Translational Genomics

Gene Editing

Gene editing has become vital in the effort to drive personalized and genomic medicine, and the generation of precisely engineered human cell lines (e.g. somatic iPSCE, ES) and animal models models (e.g. mice, rats, rabbits) is helping to lead the way. By editing the code of the genome in functional cells or animals, the effects of genetic variation found in real patients can be reproduced in a laboratory setting, letting researchers ask important and contextual biological questions much earlier in the drug discovery process.

Within the gene editing research field, Horizon has an exclusive worldwide right to use rAAV gene editing for non-therapeutic applications. In addition it has multiple commercial licenses to important patent portfolios related to CRISPR/Cas9 technology for a variety of applications. This includes non-exclusive licenses from The Broad Institute, Caribou BioSciences, ERS Genomics, Harvard University and as well as an exclusive license for engineering the rat species from Caribou BioSciences. Horizon also has a worldwide exclusive license from Sigma-Aldrich for use of ZFN technology in engineering mice, rats, and rabbits, and a non-exclusive license to use ZFN technology for gene editing services and derivation of cell line products (excluding bio-production).

These gene editing capabilities, fully available to Avvinity via the service relationship in place, enable the rapid generation of target overexpressing and non-expressing cell lines to assist SELEX, a discovery tool for genomic aptamers.

Additionally, given the near ubiquity of the GGTA1 gene and alpha-gal epitopes on the surfaces of non-human cells, the development of preclinical animal models can be a significant challenge. Gene editing however can be used to create knockout animal models and murine cell lines for testing advanced compounds, thereby avoiding this problem, and helping to drive preclinical programs.

Learn more about Horizon's technology:

Discovery Research

Horizon is positioned to give Avvinity the best data to support Alphamer drug development programs including:

  • Custom in vitro and in vivo model generation
  • Target identification and validation
  • In vitro and in vivo efficacy studies
  • Identification of responsive or resistant patient populations
  • Mechanism of action and resistance mechanism studies
  • Drug combination screening