Principal Scientist of Gene Therapy, Genomic Medicines at AstraZeneca in Cambridge, Massachusetts

Job Description:

This is what you will do:



As a Principal Scientist in the Genomic Medicine Department at Alexion - AstraZeneca Rare Disease, you will play a pivotal role in advancing genomic medicines that target genetic cardiomyopathies. The successful candidate will be responsible for identifying novel therapeutic targets and driving the development of innovative therapies from preclinical research through IND. Your leadership will be pivotal to coordinating cross-functional projects, collaborating with internal stakeholders, externally partners, and key opinion leaders. This role requires a dynamic and highly collaborative scientist with a proven track record of successfully ideating programs in gene therapy, cardiovascular research, and preclinical development and lead them towards regulatory submission.



You will be responsible for:

• Lead and driving gene therapy programs for the treatment of genetic cardiomyopathies, from discovery through preclinical development.
  
  


• Identifying novel therapeutic targets and optimizing therapeutic strategies.
  
  


• Design, implement, and oversee in vivo and in vitro studies to evaluate efficacy, biodistribution, and safety of therapeutic candidates.
  
  


• Collaborate with cross-functional teams including pharmacology, CMC, and translational science to develop preclinical strategies.
  
  


• Manage external collaborations with academic institutions, contract research organizations (CROs), and key opinion leaders (KOLs).
  
  


• Provide scientific leadership and mentorship to junior scientists and team members.
  
  


• Communicate project progress and findings to internal team, leadership and external collaborators.
  
  


• Support regulatory submissions, publications and presentations at scientific conferences.
  
  

You will need to have:

• MS or PhD in Molecular Biology, Genetics, Cardiovascular Biology, or related field.
  
  


• Minimum of 8 years (MS) 5 years (PhD) of experience in gene therapy research and development, with a focus on AAV, LNP, or gene editing technologies.
  
  


• Expertise in cardiovascular biology or rare genetic diseases, particularly cardiomyopathies.
  
  


• Excellent communication and interpersonal skills, with the ability to work collaboratively across disciplines.
  
  


• A strong publication record in peer-reviewed journals and presentations at scientific meetings.
  
  

We would prefer for you to have:

• Experience with AAV vector design, optimization, and delivery strategies specific to cardiac tissue.
  
  


• Knowledge of PK/PD modeling and bioinformatics approaches in gene therapy development.
  
  


• Prior experience in a pharmaceutical or biotech setting focused on rare diseases or gene therapy development.
  
  


• Familiarity with regulatory requirements and experience with the IND-enabling process.
  
  

AstraZeneca embraces diversity and equality of opportunity. We are committed to building an inclusive and diverse team representing all backgrounds, with as wide a range of perspectives as possible, and harnessing industry-leading skills. We believe that the more inclusive we are, the better our work will be. We welcome and consider applications to join our team from all qualified candidates, regardless of their characteristics. We comply with all applicable laws and regulations on non-discrimination in employment (and recruitment), as well as work authorization and employment eligibility verification requirements. Apply Now