Sr/ Director of Biology, Rare Genetic Disorders

Posted on April 29,

Orna Therapeutics, Inc. is a Cambridge-based biotechnology startup on a mission to create therapies for diseases inaccessible to existing approaches. Built on novel circular RNA technology developed at MIT, Orna is leading the next wave of RNA therapeutics with our best-in-class expression and delivery platform. We are seeking an exceptional Director of Biology, Rare Genetic Disorders, to join our innovative team and accelerate novel drug candidates to approval. This role will shape and lead the RGD drug discovery path from early target evaluation to development candidate nomination. The Director will design and manage the workflow to characterize circular RNA candidates and evaluate lipid formulations for LNP delivery in terms of both activity and mechanism of action. This role will lead existing programs on a critical path, propose and oversee new programs, and empower new platform biology development.  The Director will conduct and collaborate with R&D functions including platform delivery, molecular biology, preclinical pharmacology, safety, and CMC to build a robust pipeline across several disease areas with a focus on muscle and liver regeneration. The ability to seamlessly lead multiple, different workstreams with a focus on teamwork and ownership will be essential for success.

The successful candidate will have a strong track record of progressing programs from early-stage discovery to preclinical studies in a fast-paced, matrix environment. Ideal candidates will have experience advancing RNA, lipid nanoparticle, cell and/or gene therapies to IND.

The Director of Biology will report to the Chief Scientific Officer (CSO) and work cross-functionally across R&D teams, business development, legal and corporate strategy.


  • Lead the design, implementation, and analysis of preclinical data including cellular functional assays and in vivo studies as a strong partner to preclinical safety and pharmacology teams
  • Refine therapeutic workstreams and candidate evaluation strategies across integrated teams from early-stage discovery to IND
  • Provide key insights and oversight of early target prioritization and disease/indication expansion
  • Establish roadmap for building capabilities to achieve balanced infrastructure leveraging both internal expertise and external resources
  • Promote strong scientific excellence and foster an open culture of ideation, innovation, and deep scientific rigor
  • Deliver and present key data packages and data-driven scientific updates to the organization that impact pipeline milestones and corporate objectives
  • Lead the expansion of the RGD team, recruiting key talent and managing, developing, and mentoring team members as we continue our growth as an agile organization


  • Ph.D. with 10+ years of experience in biology, 7+ years in biotech/ pharmaceutical industry, and 5+ years management experience
  • Domain expertise in rare genetic diseases including muscle and liver and an established track record of advancing programs to IND
  • Track record of effectively managing contract research organizations from both the people management and complex project enablement perspective
  • Excellent oral, written, and strategic thinking skills with demonstrated ability to successfully lead the ideation and development of a plan and move to action
  • Ability to navigate, problem-solve, execute, and lead in a dynamic, fast-paced and evolving environment
  • Willingness to provide leadership and role modeling for members of the team and other parts of the organization

Orna Therapeutics, Inc. is an equal opportunity employer.