Optimization of the Gene Expression

We have engineered organotropic capsids, based on deep learning identification of target tissue recognition peptides, and have modified our transgenes to contain distinctive sequence features that drive tissue-appropriate levels of gene expression.

These features are enclosed into a recombinant AAV particle and include

  • muscle-specific promoters, modified intron, codon-optimized coding sequence
  • microRNA target sites (miRTs) that respond to off-target-tissue-specific microRNAs, ensuring transgene suppression where it is not desired

Design and Selection of Next Generation Muscle
Gene Therapies

We use two platforms to generate new viral vector capsids:

  • Rational design, introducing sequences that enhance the tropism in desired organs (muscle, heart, diaphragm) and detarget the liver and other tissues not impacted by the disease.
  • Deep learning, based on model training from generated data, for high-throughput selection of capsids and development of specific promoters.