Epigenetic editing expands the reach of gene therapy

 


Gene editing therapies are designed to be a one-and-done fix that cure a genetic disease and last a lifetime. Yet, they have some safety risks — chief among them the potential for off-target effects that permanently alter unrelated genes. Plus, there are ethical questions related to genetic editing of germline cells, as those mutations could be passed down to future generations (1).

“Because those risks exist, why not develop alternative approaches that would stand alongside or improve on what classical gene editing can do?” said Daniel Hart, the Head of Platform at Epicrispr Biotechnologies.

Rather than cutting and permanently altering the genetic code, Epicrispr Biotechnologies is one of several companies pioneering a new kind of gene therapy — epigenetic editing. This technology is inspired by a cell’s natural epigenetic mechanisms, which work by modifying chemical tags on DNA to regulate gene expression over time.

“The safety aspect is key. If one were offered the option of a cure that would leave everything as it were, except to change the gene expression in a way that would benefit you. Or, run the risk that I introduce mutations that might affect other genes and other pathways, I think the decision would be an easy one,” said Hart.

In March, Epicrispr Biotechnologies announced plans to start a clinical trial in New Zealand later this year to test their lead compound, EPI-321, in people with the genetic neuromuscular disease facioscapulohumeral muscular dystrophy (FSHD). Using their Gene Expression Modulation System (GEMS), they are also advancing programs to treat other neuromuscular diseases and blood cancers.

“I would like to see [epigenetic editing] stand alongside the other approaches, which I think have gotten their due credit and attention,” said Hart. “I think it will get there.”

GEMS leverages the CRISPR-associated (Cas) proteins, which are essentially DNA cutters. Instead of using the Cas proteins to cut the DNA, we've disabled that cutting aspect, so the Cas protein now is just very good at finding whatever we want it to bind in the genome. It brings small modulators to turn genes on or off as we desire. We also add a guide RNA that determines where the Cas protein binds. It’s basically the GPS of the system. We discovered and engineered the modulator domains at Epicrispr Biotechnologies together with the Cas proteins. Specifically, we have the exclusive license to use the CasMINI protein.

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