The Company

We are building the first inherently non-immunogenic and broadly-applicable RNA editing platform. By drawing from basic principles in RNA processing and recent advancements in gene therapy, our therapeutic platform can be used to halt or reverse the progression of hundreds of human diseases. We are looking for ambitious and talented individuals to join our core team. Success in this position will mean making central contributions to the creation of new therapeutic technology platform and eventually human therapeutics. As an early-stage company, applicants should be interested in growing with the company by expanding their scientific and professional abilities and responsibilities.

Our founders are entrepreneurs and scientists whose experience includes building therapeutic technology platforms, running clinical trials, and raising tens of millions of dollars in venture capital funding. We plan to rapidly expand to a range of therapeutic programs by utilizing automation and high-throughput sequencing to compare iterations of our therapeutic platform.

The Role

Scientist/Sr. Scientist- RNA editing therapeutic development and AAV gene therapy. Our platform can be used, in principle, to address any mutation on the RNA level. This multifunctional role will involve combining basic understanding of gene therapy and disease modeling with automation, transcriptomics, and screening techniques to motivate data-driven identification of candidate therapeutics. Success in this position will involve the development of an in vitro to in vivo development pipeline that generate potential candidate therapeutics for clinical development. Your decisions will directly inform filings with the FDA in pursuit of clinical development of new human therapeutics.

How You Will Contribute

In this role, you will create a scientific strategy by developing an in-depth understanding of our platform and workflows. You will be responsible for optimizing our technology platform as well as identifying new capabilities. You will also be responsible for conducting in vitro and in vivo characterization of therapeutic activity in tissue culture and mouse models of human disease. Assays will include transcriptome-wide analyses as well as phenotyping on a disease-by-disease basis. This is a highly collaborative position in which you will coordinate execution of strategy by integrating across the many disciplines represented within the company, from molecular biology to disease biology, automation, high-throughput sequencing, and in vivo modeling.

Responsibilities:

• Evaluate potential human therapeutics in vitro
• Identify high quality potential therapeutics and oversee packaging in gene therapy vectors
• Oversee in vivo studies in genetic mouse models of human disease (brain, eye, and muscle)
• Develop workflows involving high-throughput sequencing to compare on- and off-target activities in the transcriptome
• Develop and refine assays to compare therapeutic efficiencies
• Interpret varied readouts of therapeutic efficiencies and develop assays that identify clinically-relevant phenotypes
• Work with a range of cell biology tools
• Contribute to design of animal studies and
• Collaborate with a diverse team of wet-lab and computational scientists

Who you are

• Enjoy working in a tight-knit and focused team
• Intellectually flexible
• Ability to reduce hypotheses into experiments that will provide actionable information
• Thoughtful & detail oriented
• Decisive
• Stimulated by work at the intersection of multiple fields

Basic qualifications

• PhD in biology, biochemistry, biomedical engineering, or a related field
• Strong track record leading cross-functional projects
• 5+ years experience in molecular biology
• 3+ years experience in in vivo studies
• Expertise in transcriptomics— library preparation, sequencing, splicing/gene expression analysis

Preferred qualifications

• Experience with AAV production for in vivo studies
• 2+ years post PhD experience
• 3+ computational biology/transcriptomics experience
• Mentoring and managing experience

We are an equal opportunity employer and value diversity at our company. We do not discriminate on the basis of race, religion, color, national origin, gender, sexual orientation, age, marital status, veteran status, or disability status.