Job Details

Location:Cambridge, MA, US

Job description

• It is a clinical stage biotechnology company committed to bringing to market novel products for the treatment of rare and ultra-rare diseases with an initial focus on serious debilitating metabolic genetic diseases.
• Company has rapidly built a diverse portfolio of both large and small molecule candidates with the potential to address diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no approved therapies.
• We are looking for exceptional individuals who share our passion for developing novel therapies to treat rare diseases and advancing the field of gene therapy.

Position Summary

• Reporting to the SVP Pharmaceutical Development, the Principal Scientist will lead the Vector Engineering team to implement cutting-edge AAV technology to improve efficacy, potency, safety and production of rAAV vectors.
  

Responsibilities

• Lead the Vector Engineering team to execute on the strategic vision of improved second generation AAV vectors to support early and late stage programsOversee the design, conduct and interpretation of vector studies to enable the company's research and development goals and present finding to internal and external stakeholdersManage the team to ensure scientific rigor, and team continuity and excellence through hiring, training and performance management.
• Establish and manage scientific collaborations with external academic groups and CROs to advance our program pipelineWork closely within cross-functional teams including cell line isolation, process development and analytical development teams to implement improved rAAV vector design to enable increased yield and quality metrics for discovery and on-going programs