Technology is expected to slash manufacturing costs and result in hundreds more X-MAN cell lines.

Horizon  Discovery has negotiated exclusive, worldwide rights to University of Minnesota IP that it claims boosts the rates of recombinant adeno-associated virus (rAAV)-mediated gene editing in human cells. The firm suggests the technology will allow at least a 10-fold increase in the gene-editing frequency of its rAAV Genesis™ and enable it to expand its panel of genetically defined, patient-relevant X-Man™ disease models.

Horizon will pay the university up-front and milestone fees along with royalities on sales of future relevant products. In addition, the firm will provide $400,000 in funding to support continued research over the next two years at the laboratory of the new technology’s inventor, Eric A. Hendrickson, Ph.D., at the University of Minnesota.  

The rAAV technology that underpins Horizon’s Genesis platform is capable of performing gene-editing functions in human cells by exploiting homologous recombination (HR). Horizon says this rAAV platform thus provides it with a method for precisely altering any DNA sequence for applications such as generating accurate cell-based models of genetic diseases, as embodied by its X-MAN cell lines, or correcting genetic defects in gene therapy applications.

Dr. Hendrickson’s laboratory has discovered that shutting off an error-prone DNA repair mechanism known as nonhomologous end joining (NHEJ), which competes with HR, effectively increases the rAAV gene-editing frequency in human cells by 10–30%. The resulting technology, which Horizon licensed, has been developed as a transient, chemical-mediated method to improve the gene-editing efficiency of its rAAV Genesis technology.

“The promise of the in-licensed inventions to give us an order of magnitude improvement to our rAAV platform is huge and will allow us to reduce manufacturing costs by 75% and development timelines by half for generating complex human models of diseases such as cancer,” remarks Darrin M Disley, Ph.D., Horizon’s executive chairman. “Professor Hendrickson’s curiosity as to how Horizon’s rAAV gene-editing technology works, when scientific logic suggests it should not, is a real example of how academic research can lead to both significant enhancement in general scientific knowledge as well as the improvement of an industrial manufacturing process like ours.

“Increasing the efficiency of rAAV Genesis using Professor Hendrickson’s discovery will enable us to rapidly model the increasing array of genetic variations that are now known in human populations and linked to diseases such as cancer,” adds Chris Torrance, Ph.D., Horizo’s CEO. “Horizon and its rapidly growing network of academic rAAV collaborators will use this advance to increase our bank of 300+ disease models to a resource comprising thousands of genetically defined human cell lines.”

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