Science Director – Target Validation and Drug Discovery

Job Location

The position will be based in Princeton, N.J. with occasional travel to New York City and Los Angeles.

Job Reports To

Vice President, Drug Discovery Genotype & Phenotype Unit

Job Description

Drug Discovery Scientist with role and responsibilities to contribute to the design, implementation, and management of target-based drug discovery campaigns from Human Genetic based Target candidates to Assay Development to Lead Generation. The position involves collaborating with internal CHDI scientists and external academics, and contract research organization collaborators to accelerate new therapies for Huntington’s disease (HD).

Job Responsibilities:

The responsibilities include:

  • Therapeutic validation of candidate targets emerging from human genetic studies that identify modifiers of HD onset and progression for therapeutic modulation (current focus is on DNA mismatch repair proteins)
  • Project team lead for novel targets emerging from human genetics. Advance the understanding of the mechanism of action of candidate targets
  • Co-lead and collaborate with team members to conceptualize, implement, validate, and integrate assays into an efficient flow scheme for candidates to therapeutic programs
  • Project lead for the development and qualification of biochemical, biophysical, and cell-based assays suitable and deployable for drug discovery campaigns e.g., high-throughput screening and downstream assays.
  • Collaborate with Platform teams to identify and deliver reagents to support target advancement (Genetically modified animal models, protein production, antibody production, structural biology).
  • Co-Lead the development of proof-of-concept tool molecules across a range of modalities (small molecule, RNAi, CRISPR)
  • Contribute to development of standard operating protocols for assays and for data qualification and registration.
  • Develop and participate in project teams to achieve milestone-based goals to rapidly identify proof-of-concept candidates for in vivo testing in animal models of HD
  • Review and approve detailed reports for each project and campaign in area of responsibility
  • Forge and oversee academic collaborations relevant to human genetics targets
  • Lead and oversee contract organization scientists for implementation of drug discovery goals
  • Participate in technology assessment and implementation activities
  • Communicate results in presentations at scientific meetings and by co-authoring publications


The candidate of choice will have:

  • A PhD in Pharmacology, Biochemistry, Enzymology, Molecular/Cell Biology, or Chemical biology
  • Proven scientific track record in early drug discovery projects emphasis on early drug discovery in multiple target classes from hit qualification to lead identification phase with >5 years in the pharmaceutical/biotechnology sector.
  • Experience with molecular machines-protein complexes and specifically protein-nucleic acid complexes.
  • A detailed theoretical and practical knowledge of enzymology, biophysical and cell-based assay development in drug discovery programs.
  • Experience with establishing target engagement and pharmacodynamic endpoints.
  • Demonstrated track record of co-leading drug discovery project teams, partnering with Medicinal Chemistry/DMPK and Structural biology teams
  • Significant experience co-leading targets & projects through sanctioning milestones, e.g., hit qualification and lead declaration
  • Rigorous data analyses and interpretation
  • Knowledge and experience with industry standard quantitative biology tools, technologies, and vendors.
  • Experience in managing external contract research organization collaborations
  • Experience in managing biopharma:academic collaborations
  • Excellent written and oral communication skills
  • Demonstrated ability to both lead and be a team player in an interdisciplinary team-oriented environment

Preferred Skills:

An ideal candidate would also have:

  • Expertise in quantitative biology/enzymology/cell biology
  • Evidence of creative problem solving
  • Expertise in DNA repair biology
  • Expertise in ATPases and protein-protein interactions
  • Bridging biochemical and structural biology in drug discovery programs
  • Expertise in working in the CNS and neurodegeneration drug discovery
  • Experience with high content imaging technologies for cell-based assays.
  • Experience working with a diversity of cell-based assays including stem cells and derivativesA publication track record demonstrating rigor and evidence of cross-functional teamwork

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