Horizon exclusively licenses new base editing technology

Rutgers technology has been incorporated into Horizon Discovery Group’s next-generation gene editing platform

Horizon Discovery Group, gene editing and gene modulation technologies specialist, has announced a new addition to its portfolio licensed from Rutgers, The State University of New Jersey. The novel base editing technology has been licensed for exclusive use in therapeutic, diagnostic and services applications.

Rutgers’ technology has now been incorporated into Horizon’s next-generation gene editing platform to enable the development of novel therapeutics that rely on engineering patient’s cells either directly in the body (gene therapy), or externally before transplanting back into the patient (cell therapy).

This platform will expand the Horizon’s research tools and service provisions, allowing accurate gene editing while reducing unintended genomic changes that could lead to deleterious effects in patients.

In order to enable the use of the tech, Horizon formed an exclusive partnership with Rutgers in January 2019 to further develop the novel base editing technology invented by Dr Shengkan Jin, associate professor of pharmacology, and co-inventor Dr JuanCollantes, post-doctoral research fellow at Rutgers Robert Wood Johnson Medical School.

Since establishing the partnership, Horizon has been funding research in base editing at the University while undertaking its own evaluation and proof-of-concept studies. Horizon has a number of internal programmes designed to accelerate the clinical uptake of this technology and is now seeking 3–5 partners to assess and shape the development of its Pin-point base editing platform.

Horizon will offer partners access to a novel system that could be used to progress more effective multi-gene knockout cell therapy programmes through clinical development with an improved safety profile. Partners will also gain access to the Horizon’s expertise in genome engineering of different cell types, access to early technical data, and influence over the direction of future development.

Dr Jonathan Frampton, Corporate Development Partner at Horizon, said: “The technology could have a significant impact in enabling cell therapies to be progressed through clinical trials and towards commercialisation.”

Dr Shengkan 'Victor' Jin of Rutgers University explained: “The cytidine deaminase version of the technology alone could potentially be used for developing cell therapies such as gene modified cells for sickle cell anaemia and beta-thalassemia, HIV resistant cells for AIDS, over-the-shelf CAR-T cells for cancer, and MHC-compatible allogenic stem cells for transplantation.”

He added that other applications could include use as gene therapies for inherited genetic diseases including antitrypsin deficiency and Duchenne muscular dystrophy.

“In addition, we intend to take full advantage of the unique modularity and versatility features of Pin-point platform and develop efficient gene inactivation agents for potential treatment of many devastating diseases where the leading causal contributing factors are well defined,” Jin explained. “At the top of this disease list are Alzheimer’s disease, amyotrophic lateral sclerosis, and familial hypercholesterinemia.”

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