Preclinical Senior Research Associate
CurrentDevelop curative gene therapies using a variety of novel CRISPR associated editing systems such as nucleases, base editors, and prime editors.Assist in developing a comprehensive competitive landscape for different gene therapy indications.Propose therapeutic approaches for new indications and initiate early stage R&D.Closely work with external biotech partners to create goals and meet deadlines on joint projects.Independently propose, research, implement, and teach new processes with relevance to both company/ project objectives.Present complicated research in an easily digestible format to a broad audience with varying levels of scientific expertise.Research responsibilities:Collaborate with the early discovery team to employ novel editing technologies such as nucleases, base editors, and prime editors in therapeutically relevant models. Develop and optimize protocols to screen guides across different nuclease systems in a variety of cell lines, including primary hepatocytes, neural progenitor cells, myocytes, and iPSC using a variety of transfection methods (lipofection, nucleofection, and LNPs).Formulate and manufacture Lipid Nanoparticles (LNPs) for use during In Vivo studies.Generate novel LNPs to increase ex-hepatic specificity Establish assays for the quantification and characterization of RNA.Design qPCR and ddPCR assays to quantify gene expression levels for targets of interest.Design NGS (Illumina short read) assays to qualitatively analyze gDNA and RNA variants post-editing.Design long read (Oxford Nanopore) assay to characterize RNA variants and quantify the frequency of different outcomes.Develop RNAseq pipeline and work bioinformaticians to analyze data.Create novel assays to improve off target profiling, specifically for Type V CRISPR systems, and work cross functionally to get initial proof of concept tested.Establish medium throughput methods for harvesting soft tissues and isolating high quality gDNA/ RNA for downstream analysis.