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Toolgen Incorporated and Institute For Basic Science | Date: 2017-04-05

The present invention relates to a method for analyzing a genotype using a target-specific nuclease and, specifically, to a method for diagnosing cancer or analyzing a genotype by removing wild type DNA or particular genotype DNA using a target-specific nuclease or a variant thereof to amplify or concentrate only a small amount of DNA which has a difference in variation, such as a mutation, or genotype, and to a method for separating target DNA sesusing a target-specific nuclease or a variant thereof. Such methods are novel paradigm methods contrary to existing simple target-specific nucleases for post-PCR recognition of normal genotype and carcinogenic genotype, and can be favorably used in the early diagnosis of cancer or analysis of similar genotypes.


SEOUL, South Korea, May 15, 2017 (GLOBE NEWSWIRE) -- ToolGen, Inc. (KONEX, 199800), a biotechnology company specializing in genome editing, presented results from a study highlighting the use of CRISPR/Cas9 nucleases for specific and efficient genome editing in human primary T-cells, without causing severe cell damage and growth retardation in a poster presentation at the American Society for Gene and Cell Therapy (ASGCT) Annual Meeting being held in Washington, DC from May 10-13, 2017. The poster presentation entitled “Genome editing of human T cells to generate CAR-T cells with enhanced antitumor function” aimed to study the proposed idea that T-cell anergy serves to induce dysfunction in a wide variety of tumor microenvironments. Using CRISPR/Cas9-mediated gene editing, the researchers were able to successfully knock out DGKα, a tolerance-associated gene at an 80% efficiency rate, which allowed T-cells to reverse hypofunction induced by anergy. Based on this result, the researchers combined the lentiviral delivery of CAR and the electroporation of RNP to generate a CRISPR gene-edited CAR-T cell. These edited CAR-T cells demonstrated lower sensitivity to immunosuppressive conditions and were hyper-responsive against tumor cells. “We are honored to be recognized by ASGCT for our work using CRISPR/Cas9-mediated gene editing,” commented Seokjoong Kim, Research Director at Toolgen. “The data presented in the poster presentation demonstrates preliminary success in utilizing CRISPR/Cas9 nucleases for specific and efficient gene editing in human primary T-cells. The findings suggest that CAR and TCR T cells with a disrupted anergy-related gene may be potent effector cells against various cancers. We believe that these results continue to validate our approach to gene editing and provide support for further research to move our pipeline of opportunities into the clinic.” The authors of the presentation were Yoon-Young Kim, In Young Jung, Ho Sung Yu1, Seokjoong Kim, R&D Center, Toolgen, Inc., Seoul, South Korea ToolGen, Inc. is a biotechnology company focused on the development and application of genome editing technologies. It creates, and holds intellectual property rights for essential tools and technologies for editing the genetic information in microbial, plant, animal, and human cells. ToolGen’s mission is to translate the potential of our innovative platform technology into transformative products for biomedicine and agriculture. For more information, please visit www.toolgen.com.


SEOUL, South Korea, May 15, 2017 (GLOBE NEWSWIRE) -- ToolGen, Inc. (KONEX, 199800), a biotechnology company specializing in genome editing, presented results from a study highlighting the use of CRISPR/Cas9 nucleases for specific and efficient genome editing in human primary T-cells, without causing severe cell damage and growth retardation in a poster presentation at the American Society for Gene and Cell Therapy (ASGCT) Annual Meeting being held in Washington, DC from May 10-13, 2017. The poster presentation entitled “Genome editing of human T cells to generate CAR-T cells with enhanced antitumor function” aimed to study the proposed idea that T-cell anergy serves to induce dysfunction in a wide variety of tumor microenvironments. Using CRISPR/Cas9-mediated gene editing, the researchers were able to successfully knock out DGKα, a tolerance-associated gene at an 80% efficiency rate, which allowed T-cells to reverse hypofunction induced by anergy. Based on this result, the researchers combined the lentiviral delivery of CAR and the electroporation of RNP to generate a CRISPR gene-edited CAR-T cell. These edited CAR-T cells demonstrated lower sensitivity to immunosuppressive conditions and were hyper-responsive against tumor cells. “We are honored to be recognized by ASGCT for our work using CRISPR/Cas9-mediated gene editing,” commented Seokjoong Kim, Research Director at Toolgen. “The data presented in the poster presentation demonstrates preliminary success in utilizing CRISPR/Cas9 nucleases for specific and efficient gene editing in human primary T-cells. The findings suggest that CAR and TCR T cells with a disrupted anergy-related gene may be potent effector cells against various cancers. We believe that these results continue to validate our approach to gene editing and provide support for further research to move our pipeline of opportunities into the clinic.” The authors of the presentation were Yoon-Young Kim, In Young Jung, Ho Sung Yu1, Seokjoong Kim, R&D Center, Toolgen, Inc., Seoul, South Korea ToolGen, Inc. is a biotechnology company focused on the development and application of genome editing technologies. It creates, and holds intellectual property rights for essential tools and technologies for editing the genetic information in microbial, plant, animal, and human cells. ToolGen’s mission is to translate the potential of our innovative platform technology into transformative products for biomedicine and agriculture. For more information, please visit www.toolgen.com.


SEOUL, South Korea, May 15, 2017 (GLOBE NEWSWIRE) -- ToolGen, Inc. (KONEX, 199800), a biotechnology company specializing in genome editing, presented results from a study highlighting the use of CRISPR/Cas9 nucleases for specific and efficient genome editing in human primary T-cells, without causing severe cell damage and growth retardation in a poster presentation at the American Society for Gene and Cell Therapy (ASGCT) Annual Meeting being held in Washington, DC from May 10-13, 2017. The poster presentation entitled “Genome editing of human T cells to generate CAR-T cells with enhanced antitumor function” aimed to study the proposed idea that T-cell anergy serves to induce dysfunction in a wide variety of tumor microenvironments. Using CRISPR/Cas9-mediated gene editing, the researchers were able to successfully knock out DGKα, a tolerance-associated gene at an 80% efficiency rate, which allowed T-cells to reverse hypofunction induced by anergy. Based on this result, the researchers combined the lentiviral delivery of CAR and the electroporation of RNP to generate a CRISPR gene-edited CAR-T cell. These edited CAR-T cells demonstrated lower sensitivity to immunosuppressive conditions and were hyper-responsive against tumor cells. “We are honored to be recognized by ASGCT for our work using CRISPR/Cas9-mediated gene editing,” commented Seokjoong Kim, Research Director at Toolgen. “The data presented in the poster presentation demonstrates preliminary success in utilizing CRISPR/Cas9 nucleases for specific and efficient gene editing in human primary T-cells. The findings suggest that CAR and TCR T cells with a disrupted anergy-related gene may be potent effector cells against various cancers. We believe that these results continue to validate our approach to gene editing and provide support for further research to move our pipeline of opportunities into the clinic.” The authors of the presentation were Yoon-Young Kim, In Young Jung, Ho Sung Yu1, Seokjoong Kim, R&D Center, Toolgen, Inc., Seoul, South Korea ToolGen, Inc. is a biotechnology company focused on the development and application of genome editing technologies. It creates, and holds intellectual property rights for essential tools and technologies for editing the genetic information in microbial, plant, animal, and human cells. ToolGen’s mission is to translate the potential of our innovative platform technology into transformative products for biomedicine and agriculture. For more information, please visit www.toolgen.com.


SEOUL, South Korea, May 15, 2017 (GLOBE NEWSWIRE) -- ToolGen, Inc. (KONEX, 199800), a biotechnology company specializing in genome editing, presented results from a study highlighting the use of CRISPR/Cas9 nucleases for specific and efficient genome editing in human primary T-cells, without causing severe cell damage and growth retardation in a poster presentation at the American Society for Gene and Cell Therapy (ASGCT) Annual Meeting being held in Washington, DC from May 10-13, 2017. The poster presentation entitled “Genome editing of human T cells to generate CAR-T cells with enhanced antitumor function” aimed to study the proposed idea that T-cell anergy serves to induce dysfunction in a wide variety of tumor microenvironments. Using CRISPR/Cas9-mediated gene editing, the researchers were able to successfully knock out DGKα, a tolerance-associated gene at an 80% efficiency rate, which allowed T-cells to reverse hypofunction induced by anergy. Based on this result, the researchers combined the lentiviral delivery of CAR and the electroporation of RNP to generate a CRISPR gene-edited CAR-T cell. These edited CAR-T cells demonstrated lower sensitivity to immunosuppressive conditions and were hyper-responsive against tumor cells. “We are honored to be recognized by ASGCT for our work using CRISPR/Cas9-mediated gene editing,” commented Seokjoong Kim, Research Director at Toolgen. “The data presented in the poster presentation demonstrates preliminary success in utilizing CRISPR/Cas9 nucleases for specific and efficient gene editing in human primary T-cells. The findings suggest that CAR and TCR T cells with a disrupted anergy-related gene may be potent effector cells against various cancers. We believe that these results continue to validate our approach to gene editing and provide support for further research to move our pipeline of opportunities into the clinic.” The authors of the presentation were Yoon-Young Kim, In Young Jung, Ho Sung Yu1, Seokjoong Kim, R&D Center, Toolgen, Inc., Seoul, South Korea ToolGen, Inc. is a biotechnology company focused on the development and application of genome editing technologies. It creates, and holds intellectual property rights for essential tools and technologies for editing the genetic information in microbial, plant, animal, and human cells. ToolGen’s mission is to translate the potential of our innovative platform technology into transformative products for biomedicine and agriculture. For more information, please visit www.toolgen.com.


Patent
Toolgen Incorporated | Date: 2017-01-31

The disclosure provided herewith relates to a Campylobacter jejuni CRISPR/CAS system-derived RGEN and a use thereof.


SEOUL, South Korea, Feb. 22, 2017 (GLOBE NEWSWIRE) -- ToolGen, Inc. (KONEX, 199800), a biotechnology company specializing in genome editing, today announced encouraging data from a study evaluating Campylobacter jejuni Cas9 (CjCas9), the smallest Cas9 orthologue characterized to date, for efficient genome editing in vivo. Results from the study entitled “In vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni,” were published online in the peer-reviewed journal Nature Communications. The study was led by researchers from ToolGen, Dr. Jin-soo Kim, Principal Investigator, from the Institute for Basic Science and Dr. Jeong Hun Kim from Seoul National University Hospital. The study demonstrated the ability to package the CjCas9 gene, its sgRNA sequence into a single Adeno-associated virus (AAV) vector for in vivo gene surgery. Furthermore, CjCas9 was shown to be highly specific in cleaving the target sites in the human or mouse genome in vitro, which could have significant potential precision genome editing and gene surgery. CjCas9 delivered via AAV, induced target mutations in mouse muscle cells and retinal pigment epithelium cells (RPE) with no off-target mutations detected in the genome. The study confirmed that CjCas9 targeted to the Vegfa or Hif1a gene in RPE cells reduced the size of laser-induced choroidal neovascularization, a condition leading to the formation of new blood vessels in the choroid layer of the eye. This suggests that gene surgery with CjCas9 could be a promising treatment option for age-related macular degeneration, a leading cause of blindness in adults. Seokjoong Kim, Research Director of ToolGen commented, “Despite the recent advances in CRISPR/Cas9 genome editing technology, it has been difficult to package a whole cassette of Cas9 and sgRNA into certain viral vectors such as AAV owing to the large size of the most commonly used Cas9 genes. The need to split the gene and package it into multiple AAV vectors results in a less than optimal delivery method, and the split gene is less active than its intact counterpart. However, our study, utilizing CjCas9, which is significantly smaller than other commonly used Cas9 genes, enables us to overcome these problems. We are very pleased with the findings obtained from this study and strongly believe that this data can serve as a foundation for further exploration in the CRISPR/Cas9 arena.” Jeong Hun Kim, Clinical Professor in the Department of Ophthalmology, Seoul National University Hospital commented, “This work shows promising results for the application of CRISPR/Cas9 for eye diseases. We look forward to extending these observations on the successful modulation of neovascularization through targeted in vivo genome editing to additional disease models and large animals that could potentially establish a new class of therapeutic options. Additionally, with an efficient genome editing process established in the eye, we can broaden target indications to include rare diseases.” For more information, contact Seokjoong Kim, Director of Research Center Tel. +8210-6776-7824, sj.kim@toolgen.com. ToolGen, Inc. is a biotechnology company focused on the development and application of genome editing technologies. It creates, and holds intellectual property rights for essential tools and technologies for editing the genetic information in microbial, plant, animal, and human cells. ToolGen’s mission is to translate the potential of our innovative platform technology into transformative products for biomedicine and agriculture. For more information, please visit www.toolgen.com.


The present invention relates to targeted genome editing in eukaryotic cells or organisms. More particularly, the present invention relates to a composition for cleaving a target DNA in eukaryotic cells or organisms comprising a guide RNA specific for the target DNA and Cas protein-encoding nucleic acid or Cas protein, and use thereof.


The present invention relates to targeted genome editing in eukaryotic cells or organisms. More particularly, the present invention relates to a composition for cleaving a target DNA in eukaryotic cells or organisms comprising a guide RNA specific for the target DNA and Cas protein-encoding nucleic acid or Cas protein, and use thereof.


The present invention relates to targeted genome editing in eukaryotic cells or organisms. More particularly, the present invention relates to a composition for cleaving a target DNA in eukaryotic cells or organisms comprising a guide RNA specific for the target DNA and Cas protein-encoding nucleic acid or Cas protein, and use thereof.

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