, legal, and business requirements to enable our scientific efforts


The candidate of choice will have:

  • Ph D. in life sciences and/or chemistry and relevant postdoctoral training
  • A minimum of 5 years of experience as an independent innovative scientist with applied drug discovery relevance
  • A track record of scientific accomplishment as evidenced by publications in refereed journals and securing of investigator proposed research funding support
  • Expertise in investigating and elucidating protein’s mechanism of action, specifically with respect to protein:nucleic acid interactions
  • Experience and accomplishment in elucidating protein complexes and their cellular function(s)
  • Knowledge and experience in the design and characterization of protein isolation and characterization technologies
  • Training and experience in biophysics, structural and chemical biology
  • Experience with proteomics and mass spectroscopy experimental design data analysis
  • Experience in enzyme assay development and adaptation to high throughput biological assays suitable for screening
  • Significant experience in planning and conducting genetic, biochemical and pharmacologic perturbation studies directed towards elucidating protein biological mechanism of action, optimally with experience in the central nervous system and neurodegeneration
  • Drug discovery experience in validating targets for therapeutic advancement including program advancement of integrated medicinal chemistry optimization programs with clinical trajectory
  • Experience in developing target engagement and pharmacodynamics biomarkers
  • Demonstrated ability to identify, design, and drive innovative programs and demonstrated evidence of scientific “problem solving”
  • Demonstrated ability to provide scientific leadership to scientific teams
  • Strong analytical skills, critical reasoning, and scientific rigor
  • Excellent written and oral communication skills, and effective interpersonal skills necessary for the coordination of a large network of external global collaborators
  • Demonstrated ability to work collaboratively in a technical, interdisciplinary team oriented environment and with external academic and industrial partners
  • Evidence of being highly self-motivated with ability to work both independently and as a collaborative team member
  • Ability to travel to the CHDI Los Angeles CA office and also globally and domestically to interact with collaborators

Preferred Skills:

A compelling candidate would also have:

  • Experience in the biopharmaceutical sector
  • Experience working on Huntington’s disease and/or other neurodegenerative diseases
  • Experience in translating genetic results into therapeutic candidates
  • Expertise in y DNA repair
  • Experience in developing drugs for the central nervous system
  • Experience in contributing efficacy and translational data for IND filing packages
  • Productive history of having accountability for managing/overseeing external collaborators and collaborations
  • Familiarity with contemporary ‘omics technology platforms and data analyses
  • Familiarity with computational protein modeling
  • Training and knowledge of contemporary genetics and genomics

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