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Technology Breakthroughs
  • The creation of highly defective HSV vectors that are completely devoid of toxicity in any cell type.
  • The development of rapid vector engineering strategies for rapid vector production.
  • The creation of highly engineered cell lines for high titer vector propagation and vector manufacture.
  • The design of transgene expression cassettes for long term expression in specific cell types, whose expression is controlled by cellular or inducible promoter.
  • Engineered vector envelopes for retargeting to specific cellular structures.

Advantages of HSV over other vector platforms
  1. Large size permits accommodation of large payloads -- up to 40Kb.
  2. Efficient infectivity allowing single particle transduction of cells avoiding immune system surveillance.
  3. Vector retargeting to allow selective infection and gene expression in neuronal subtypes.
  4. Rapid and efficient axonal transport for delivery to distal sites in sensory neurons and the in the brain.
  5. Long-term persistence in neurons without toxicity, integration of vector DNA into cell chromosomes, or disturbance of normal cellular functions.
  6. Proven safety in patient trails.
  7. High vector particle yield manufacturing methods available and proven.