Senior Scientist Molecular/Cellular Biology

About the company:

We are a biotechnology company developing a new class of genomic medicines based on our platform of gene targeted chimera small molecules. The molecules are designed to either turn-on or turn-off specific disease-causing genes to address the underlying cause of disease. The company's lead program is focused on the treatment of Friedreich ataxia, followed by a program in myotonic dystrophy type-1 and discovery efforts for multiple other serious degenerative disorders caused by nucleotide repeat expansions.

We are a group of passionate and experienced drug developers based in Carlsbad, California and since our inception in 2017 we have raised over $400 million from leading healthcare investors. Following our successful IPO in March 2021 we are advancing our lead programs and expanding our pipeline.

job title: Senior Scientist/Associate Director - Molecular/Cellular Biology

Position Summary:

This scientist will be a key member of the team responsible for driving an early-stage discovery effort in new disease areas. This will include designing and implementing biochemical and cellular assays for assessing GeneTAC efficacy. This position will collaborate closely with colleagues working on compound design and in vivo performance. Core technologies used include tissue culture of primary and differentiated cells relevant to the disease, molecular characterization of gene expression and chromatin marks, and cytology. In addition to designing, conducting, and interpreting experiments, the successful candidate will help identify new areas for discovery research, be a member of cross functional teams that set program strategy, objectives, and project plans, and manage work with outside collaborators.

Responsibilities:

• Develop expertise in new disease and technical areas by reading literature and consulting with outside experts. This includes a detailed understanding of the molecular / transcriptional / epigenetic dynamics at play in genetic diseases of interest, and use this knowledge to devise strategies to treat disease using GeneTACs
• Develop and conduct cellular, molecular, and biophysical assays to support mechanistic research and lead optimization campaigns. Cellular systems include iPSC differentiated cells from patients. Molecular assays include transcriptional assays (RT-qPCR, RNAseq, ddPCR), epigenetic assays (ChIP-Seq, Cut and Run, etc.), protein assays (western, ELISA). Biophysical assays include SPR.
• Independently develop technical science plans including development and deployment of new technical approaches
• Collaborate with clinical and academic partners
• Contribute to cross functional project teams responsible for drug development, from target selection to lead identification to ongoing development
• Management of Research Associate(s)

Qualifications:

• Ph. D. in relevant field and post-doctoral experience required
• Deep experience in gene regulation required
• Hands-on experience in developing molecular methods required, ideally including NGS and RT-qPCR
• Hands-on experience in mammalian cell culture required, ideally including iPSC experience
• Hands-on experience in wide variety of cell biology techniques required, e.g., immunohistology, FISH, NanoBRET, etc.
• Willingness to learn and support in vivo experiments as needed.
• Demonstrated ability to problem solve and multitask in a fast-paced biotech environment
• Effective oral and written communications
• Industry drug discovery experience preferred

Mikey Ryerson

Mikey@peoplewithchemistry.com

858-429-6069