Chief Scientific Officer

About the Role

We are seeking a visionary and hands-on Chief Scientific Officer to lead our R&D strategy as we transition from discovery-driven science to a clinical-stage organization. This is a scientist-leader role requiring a balance of blue-sky innovation and disciplined execution to file our first IND and advance VRG-201 into the clinic. You will own the entire nonclinical science stack—from target identification and vector engineering to CMC scale-up and regulatory toxicology—while serving as the external face of the science to investors, regulators, and academic partners. Internally, you will cultivate a culture of intellectual fearlessness and operational rigor.

Responsibilities

• Strategic Leadership
  • Define and drive the long-term R&D roadmap, prioritizing pipeline assets based on unmet medical need, competitive differentiation, and commercial viability.
  • Lead the transition from research to development, ensuring seamless handoffs between discovery, nonclinical safety, and CMC teams.
  • Partner with the CEO and Board on corporate strategy, fundraising, and business development opportunities (including in-licensing and strategic alliances).
• Scientific Execution
  • Oversee all nonclinical programs, including vector design, LNP formulation, in vivo efficacy models, PK/PD, and GLP toxicology studies.
  • Champion our LNP delivery platform and tissue-targeting strategies, driving continuous innovation to maintain a competitive edge.
  • Ensure a robust CMC strategy for plasmid production, LNP manufacturing, and analytical methods suitable for Phase I/II clinical trials.
• Regulatory & External Engagement
  • Lead scientific interactions with the FDA (CBER) and other global health authorities, including pre-IND meetings and IND-enabling package preparation.
  • Represent the company at scientific congresses, investor presentations, and engagements with key opinion leaders (KOLs) in neuromuscular and CNS gene therapy.
  • Cultivate and manage academic collaborations with Duke, UNC, and other translational research institutions.
• Team & Culture
  • Build, mentor, and retain a world-class R&D organization of ~40 scientists and associates.
  • Foster a culture of scientific rigor, intellectual honesty, and collaborative problem-solving.
  • Champion diversity of thought and create an environment where bold hypotheses are welcomed.

Requirements

• Essential
  • Ph.D. in Molecular Biology, Genetics, Bioengineering, or a related field, with 12+ years of industry experience in gene therapy, oligonucleotide therapeutics, or genetic medicine.
  • Proven track record of advancing at least one gene therapy asset from discovery through IND filing and into early-stage clinical development.
  • Deep expertise in at least two of the following: AAV or non-viral (LNP) delivery, CRISPR/epigenetic editing, CNS gene therapy, or neuromuscular disease biology.
  • Strong understanding of CMC challenges specific to gene therapies (e.g., vector production, potency assays, stability).
  • Experience authoring and contributing to regulatory submissions (IND, CTA).
  • Exceptional communication skills, able to translate complex science for boards, investors, and non-scientific stakeholders.
• Preferred
  • Existing relationships with key opinion leaders, FDA reviewers, or CRO partners in the gene therapy space.
  • Experience with both rare disease and larger indication development strategies.
  • Prior Series B/C-stage company experience.