Jennifer Broderick Email & Phone Number

I make RNA medicines that combine tissue-specific target silencing with the precision of oligonucleotides. Solving the on-target, off-tissue toxicity problem is crucial to provide safer medicines to patients. Our technology is a discovery platform for creating RNA drugs, without dose-limiting toxicity, that surpass current standard of care in many disease areas. Our technology represents a breakthrough in precision medicine because it harnesses the potent gene silencing function of endogenous microRNAs (miRNAs), unlike siRNA or antisense oligonucleotides (ASO) therapeutics. We have demonstrated that single-stranded oligonucleotide “tethers” can be designed to recruit tissue-specific miRNAs to mRNAs that are not normally regulated by the recruited miRNA. Once recruited, the tethered miRNA can repress mRNA expression in cultured mouse and human cells and in mouse liver and skeletal muscle. Tether-mediated gene silencing occurs only in one or a small number of tissues, irrespective of the specificity of tether delivery, because silencing occurs only in those cells that produce a specific miRNA. Tether-mediated gene silencing is one of few therapeutic RNAi strategies that limit gene silencing to the targeted cell type even when the therapeutic agent is delivered systemically to many tissues. miRNAs with increased expression as a result of a particular pathological state can be recruited to silence an mRNA whose expression drives the development of disease (OncomiR to silence mRNA only in tumor cells or viral miRNA to silence viral or host gene only in infected cells). This platform will enable rapid validation of many clinically relevant extra-hepatic mRNA targets.Our goal for this platform is to generate a pipeline of best-in-class RNA drugs that ensures frequent value creation events through partnership agreements with pharmaceutical companies.

Listed skills include Genome Editing, Rnai, Gene Therapy, Molecular Biology, and 25 others.