Manage preclinical development activities for lead programs in human health and animal health. Responsibilities include:• Evaluation and selection of CROs for IND-enabling tox/BD studies• Management of all preclinical studies• Edit/approve study reports• Author nonclinical sections of regulatory filings to FDA (CBER and CVM)• Provide input and support for discovery stage programs

Led discovery and translational research efforts for a clinical stage gene therapy company focused on regeneration of neurons lost in neurodegenerative disease. Managed cross-functional team (CMC, Nonclinical, Clinical, and Regulatory) to deliver Pre-IND Briefing Package to FDA for company’s lead program.Areas of research focus:• Neurodegenerative disease and CNS injury – oversee all pipeline targets including stroke, Huntington’s Disease, traumatic brain injury, Alzheimer’s Disease, and spinal cord injury• mRNA delivery – design and execute studies to develop nonviral methods for transgene expression in vivo• AAV vector engineering – oversee efforts to improve targeted delivery of AAVs and enhance safety profilesKey responsibilities:• Lead discovery and translational research efforts including coordination of AAV manufacturing, design and execution of IND-enabling nonclinical studies, and clinical trial design• Manage company operations including selection of laboratory space, build-out of facilities, and hiring of research team• Facilitate partnership with Spark Therapeutics to advance program in Huntington’s Disease• Identify KOLs to establish and execute research collaborations and build company’s Scientific Advisory Board

Employee #3 (after 2 co-founders) for a startup biotech company spun out of Janssen Research & Development to advance Centyrin platform technology for targeted, extrahepatic delivery of oligonucleotides (siRNA and ASO) and other payloads. Equipped lab space, hired a team of biologists, designed and executed key experiments resulting in Series A financing.Areas of research focus:• Extrahepatic delivery of oligonucleotides – Led efforts to improve PK/PD properties of siRNA and ASO conjugates in vivo• Neuromuscular disease – Discovery Lead for project ABX1100, targeting mRNA in Pompe Disease with a Centyrin-siRNA conjugate• Oncology – Led project ABX900, targeting EGFR and cMET in NSCLC; and ABX300, targeting KRAS mRNA with a Centyrin-siRNA conjugateKey Responsibilities:• Manage a team of scientists (6 direct reports, PhD/M.S.) to design and execute all biology experiments, in vitro and in vivo• Identification and validation of novel targets for drug discovery• Work with CROs to execute all in vivo studies including rodent PK/PD, xenograft studies, transgenic models of neuromuscular disease, and non-human primate PK/PD• Generate datasets, slides, and answers to investor questions to support fundraising • Interact with biopharma partners and KOLs to advance platform technology, including efforts to cross the BBB and enhance AAV targeting using Centyrin-capsid fusions

Spearheaded the biological side of drug discovery efforts in a startup biotech company. Identifed novel targets for possible therapeutics, performed target validation using in vitro and in vivo models, and developed assays to screen candidate therapeutics (both small molecules and peptides). Worked with CEO to shape company direction and target external funding sources.Key responsibilities:• Identification and validation of novel targets for drug discovery• Development of in vitro models of disease for use as assays of candidate therapeutics• Management of all in vivo studies including ADME/PK and efficacy models

Served as Senior Scientist and Study Director on projects covering a broad spectrum of disease areas with an emphasis on gene therapy, regenerative medicine, and stem cell research. Extensive experience with disease modeling in vitro and in nonhuman primates (NHPs). NHP studies conducted at the St. Kitts Biomedical Research Foundation.Areas of research focus:• AAV vector development and delivery for gene therapy applications• Biomarkers of aging and disease in NHPs (using SOMAscan proteomics platform)• Alzheimer’s disease – characterization of pathology and biomarkers in NHPs• Generation and genetic modification of stem cells, neurons, and cardiomyocytes• Immunophenotyping of NHPs using flow cytometrySenior Scientist responsibilities include:• Execution of internal R&D projects• Identification of new disease models for potential commercial application• IACUC protocol preparation• Data analysis• Report and manuscript preparation• Grant writing• Management and training of research staff• Business development through outreach to commercial and academic partnersStudy Director responsibilities include:• Preparation and optimization of study protocols• Pre-study planning, including resource management• Study execution and oversight• Data analysis and preparation of final reportsGLP: Completed GLP Training for Study Directors & Study Monitors, Hood River, OR - 10/2014

Mentors: John Gearhart and Jonathan Epstein• Worked across departments to build teams of cardiologists, electrophysiologists, and neuroscientists to develop new strategies for regenerative medicine• Developed and published novel system to generate dopaminergic neurons via direct reprogramming of astrocytes for application to Parkinson's Disease• Filed provisional patent disclosure #Y6288, May 2012: “Direct conversion of astrocytes into dopaminergic neurons using a single polycistronic vector.” • Engineered dynamic fluorescent reporter system for identification and characterization of functional cardiomyocytes• Optimized direct conversion of fibroblasts to cardiomyocytes for regenerative medicine applications• Isolated human cardiac myocytes and cardiac fibroblasts from failing and non-failing hearts in Penn's Heart Failure & Transplantation Research Lab

• Completed and published graduate research on dopaminergic neuron development and embryonic stem cell biology• Designed and executed studies using rodent models of Parkinson’s Disease, cardiovascular disease, and Down Syndrome