Establish and lead the in vivo pharmacology function to advance RNA editing therapeutics.
Lead all in vivo work from design, model development, execution, analysis and interpretation.
Design, execute and interpret translational studies for genetic medicines in mice and NHP.
Collaborate closely with therapeutic program teams on relevant in vitro models and development of translational assays.
Collaborate closely with the target discovery team on availability, feasibility and predictive power of pre-clinical models for new targets.
Provide strategic leadership on the clinical development path including regulatory filings and biomarker development.
Plan and contribute to writing regulatory submissions (e.g., IND) and biomarker strategies in collaboration with internal leaders, external consultants and KOLs.
Lead studies with external research organizations and act as a connector between internal groups.
Analyze, interpret and present data to scientists and management in internal and external venues and publish in peer-reviewed journals as appropriate.
Mentor and potentially manage direct reports; demonstrate leadership and cross-functional influence.
Requirements
PhD in biological sciences with 6+ years of post-graduate experience in relevant roles (or equivalent) in translational biology for the development of genetic medicines.
3+ years building, managing, and leading teams to meet corporate goals.
Direct experience with pre-clinical models of liver disease.
Direct experience with LNP or GalNAc-mediated delivery.
Familiarity with NGS datastreams and interpretation.
Prior experience executing studies with contract research organizations.
Breadth of therapeutic area expertise and exposure to multiple therapeutic modalities, ideally including gene editing and RNA-targeted therapies.
Proven ability to work independently and cross-functionally in a fast-paced environment.
Strong personal qualities, including attention to detail, emotional intelligence, integrity, creativity, and a willingness to have ideas challenged by team members and to challenge them.
Ability to communicate complex data sets to key stakeholders from diverse backgrounds.
Preferred: Experience with oligonucleotide or gene editing based therapeutics.
Preferred: Experience with advanced in vitro models e.g. iPSC derived cells, organoids.
Preferred: Experience planning and writing regulatory packages including IND submissions.
Preferred: Experience with clinical biomarker development strategy.
Preferred: Understanding of target discovery via human genetics.
Preferred: Network of relevant external KOLs.
Preferred: Strong computational background or demonstrated ability to interface with computational biology teams.
Preferred: Experience with multi-site project management and team leadership.