Rare Daily Staff

Horizon Discovery Group said it has entered into an exclusive strategic partnership with Rutgers University to develop and commercialize a novel gene editing technology, known as base editing.

Base editing is a novel technology platform for engineering DNA or genes in cells that has the potential to correct errors or mutations in the DNA by modifying genes using an enzyme. Compared with currently available gene editing methodologies, such as CRISPR/Cas9 that creates cuts in the gene that can lead to adverse or negative effects, this new technology allows for more accurate gene editing while reducing unintended genomic changes.

Horizon expects the technology will have a significant impact in enabling cell therapies to be progressed through clinical development and towards commercialization.

The technology potentially has applications in the development of new cell therapies and will augment Horizon’s research tools and services.

“Base editing is potentially transformative for all gene editing technologies with the potential to help target many diseases that to date have no treatment,” said Terry Pizzie, Horizon Discovery Group’s CEO.

Horizon will collaborate with Rutgers University to further develop the novel base editing platform from the laboratory of Shengkan Jin, associate professor of pharmacology at Rutgers Robert Wood Johnson Medical School. As part of the agreement, Horizon has made a non-material payment to Rutgers for an option to exclusively license the base editing technology for use in all therapeutic applications.

As part of the collaboration, Horizon will also fund further research in base editing at Rutgers University while undertaking evaluation and proof of concept studies at Horizon.

“The potential is enormous,” said Jin of Rutgers. “In addition to the ‘simple’ diseases caused by a single genetic alteration event, the therapeutic strategy, in principle, could also be useful for treating diseases where permanently targeting a disease-related gene is beneficial.”

 

January 29, 2019
Photo: Terry Pizzie, Horizon Discovery’s CEO

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