Workforce Genetics is recruiting for a Principal Scientist for a start-up focused on the development of RNA therapeutics.
Overview:
Our client is a rapidly growing biotechnology company focused on the development of RNA therapeutics to treat a variety of diseases. The company's genomics-based discovery platform is designed to create best-in-class RNA therapies. This platform can identify critical RNA targets for drug development in specific subsets of patients that would benefit from treatment. They then create proprietary chemically-modified RNA therapeutics with improved pharmacology, while utilizing antibodies for targeted delivery to the diseased tissue. The company is developing a treatment for Facioscapulohumeral muscular dystrophy (FSHD). This treatment will eliminate expression of the DUX4 gene, which causes the disease in 95% of patients, halting disease progression and vastly improving quality of life.
Responsibilities:
- As the lead scientist on the FSHD program, you will be responsible for establishing and executing preclinical development plans.
- You will focus on optimizing in vitro discovery assays, and directing mechanism of action and pharmacology studies in animals to identify and validate our lead candidate.
- You will also work closely with the VP of Research to gain exposure to CMC, TOX, and pharmacology studies, as well as with drug synthesis partners to manufacture and scale up candidates in preparation for a Pre-IND or IND meeting.
- Success in this role will lead to management of a team of direct reports pursuing our FSHD program and future related programs.
Preferred Qualifications:
- A PhD in biology, pharmaceutical sciences, life science technology or related field.
- Experience in muscular dystrophies, myopathies, neuromuscular or other musculoskeletal disorders is a must.
- Experience in delivery of RNA therapeutics is a plus.
- Experience in managing outside vendors and internal team members is preferred.
- Evidence of scientific acumen in the form of peer-reviewed publications and patents
- Ability to manage multiple projects and execute effectively on pre-determined timelines.
- Ability to lead and motivate colleagues, and help build a positive and productive company culture.