Key responsibilities:

• Oversight of design, construction, optimization, purification, delivery, and characterization of viral vectors for therapeutic targets
• Collaboration with external resources and direction of internal resources (direct and matrix) necessary to produce virions and conduct in vivo studies
• Understanding the disease deficiency and assessing normal target expression as a benchmark for viral expression disease-relevant tissues
• Design, oversight, and interpretation of in vivo evaluation of gene delivery efficacy
• Interfacing with clinical, regulatory, and CMC groups to ensure alignment on gene therapy development
• Developing innovations for the design, production, purification, and delivery of gene therapies

• PhD with 10+ years’ experience in gene therapy
• Proven track record using a range of AAV capsids to target specific tissues, design of transgene for desired cell and tissue specificity, viral production, viral purification, and administration of AAV and lentiviral gene delivery systems in cell and animal models
• Adept at supporting and prioritizing multiple therapeutic targets simultaneously
• Hands-on experience with a variety of AAV capsids targeting the CNS, liver, hematopoietic, and other specific tissues