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28 January, 2020 - 12:15 By Kate Sweeney

Horizon and New Jersey university power up gene therapy technology

Horizon Discovery is to provide access to a novel base editing technology licensed from Rutgers, The State University of New Jersey, for exclusive use in therapeutic, diagnostic and services applications. 

The technology is incorporated into Horizon’s next-generation gene editing platform. It will 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). 

The platform will also expand the company’s research tools and service provisions.

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 Juan C. Collantes, post-doctoral research fellow at Rutgers Robert Wood Johnson Medical School, and has since 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 programs 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 programs through clinical development with an improved safety profile. 

Partners will also gain access to the company’s expertise in genome engineering of different cell types, access to early technical data, and influence over the direction of future development.

Base editing is a novel technology for engineering DNA in cells, which the potential to correct certain errors or mutations in the DNA, or inactivate disease-causing genes. 

Compared to currently available gene editing methodologies such as conventional CRISPR/Cas9, which creates ‘cuts’ in the gene that can lead to adverse or negative effects, this new technology allows for accurate gene editing while reducing unintended genomic changes that could lead to deleterious effects in patients.

Dr Jonathan Frampton, corporate development partner at Horizon Discovery, said: “The technology could have a significant impact in enabling cell therapies to be progressed through clinical trials and towards commercialisation.

“Horizon is pleased to offer an effective and precise base editing technology and, alongside Rutgers, aims to make base editing available to all appropriate cell and gene therapy companies as well as research departments. Partnering with leading organisations will help us to drive innovation and deliver the best therapy for the patient.”

Dr Shengkan ‘Victor’ Jin of Rutgers University added: “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. 

“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. 

“At the top of this disease list are Alzheimer’s disease, amyotrophic lateral sclerosis, and familial hypercholesterinemia.”

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