Open positions

Back to Careers

Head of Gene Therapy Research

Location: South San Francisco, CA

Company Description:

Ambys Medicines is an early-stage biotech company researching and developing medicines that harness the natural regenerative properties of the liver for patients suffering from devastating liver diseases.  Founded by Third Rock Ventures with world-renowned academic thought leaders and a substantial collaboration with Takeda, Ambys is pioneering novel approaches to liver restoration and regeneration.  The company’s core technologies of cell therapy, gene therapy, drug therapy, and unique translational models are the foundation of our research and development platform.  We at Ambys believe that revolutionary medicines result from bold research, performed to the highest standards, following the biology, putting patient needs first and performed by a collaborative, committed, and curious team.  We are seeking smart, creative, and motivated people to join our team and become part of the Ambys adventure.

Position Summary:

We are seeking an innovative scientific thought leader with expertise in gene therapy and viral and protein engineering to lead a team focused on the discovery and advancement of novel therapies for severe liver diseases. The Head of Gene Therapy will assume a key leadership role on a multidisciplinary team.  This critical addition to our R&D leadership team will develop strategies for treating liver diseases using hepatocyte cell therapy and gene therapy. Ideally, the successful candidate will possess deep expertise in multiple gene therapy modalities, including, but not limited to AAV, LV, CRISPR/Genome Editing, and ASOs. The Head of Gene Therapy will:

  • Partner with the CSO to develop a vision and strategy to build a core gene therapy platform focused on developing novel therapies to treat severe liver disease and ultimately lead a team to execute against that strategy.
  • Provide scientific leadership in the gene therapy function to support target discovery, cellular engineering, transgene and vector engineering, transduction evaluation, and model development.
  • Lead early gene therapy production efforts (process development/R&D vector production) as well as early gene therapy manufacturing strategy and execution.
  • Partner with cross functional groups internally as well academic collaborators, CROs and alliance partners to build and deliver a portfolio of gene therapy candidates.
  • Organize, communicate and present complex data sets to executive management and key stakeholders.
  • Develop talent by recruiting research team members and providing professional and personal growth opportunities and mentorship to that team.
  • Develop and manage a budget, capital equipment and resources internally and externally across projects.

Job Requirements:

  • Ph.D. degree a biological science discipline including Virology, Bioengineering, Molecular Biology, Biochemistry or a related field
  • 12-15+ years of industry experience in a drug discovery/therapeutic discovery organization
  • Experience developing and managing a product pipeline of different targets and therapies.
  • Experience leading and developing a diverse group of scientists in a drug/therapeutic discovery setting
  • Prior experience with AAV and/or gene therapy in liver disease is strongly desired, but not required.
  • Ability to work cross functionally with CROs, academic labs and cross functional groups is essential to this role.
  • Prior experience with gene therapy process development and manufacturing strongly preferred.
  • Excellent communication, presentation, collaboration, time-management, and organizational skills are required.


If you would like to be considered for an opportunity at Ambys Medicines, please submit your CV with the title of the position in the subject line to

The well-being of our employees and their families is important to us. We offer comprehensive and competitive health, financial, paid time off and many other benefits designed to promote the health and financial well-being of our employees and their families.

Note to Recruitment Agencies: Please do not forward any agency resumes. Ambys Medicines is not responsible for any fees related to resumes that are unsolicited.

Ambys Medicines is an equal opportunity employer.