Program Lead, Rare Disease Drug Discovery - Takeda Pharmaceutical Company (legacy Shire Pharmaceuticals) - Cambridge, MA
(2017-01 - 2023-12)
- Experience leading cross-functional teams to support drug discovery programs in rare diseases, including programs in respiratory (COPD, asthma) and gene therapy.
- Led a program to address immune response challenges to the gene therapy platform, which involved evaluation of immune and cytokine response in human PBMCs, rodents, and NHPs.
- Nonclinical pharmacology lead on a gastroenterology program using a GLP-2 analog that led to a successful IND submission. Prepared pharmacology sections of IND, IB, other regulatory submissions, and study reports.
- Designed and executed in vitro and in vivo pharmacology studies for drug discovery research programs utilizing small peptide, protein therapeutics, and rAAV gene therapy to demonstrate PK/PD, proof of concept, drug safety, and identify lead drug candidates.
- Identified potential target engagement markers to be used to predict clinical efficacy of drug candidates.
- Delivered clinically- translatable in vivo models to preclinical programs by generating new transgenic animal models or by utilizing existing models by establishing collaborations with academic institutions.
Research Scientist III, Preclinical Pharmacology - ALEXION PHARMACEUTICALS - Lexington, MA
(2015-01 - 2017-12)
- Developed and executed preclinical studies for a portfolio of rare disease programs, including vascular calcification disorders, glycogen storage disorder, and complement nephrology disease.
- Utilized therapeutics of different modalities/platforms, including enzyme replacement therapy, mRNA LNP, and monoclonal antibody therapy.
- Designed and executed studies to investigate POC, PK/PD, mechanism of action, lead candidate selection, and biomarkers in various rodent disease models.
- Functioned as preclinical lead on multi-site/ cross-functional research teams to provide strategy for preclinical study design and animal model selection, and to achieve program milestones.
- Strategized new disease indications and drug targets for the early-stage drug discovery pipeline, including identification of target patient population and biomarker screening strategy for prospective clinical trials.
- Developed SOPs to support platform building and therapeutic program development.
Senior Research Scientist, In Vivo Pharmacology - SYNAGEVA BIOPHARMA (Acquired by Alexion Pharmaceuticals on 6/2015) - Lexington, MA
(2014-01 - 2015-06)
- Characterized preclinical animal models for evaluation of in vivo efficacy, non-GLP tolerability, and biomarker validation for protein therapeutics.
- Designed, managed, and executed in vivo pharmacology studies for rare disease biologics, specifically cardiovascular and vascular calcification disorders.
- Experience writing animal protocols and familiar with IACUC and AAALAC regulations.
- Developed key methodology to support programs, including quantitative tissue calcification assays and blood pressure monitoring in small rodents.
Senior Scientist, RNA Therapeutics - MERCK & CO., INC - West Point, PA
(2012-01 - 2013-12)
- Led efforts in a siRNA extrahepatic targeting initiative. Designed and coordinated in vivo rodent studies to investigate siRNA design and delivery strategies to extrahepatic tissues.
- Supported LNP- and GalNAc- siRNA platform development, including siRNA lead identification and optimization, and benchmarking studies in cell-based and preclinical animal models.
- Collaborated with functional leads of therapeutic disease areas to support application of the siRNA platform to target validation, mechanism of action, and target de-risking studies.
- Received award of excellence for scientific leadership of the exploratory extrahepatic initiative.
Senior Scientist, Metabolic Diseases - HOFFMANN-LA ROCHE - Nutley, NJ
(2009-01 - 2011-12)
- Designed and performed preclinical studies to investigate the role of ceramides in diabetes and metabolic disease. Performed target identification and validation studies of drug candidates involved in de novo ceramide biosynthesis.
- Validated drug targets using small molecule inhibitors and siRNA/antisense gene knockdown in myocyte and hepatocyte models of FFA-induced insulin-resistance. Conducted proof of concept studies in acute and chronic mouse models of insulin-resistance and examined effects on toxicology.
- Developed a radiochemical assay to measure ceramides and diacylglycerol, as well as a high-throughput enzymatic assay to measure levels of a target enzyme in ceramide biosynthesis.
MS, PhD candidate, Cell biology - ALBERT EINSTEIN COLLEGE OF MEDICINE - Bronx, NY
(2003-01 - 2009-12)
Principal Investigator: Philipp Scherer, PhD. Dissertation: Obesity-Associated Extracellular and Ultrastructural Changes in Adipocytes: Impact on Insulin Sensitivity.
- Conducted studies to evaluate the function of the adipose tissue extracellular matrix during obese and diabetic states. Established the novel concept of "adipose tissue fibrosis" in diabetic/obese mouse models by gene expression profiling and pathway analysis using Spotfire and Ingenuity software.
- Investigated role of collagen VI on adipocyte expansion, extracellular matrix environment, and fibrotic markers during states of excess nutrient intake in preclinical mouse models. Evaluated effects on insulin sensitivity, glucose tolerance, adipocyte apoptosis/necrosis, inflammatory markers, cholesterol and lipid markers, and energy homeostasis/ nutrient usage.
- Examined the effect of HMG-CoA reductase inhibitors on adipose tissue metabolism, focusing on a novel mechanism of action involving adipocyte differentiation, adipokine secretion, insulin sensitivity, and chemokine/cytokine secretion.
- Investigated role of caveolae trafficking on the secretion of the adipokine adiponectin and its impact on insulin sensitivity in different mouse models.
