Rewriting RNA

Pioneering targeted therapies for serious muscle diseases

• Built translational research programs focused on developing Antibody, Enzyme replacement and therapeutic RNAs as medicines for rare and devastating diseases• Led a new research initiative at Alexion to develop RNA therapeutics for the treatment of rare and ultra-rare diseases• Established and led a new state-of-the-art RNA laboratory in Cambridge, MA

A start-up biotechnology company focused on selectively activating protein expression by modulating the interaction between PRC2 and long non-coding RNA1. Formulated, initiated and executed research strategy to validate novel long non-coding RNA technology2. Externalized in vitro/in vivo studies, coordinated project timelines/deliverables with CROs3. Directed research to identify/nominate candidate leads for therapeutic indications4. Key contributor to company research, business and IP strategy5. Initiated and managed external research collaborations with consultants/academic collaborators6. Presented scientific results to investors and at international scientific meetings 7. Expanded RaNA research team to 14 FTEs with research and budgetary oversight8. Worked with Foundations and Government organizations to obtain non-dilutive funding

1. Group leader with responsibilities to set research direction, manage colleagues (line and matrix) and nominate lead molecule for IND submission2. Progressed lead candidate through in vitro and preclinical testing to pre-IND stage3. Led Oncology project teams from target validation to lead identification and nominated lead molecule as CandidateOrganized and executed cross-site oncology & metabolic disease projects to prioritize drug candidates and enable patient stratification for clinical trials based on pathway and phenotypic screens4. Project Leader - Cancer cell metabolism. Led in a matrix role and integrated scientists from Biology, Chemistry, ADME, Systems Biology, Biological Profiling, and the Oncology Business Unit to identify the Target Product Profile and clinical feasibility5. Led research teams in drug discovery, translational medicine, biomarkers and personalized medicine6. Developed target dependent cell-based assays for the Pfizer Kinase Program to identify translational biomarkers for mechanism of action and efficacy7. Project Leader - Duchenne Muscular Dystrophy. Built in vitro and in vivo team and developed assays to ascertain delivery, biodistribution and efficacy of exon-skipping oligonucleotides. Managed collaboration with leading academic scientists and CROs8. Biology lead on oligonucleotide therapeutic target - Established in vivo pharmacology models to validate target and nominate lead for progression to IND9. Evaluated compound delivery & efficacy on cancer stem cells in solid tumors10. Determined effects of RNAi on target and pathway genes, andmiRNA signatures of human tumors

Led in vitro/in vivo cell biology team and implemented cell-based assays to evaluate mechanism of action and rank compound potencyDesigned rodent studies, evaluated compound PK/PD and efficacy in close collaboration with CROs and academic labsProvided business development support for VC presentations, scientific support for IP filing and pre-IND FDA meeting

Discovery/validation of novel kinase inhibitors for cancer therapyDesigned and implemented cell-based assays for signal transduction mechanisms (apoptosis, oncogenic transformation and cell proliferation)Applied gain of function screens to identify oncogenic kinases across the kinome Generated human tumor xenograft models of melanoma and CRC in nude miceEvaluated activity of compounds and RNAi in tumor regression in vivo

Function of 14-3-3 isoforms in cell survival and apoptosisDetermined the functional similarity between different 14-3-3 isoforms to inhibit Bad-induced apoptosisDissected the structural determinants for 14-3-3 interaction with ASK1Ascertained the chaperone function of 14-3-3 in ASK1 sub-cellular localization