Horizon Discovery Grants Access to Base Editing Technology

January 16, 2020
Pharmaceutical Technology Editors

The next-generation gene editing system can be applied to the development of novel cell and gene therapies.

Horizon Discovery Group, a Cambridge, United Kingdom-based provider of gene editing and gene modulation technologies, announced on Jan. 14, 2020 that it will provide access to a novel base editing technology licensed from Rutgers University, The State University of New Jersey, for exclusive use in therapeutic, diagnostic, and services applications. The technology, which expands Horizon’s research and service tools, is incorporated into the company’s next-generation gene editing platform to enable the development of novel gene and cell therapies.

Horizon will offer partners access to the technology, which 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.

Horizon formed an exclusive partnership with Rutgers in January 2019 to further develop the 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. Since then, Horizon has been funding research in base editing at Rutgers while undertaking its own evaluation and proof-of-concept studies. The company has a number of internal programs designed to accelerate the clinical uptake of this technology and is now seeking three to five partners to assess and shape the development of its Pin-point base editing platform.

Base editing is a novel technology for engineering DNA in cells with the potential to correct certain errors or mutations in the DNA or inactivate disease-causing genes. Compared with currently available gene-editing methodologies, such as conventional CRISPR/Cas9 that creates “cuts” in the gene, which can lead to adverse or negative effects, this new base editing technology allows for accurate gene editing while reducing unintended genomic changes that could lead to deleterious effects in patients, according to Horizon.

“The technology could have a significant impact in enabling cell therapies to be progressed through clinical trials and towards commercialization. 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 organizations will help us to drive innovation and deliver the best therapy for the patient,” said Dr. Jonathan Frampton, corporate development partner, Horizon Discovery, in a company press release.

“The cytidine deaminase version of the technology alone could potentially be used for developing cell therapies, such as gene-modified cells for sickle cell anemia and beta thalassemia, HIV resistant cells for AIDS, over-the-shelf CAR-T [chimeric antigen receptor T] cells for cancer, and MHC [major histocompatibility complex]-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,” said Jin in the press release.

“The gene editing technology developed by Rutgers has the potential to revolutionize how scientists think about their search for better options and outcomes in the treatment of disease. It has the potential to solve some of the most persistent global health challenges. This partnership with Horizon Discovery is paving the way to deliver biotherapies for precision medicine and diagnostics and improve human health. I am proud that Rutgers, together with Horizon, is among the frontrunners in the field of gene editing,” Dr. S. David Kimball, senior vice-president for Research and Economic Development at Rutgers University, added in the press release.

Source: Horizon Discovery