Technical consult on RNA biopharmaceutical design, process development, and manufacture

- Led a new development initiative at Alexion for non-viral formulation and delivery of mRNA drug candidates for the treatment of rare diseases- Oversaw characterization and preclinical (rodent and NHP) studies to define critical quality attributes of mRNA therapeutics- Developed production and purification processes for mRNA therapeutics that reduced COGs while maintaining product quality- Initiated and managed external collaborations with industry and academic partners as well as CMO/CROs

- Designed and performed in vitro and in vivo studies to evaluate efficacy, safety, biodistribution, physicochemical properties, and stability of nucleic acid delivery technologies- Established laboratory capabilities and external network for synthesis and characterization of nanoparticle formulations- Provided analytical and formulation support for nucleic acid discovery programs- Contributed to search and evaluation of external nucleic acid delivery and therapeutic opportunities

Pursued RNAi-driven cancer therapy research in the laboratories of Frank Slack, Mark Saltzman, and Don Engelman. The overarching goals of my work were to elucidate the roles of microRNAs in the onset and maintenance of cancer, and to engineer therapeutic systems to exploit these lynchpin biological targets.Specialties: RNA interference, nucleic acid biology, microRNA therapeutics, nanotechnology, cancer research, and tumor biology.

Dissertation: Ligand-enhanced polymeric nanoparticles for targeted RNA-based gene therapyAdvisers: Mark Saltzman, Don Engelman, Lynne Regan, and Frank SlackDeveloped and characterized novel multifunctional polymer nanoparticles coated with cell-penetrating peptides and targeting ligands that effectively delivered RNA-based gene therapy to induce gene knockdown, microRNA inhibition, and alternative splicing. Utilized these nanoparticles to treat a variety of cancer models, most notably a tumor model of lymphoma (in mouse) that was dependent on the induction of a specific microRNA oncogene; nanoparticle treatment showed therapeutic effects through the delivery of anti-microRNA gene therapy.Supervised research on the following projects: loading of siRNA into nanoscale delivery vehicles, optimization of nanoparticle synthesis processes, breast cancer chemotherapy delivered by folate-enhanced nanoparticles, enhancing nanoparticle delivery through functionalization with cell-penetrating peptides, delivery of glioma-targeted miRNA therapeutics, and enhancing nanoparticle delivery using biomimetic polymers