Life Sciences

CRISPR/Cas9: patented gene editing technology

ERS Genomics is a biotechnology company co-founded by Nobel Prize Laureate Dr Emmanuelle Charpentier to provide broad access to the fundamental CRISPR/Cas9 patent estate, a gene-editing technology with a broad range of applications across life sciences.

CRISPR refers to a group of DNA sequences that occur in prokaryotic organisms such as bacteria and archaea and that are derived from DNA fragments of bacteriophages that previously infected the organism in question. These CRISPR DNA sequences are used to detect and destroy DNA from similar bacteriophages during subsequent infections, enabling the organism to counter such infections.

Gene-editing patent families

CVC, which stands for University of California, University of Vienna, and Emmanuelle Charpentier, is the acronym used to describe the owners of what are commonly referred to as the UC Berkeley CRISPR patents. In Europe, the CVC inventors have been awarded three broad patents on the CRISPR/Cas9 technology covering a composition comprising a chimeric Cas9 protein and a DNA-targeting RNA which includes a DNA-targeting segment and a protein-binding segment comprising tracrRNA and CRISPR RNA (crRNA); a method of modifying a target DNA in a single-cell eukaryotic organism, an animal cell, or a plant cell comprising contacting the target DNA with a CRISPR/Cas9 complex; and a method of modifying a target DNA comprising contacting the target DNA with a CRISPR/Cas9 complex where the crRNA and tracrRNA are covalently linked by intervening nucleotides (single guide RNA).

 

In the US, the CVC portfolio currently contains patents that include claims to compositions and methods of using Cas9 with a guide RNA to form a CRISPR/Cas9 complex that binds a specified DNA sequence; various formats for delivering these complexes in cells of all types; the use of the CRISPR/Cas9 complex to cleave DNA, resulting in knock-outs, insertions or mutations; the use of mutated Cas9 to ‘nick’ DNA in place of cleavage or to bring an effector domain to a specified DNA sequence to regulate gene expression in a cell; and, finally, compositions of guide RNAs in a variety of formats including various lengths, chemical modifications, and base compositions.

Biotech applications

The CRISPR patent portfolio licensed by Dublin, Ireland based biotechnology company ERS Genomics (the CVC portfolio) is the most comprehensive collection of proprietary rights to the fundamental CRISPR/Cas9 gene editing platform. ERS grants non-exclusive licences to a growing list of issued and pending patent applications globally, in all fields excluding human therapeutics, and non-exclusive licences are available for research and sale of products and services across multiple fields including research tools, kits, and reagents; discovery of novel targets for therapeutic intervention; cell lines for discovery and screening of novel drug candidates; GMP production of health care products; companion animal and livestock health; the production of industrial materials such as enzymes, biofuels and chemicals; and synthetic biology.

According to Eric Rhodes, CEO of ERS Genomics, the versatility and programmability of Cas9 has enabled the CRISPR/Cas9 technology to become a revolutionary approach in biological research.

Licence agreements

In March this year, ERS Genomics signed a non-exclusive licence agreement granting Setsuro Tech, a Japanese biotechnology start-up using genome-editing techniques to develop and supply cell and animal models, access to its CRISPR/Cas9 patent portfolio. Setsuro Tech has developed a high-throughput genome editing method for mammalian embryos, termed ‘genome editing by electroporation of Cas9 protein’ (GEEP). Using this method, the company is able to generate genetically engineered mice at low cost and in a short timeframe. Setsuro Tech is applying the newly licensed technology to create genome-edited cell and animal models, based on end-user requirements.

Setsuro Tech Inc is a biotechnology startup established by the company’s Chief Technology Officer, Professor Tatsuya Takemoto and his team at the Institute of Advanced Medical Sciences, Tokushima University. Professor Takemoto and Dr Masakazu Hashimoto of Osaka University developed the GEEP method in 2015 and by using this technology, Setsuro Tech aims to become a provider of a wide range of genetically engineered animals, starting with the generation and sale of genetically engineered mice, foreseeing the application of genetically engineered animals in industry.

Also in March of this year, ERS Genomics signed a licence agreement with Otsuka Pharmaceutical Co. Ltd. (Otsuka) granting Otsuka access to the CRISPR/Cas9 genome editing technology for its internal research and development initiatives to address areas of unmet medical need and in February ERS established a non-exclusive licence agreement granting South Korean company G+FLAS Life Sciences access to the patent portfolio. G+FLAS Life Sciences is a Korean biotechnology company developing research tools and reagents associated with CRISPR technology and supports research organizations by providing CRISPR-related biological research services, tools, and reagents. The company is also applying CRISPR PLUS technology to the development of drugs and production of CRISPR genome-edited plants in a non-GMO manner.

Also in February, ERS Genomics Limited and ZeClinics, a contract research organization using zebrafish to carry out services focused on analyzing the safety, efficacy and biomedical relevance of new compounds, formed a non-exclusive licence agreement granting ZeClinics access to the CRISPR/Cas9 patent portfolio. ZeClinics supports academic, pharmaceutical and biotech organizations in preclinical studies by providing services based on its zebrafish disease models. The company is applying CRISPR/Cas9 technology to create single knock-out, double knock-out and somatic F0 knock-out zebrafish variants.

Eric Rhodes, CEO of ERS Genomics: “The versatility and programmability of Cas9 has enabled the CRISPR/Cas9 technology to become a revolutionary approach in biological research.”

 

 

 

 

 

Further information

ERS Genomics Ltd

Dublin D02 YT22, Ireland

Ph: + 353-1-539-0082

E: info@ersgenomics.com

W: www.ersgenomics.com