Partners wanted for base editing platform

CAMBRIDGE, U.K.—In an effort to add new technologies to its suite of research tools and offerings, Horizon Discovery Group is seeking partners to explore the functionality of a new Pin-point base gene-editing platform. Building upon an exclusive collaboration with Rutgers, The State University of New Jersey, Horizon is seeking three to five new partners to assess the platform in its current state and to help shape its future iterations.

 

Horizon specializes in gene editing and gene modulation technologies, offering a broad portfolio of tools to assist in understanding gene function, identifying genetic drivers behind human disease and gene therapies for precision medicine, and developing and validating diagnostic workflows. The new Pin-point platform would build upon their extensive library of tools and introduce a new methodology that allows for gene editing without having to “cut” the gene, as with CRISPR techniques.

 

The basis of the Pin-point system is a novel base-editing technology developed by Dr. Shengkan Jin, associate professor of pharmacology at Rutgers University, and co-inventor Dr. Juan C. Collantes, postdoctoral research fellow at Rutgers’ Robert Wood Johnson Medical School. That technology has been licensed to Horizon to explore its uses in therapeutic, diagnostic and service applications.

 

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

 

What makes base-editing technology unique is its reliance on manipulating a patient’s cells either inside the body (gene therapy) or externally and then transplanting back into the body (cell therapy). The ability to engineer DNA in cells without cutting the gene like in CRISPR methodologies can allow scientists to correct specific mutations or errors, or, in some cases, deactivate certain pathological genes. Thus, the Pin-point system will enable accurate gene editing without creating some of the genomic changes that in some instances create adverse effects in people.

 

Horizon indicates that it has earmarked considerable resources and investment to fully develop this technology, both internally and at Rutgers. In order to accelerate the clinical progression of the utility of the technology, they are currently seeking partners that will be granted access to the technology. According to Horizon’s press release, this novel system 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.

 

As stated on company’s website, “The Pin-point system utilizes a nuclease-deficient CRISPR protein that recruits a non-nuclease DNA modifying enzyme, such as a cytidine deaminase, to the disease-causing gene via an RNA-based recruitment mechanism, where the deamination achieves the desired genotypic effect while minimizing the generation of the potentially oncogenic DNA breaks. This allows accurate editing of genes with an improved safety profile due to minimized unintentional genomic changes.”

 

Jin stated, “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 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 hypercholesterolemia.”

 

“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,” according to Dr. S. David Kimball, senior vice president for research and economic development at Rutgers University. “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.”