Head of Discovery

OCUGEN OPCO INC • East Whiteland Township, PA

5d • Onsite

About The Position

The Head of Discovery Research will lead Ocugen’s discovery engine—defining scientific strategy, advancing early-stage programs, and building a high-performing organization that delivers IND-ready candidates. This is a highly visible leadership role responsible for shaping the next generation of gene therapy programs, with a focus on translational science, target validation, and biomarker-driven development. You will partner closely with R&D, Translational Medicine, CMC, and External Innovation to move programs from concept to IND-enabling milestones.

Requirements

• PhD (or MD/PhD) in relevant life sciences molecular biology, genetics, cell biology, neuroscience, ophthalmology/visual sciences, or biomedical sciences with a focus on gene therapy or rare disease).
• 15+ years of research experience in discovery and early translational settings, with increasing leadership responsibility; direct experience in ophthalmology, retinal disease, or neurological disease research is required.
• Demonstrated success leading discovery teams that advanced candidates through preclinical development, including IND-enabling packages for gene therapy or genetic medicine programs.
• Track record of scientific innovation with significant contributions (peer-reviewed publications, patents, or novel platforms) in the areas of gene therapy, ocular biology, neurodegeneration, or rare genetic disease.
• Ability to work collaboratively and influence across functions, with demonstrated experience translating genetic and molecular insights into therapeutically actionable programs.
• Deep expertise in human genetics and genomics, including interpretation of variant data, GWAS findings, and genotype-phenotype correlations relevant to ophthalmic and neurological conditions.
• Strong foundation in molecular and cellular biology techniques, including gene editing, viral vector biology, transcriptomics, proteomics, and iPSC-based disease modeling.
• Experience in biomarker discovery, assay development, and translational biomarker strategy to support IND submissions and early clinical trials.

Nice To Haves

• Experience at a clinical-stage company transitioning discoveries to human studies, ideally within gene therapy, ocular medicine, or rare neurological disease.
• Broad modality experience, including gene therapy, biologics, or small molecules, with primary depth in AAV-mediated gene therapy for retinal or CNS applications.
• Expertise in ocular biology and retinal disease mechanisms strongly preferred, including inherited retinal dystrophies, age-related macular degeneration, glaucoma, and neurodegeneration with ophthalmic manifestations.
• Experience managing external research collaborations and academic partnerships with leading ophthalmology and neuroscience research institutions and retinal disease foundations.
• Familiarity with regulatory expectations for gene therapy IND filings, including FDA CBER guidance and relevant ICH guidelines for advanced therapies.

Responsibilities

• Scientific & Portfolio Leadership Define and execute discovery strategy across gene therapy and emerging modalities Build and prioritize a discovery portfolio with clear milestones, decision criteria, and success metrics Ensure programs are grounded in human genetics, genotype–phenotype relationships, and translational relevance
• Program & Translational Impact Lead target identification, validation, and early in vivo proof-of-concept in relevant disease models Integrate genetic, transcriptomic, and functional data to prioritize high-confidence targets Advance biomarker strategies (pharmacodynamic, predictive, and patient stratification) to support IND-enabling studies and early clinical design Establish and leverage disease-relevant cellular and in vivo models (including retinal and CNS systems)
• Innovation & Platform Development Integrate emerging technologies such as CRISPR-based screening, single-cell genomics, iPSC-derived models, and advanced organoid systems Drive adoption of novel approaches to improve discovery efficiency and translational success
• Team & Organizational Leadership Build, lead, and mentor a high-performing, cross-functional discovery team Foster a culture of scientific rigor, innovation, and accountability Attract and develop top-tier scientific talent
• Cross-Functional Integration Partner with Translational Medicine on biomarker and model development predictive of human biology Collaborate with CMC and Process Development to ensure early manufacturability and feasibility of gene therapy constructs Align discovery programs with clinical strategy, unmet need, and competitive landscape
• External Innovation Establish and lead collaborations with academic institutions, biotech partners, and scientific consortia Evaluate licensing opportunities and external platforms (e.g., capsid engineering, delivery technologies) to expand capabilities Represent Ocugen in scientific forums and publications Maintain visibility in scientific communities through publications and presentations in leading ophthalmology, neuroscience, and gene therapy journals and conferences (e.g., ARVO, SFN, ASGCT).
• Governance & Risk Management Chair or participate in Discovery Portfolio Review and Scientific Steering committees, providing scientific leadership on target selection, go/no-go criteria, and resource allocation across ophthalmology and neurology programs. Ensure sound risk assessment and mitigation early in research planning, including genetic and molecular target tractability assessments and immunogenicity considerations for gene therapy approaches. Drive decisions with rigor, transparency, and data-driven judgment.

What This Job Offers

• Job Type: Full-time
• Career Level: Manager
• Education Level: Ph.D. or professional degree
• Number of Employees: 11-50 employees