Expanding possibilities with gene insertions
Curing genetic and age-associated
diseases
HarborSite’s targeted gene insertion platform enables precise and efficient integration of therapeutic genes into safe genomic sites, creating the potential for one-time, durable treatments across thousands of genetic diseases and age-related disorders with a single intervention.
Our APPROACH IS UNIQUE
We engineer recombinase enzymes to catalyze targeted, specific and efficient therapeutic gene insertions
HarborSite pioneers engineering of site specific recombinases for precise insertion of genes of interest into safe and robustly expressing genomic safe harbors
Recombinases are ideal for large gene insertion: they are compact enzymes that operate on large DNA sequences efficiently and specifically in any cell type, in vitro and in vivo.
Using structural modeling, large-scale multiplexed screens and state-of-the-art computational protein design, we have learned to program recombinase specificities to direct insertions into desired genomic sites.
We have demonstrated programmable recombinase-mediated full-length gene insertion in human cells, enabling the same recombinase to insert any therapeutic gene into a validated safe genomic site.
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