Tom Hartl

Drug Discovery Scientist & Program Leader | Oligonucleotides, Small Molecules & Gene Therapy | Target Evaluation & Translational Strategy

San Francisco, California, United States

About

Throughout my career, I have been motivated by three things: scientific discovery, helping talented scientists grow, and advancing therapies that improve patients' lives. My training in genetics, molecular biology, and cell biology has provided a broad scientific foundation that I have applied across therapeutic discovery and development in rare disease and genetic medicine. Over the past 15+ years, I have worked across small molecule, antisense oligonucleotide (ASO), and gene therapy programs, contributing to target evaluation, assay and screening platform development, lead identification and optimization, translational strategy, and program leadership. I enjoy integrating diverse scientific, operational, and competitive information to evaluate opportunities, prioritize candidates, and make data-driven decisions that advance programs. A recurring theme throughout my career has been building bridges between disciplines. I enjoy working closely with scientists, clinicians, pharmacologists, toxicologists, CMC experts, and business partners to translate scientific discoveries into therapeutic strategies. Whether developing new screening systems, investigating disease biology, leading cross-functional teams, or mentoring junior scientists, I am energized by solving complex problems and helping teams move promising ideas toward meaningful impact for patients.

Experience

  • BioMarin Pharmaceutical Inc. (9 yrs)
    • Director and Principal Scientist in Musculoskeletal Therapeutics
      Mar 2025 - Apr 2026 · 1 yr 2 mos

      Therapeutic discovery and development for inherited rare diseases. As project and team leader, experienced in oligonucleotide, gene therapy, and small molecule approaches for rare genetic muscle, metabolic and heart diseases.

    • Associate Director, Cell Biology Lead, Musculoskeletal Research
      Mar 2022 - Mar 2025 · 3 yrs 1 mo

    • Senior Scientist I
      Sep 2020 - Mar 2022 · 1 yr 7 mos

  • Honorary Associate Professor at Keck Graduate Institute
    Aug 2024 - Apr 2026 · 1 yr 9 mos

  • Honorary Associate Professor at Dominican University of California
    Sep 2022 - Aug 2023 · 1 yr

  • Senior Scientist and Director of High Throughput Screening at Perlara PBC
    May 2014 - Apr 2017 · 3 yrs

    Applied molecular genetics to aid in the discovery of new small molecule therapies for patients with rare diseases. Developed high throughput screening platforms to find new compounds that will reverse or prevent the onset of these cellular disease states.

  • Post-Doctoral Researcher at Stanford University
    2010 - May 2014 · 4 yrs 5 mos

    Conducted proteomics (SILAC) and genetic methods with fruit flies and mammalian tissue culture to gain a greater insight into Hedgehog (Hh) signal transduction. Made the discovery that levels of a particular phospholipid are essential for Hh signaling, thus laying the groundwork for future therapeutic methods to manipulate these phospholipids in cancers generated by hyperactive Hh signaling.