Scientist I/II, Novel Capsid Discovery - AAV Transgene Engineering

Voyager Therapeutics

Voyager Therapeutics

Cambridge, MA, USA
Posted on Friday, May 26, 2023

About Voyager

Voyager Therapeutics (Nasdaq: VYGR) is a biotechnology company dedicated to breaking through barriers in gene therapy and neurology. The potential of both disciplines has been constrained by delivery challenges; Voyager is leveraging innovative expertise in capsid discovery and deep neuropharmacology capabilities to address these constraints. Voyager’s TRACER AAV capsid discovery platform has generated novel capsids with high target delivery and blood-brain barrier penetration at low doses, potentially addressing the narrow therapeutic window associated with conventional gene therapy delivery vectors. This platform is fueling alliances with Pfizer Inc., Novartis and Neurocrine Biosciences as well as multiple programs in Voyager’s own pipeline. Voyager’s pipeline includes preclinical programs in Parkinson’s disease, Alzheimer’s disease, and amyotrophic lateral sclerosis (ALS), each with validated targets and biomarkers to enable a path to rapid potential proof-of-biology. For more information, visit www.voyagertherapeutics.com

Job overview: We are looking for a talented and motivated Scientist I/Scientist II to join the Gene Therapy team to support our RNA interference knock-down programs, specifically in the context of trinucleotide repeat expansion disorders. This will involve optimization, AAV vectorization and screening of candidate RNA interference for our Huntington’s disease program and other upcoming targets. The position will emphasize the engineering of complex second-generation multicistronic/multitarget transgene conformations and their impact on trinucleotide repeats biology.

Responsibilities

  • Collaborate with computational scientists to identify novel candidate siRNA for efficient and specific knock-down of therapeutic targets
  • Design and build AAV-compatible constructs with full optimization of regulatory sequences, scaffolds and artificial microRNA
  • Analyze the efficiency, specificity, toxicity and processing of miRNA constructs in cell culture and animal models
  • Analyze the impact of various constructs on trinucleotide repeat biology and expansion

Required qualifications

  • PhD in biology, biochemistry, biomedical engineering, or a related field
  • 5+ years hands-on wet lab experience
  • Proficiency in cloning design and techniques
  • Expertise in cell culture and aseptic techniques
  • Experience in tissue handling and DNA/RNA purification
  • Experience in quantitative assays (RT-PCR, ddPCR, reporter gene assays)
  • Strong collaborative, organizational, and presentation skills
  • Ability to document, maintain, and share an organized account of lab work and results

Preferred qualifications

  • Experience with nucleotide repeat biology
  • Experience with animal tissues
  • Experience with AAV vectors and library generation/screening