Science Director, RNA Biology

Job Location

The position will be based either in Princeton, N.J. or Los Angeles, CA

Job Reports To

VP of Biology, Los Angeles, CA

Job Description

The Scientific Director, RNA biologist will be responsible for the design and oversight of target and therapeutic agent identification and validation approaches in cell and animal models relevant to Huntington’s disease (HD) research. For example, developing small molecule mHTT lowering therapeutic strategies targeting: (i) unique features of expanded CAG vs non-expanded mRNA structure; (ii) regulatory sequences in HTT mRNA; (iii) induced nonsense mediated mRNA decay pathway via alternative splicing; (iv) selective modulation of mRNA translation or gene expression.

The position involves collaborating with internal scientific directors and program managers at CHDI and the management of a larger “virtual” team of external collaborators (at contract research laboratories and academic institutions) to discover and develop therapies for Huntington’s disease.

Job Role Description:

The responsibilities include:

  • Managing external CROs to develop suitable assay systems to identify and validate small molecule RNA targeting approaches in HD
  • Identifying and working closely with academic collaborators in areas relevant to RNA biology in HD cell and animal models
  • Working closely with other scientific directors for target biology validation, intervention strategies, small molecule screening and lead optimization, target engagement, pathway modulation and assessment of disease modification using various molecular, behavioral and imaging endpoints in relevant animal models.
  • Selecting and managing external vendors to providing relevant services and qualification of data and reports.
  • Writing peer-review publications and presenting internally and at relevant conferences.


The candidate of choice will have:

  • PhD in Biochemistry or Molecular Biology involving an RNA biology related discipline plus several years of relevant post PhD laboratory experience
  • 5 or more years of post-doctoral experience in the biopharmaceutical sector, with an emphasis on RNA drug discovery
  • Extensive knowledge and experience in biochemistry and biology of RNA, including analysis of RNA:protein complexes
  • Experience in designing and performing research related to RNA and translation as documented by a strong publication record
  • Experience in partnering with medicinal chemistry to develop a strategy for drug discovery program.
  • A detailed theoretical and practical knowledge of RNA biology.
  • Evidence of experience leading programs, influencing skills, team-building, cross-disciplinary collaboration, and a deep understanding of the drug discovery process.
  • Excellent written and oral communication skills.

Preferred Skills:

An ideal candidate might also have:

  • An understanding of methods to study RNA interaction with proteins and small molecule ligands
  • Experience of CNS drug discovery
  • Experience in the oversight and management of contract research organizations.
  • A publication record showing creative thinking as well as evidence of cross-functional team work.

Send CV’s to