Nicholas DeMarco

As the Preclinical Scientist, I serve as the scientific lead and primary study monitor for nonclinical development programs supporting antisense oligonucleotide (ASO) therapeutics for nano-rare diseases. I am responsible for the planning, execution, and oversight of preclinical studies conducted with external CRO partners, ensuring scientific rigor, data integrity, and alignment with regulatory expectations. I review and approve study protocols, amendments, deviations, and reports while evaluating toxicology and pharmacology data to inform program strategy and go/no-go decisions. I contribute to IND-enabling activities by authoring or co-authoring nonclinical sections of regulatory submissions and briefing documents. I oversee CRO performance, timelines, and budgets, and collaborate cross-functionally with internal teams and external partners to maintain compliance with GLP/non-GLP standards and sponsor SOPs. I maintain comprehensive study documentation and tracking systems while proactively identifying program risks and implementing mitigation strategies to support efficient advancement of preclinical programs.

Led end-to-end planning, execution, and delivery of in vivo and in vitro preclinical programs across immuno-oncology and gene therapy, supporting IND-enabling and early clinical development. Directed cross-functional teams spanning pharmacology, toxicology, bioanalytical, regulatory, quality, manufacturing, and clinical operations to align scientific execution with program milestones, submission timelines, and first-in-human transitions, including advancement of an allogeneic CAR-T program. Designed and optimized disease-relevant animal models and translational biomarker, potency, and release assays, delivering high-confidence data that contributed to multiple IND packages. Managed budgets up to $2M, schedules, and risks using enterprise project-management tools, while providing executive-level reporting. Implemented and validated ELN, EDC, and GxP-compliant workflows (GLP/GCP/ALCOA+), ensuring data integrity, audit readiness, and inspection-ready documentation. Established standardized governance, communication, and risk-mitigation frameworks adopted department-wide, mentored and developed junior staff, oversaw resource planning, and drove continuous process improvements to enhance efficiency, data quality, and on-time program delivery.

Supported translational development of siRNA-based therapeutics in ophthalmology by leading and executing in vivo and in vitro preclinical and translational research programs from discovery through IND-enabling readiness. Designed, developed, validated, and optimized RNA- and protein-based diagnostic and biomarker assays—including HTRA1 detection and quantification—to support PK/PD strategies, biomarker discovery, and clinical translation. Conducted hands-on in vivo studies across mouse, rat, rabbit, and non-human primate tissues, including dosing, anesthesia, surgical administration, tissue harvest, and downstream sample processing for discovery, toxicology, and regulatory studies. Managed concurrent translational projects, coordinating internal teams, vendors, and CROs to deliver studies on time and within budget while maintaining compliance with GxP, GLP, IACUC, and ICH-GCP standards. Implemented scheduling, resource optimization, and standardized workflows; delivered cross-site training programs; and partnered with toxicology, bioanalytical, quality, clinical operations, and regulatory teams to ensure data integrity, traceability, and seamless integration of preclinical data into IND submissions and first-in-human clinical readiness.

Supported clinical-stage and commercial gene therapy programs by executing, qualifying, and reviewing cGMP-compliant in vivo functionality and potency assays for Zolgensma (spinal muscular atrophy), directly enabling batch release and post-market quality oversight. Authored and executed assay validation, method qualification, SOPs, and specifications in alignment with FDA/EMA requirements and ALCOA+ data integrity principles. Coordinated and contributed to deviation, CAPA, and OOS/OOT investigations, improving documentation accuracy and closure timelines while maintaining inspection-ready quality systems. Reviewed analytical and batch records for regulatory compliance, supported software and electronic record validation initiatives, and managed PCR-based genotyping, reagent preparation, and vivarium operations to ensure reliable assay execution. Developed and delivered technical training programs for QC staff to support trainer qualification and consistent performance of regulated assays. Partnered cross-functionally with quality, manufacturing, and R&D teams to ensure traceability, compliance consistency, and high-confidence data using STARLIMS and TrackWise in support of ongoing clinical use and commercial supply.

Led end-to-end management, development, and execution of client-sponsored preclinical pharmacology and translational research programs across immunology, inflammation, oncology, and metabolic indications, contributing data to more than 500 successful studies supporting regulatory and clinical decision-making. Designed, validated, and executed murine disease models and complex in vivo experiments, including dosing, survival surgery, necropsy, tissue isolation, and PK/PD and downstream ex vivo and in vitro analyses, delivering high-quality, reproducible data. Served as primary client interface, overseeing scope, timelines, deliverables, and cross-functional coordination with toxicology, bioanalytical, and quality teams. Authored and maintained SOPs and Animal Care and Use Protocols, ensured GLP compliance, and served as IACUC Administrator and Assistant AAALAC Quality Assurance Officer to support audits, maintain accreditation, and uphold ethical oversight. Mentored junior staff, troubleshot assay variability through root-cause analysis, streamlined documentation and reporting workflows, and improved operational efficiency while supporting client submissions and development timelines.