Viromed Co., Ltd.

Viromed Limited

Neuracle Genetics Inc.

Neuracle Science Co., Ltd.

Helixmith Co., Ltd

Jong-Mook Kim

Woong Hahn

Eun-Jin Park

Sunyoung Kim

Sujeong Kim

Song Xu

Seung Shin Yu

Dong Sik Kim

Soon-gu Kwon

Juwon Shim

Seong-Hyun Ho

Joo Seok Han

Hoon Young Kong

Youngtae Hong

WonSun Yoo

Eun Young Han

Jee Yong Kim

Sunhwa Shin

Kyungwon Lee

Wonsun Yoo

Song Shan Xu

The present disclosure provides adeno-associated vims (AAV) vectors and uses thereof. In certain embodiments, the AAV vectors comprise a nucleic acid that encodes an antagonist against a family with sequence similarity 19, member A5 (FAM19A5) protein, e.g., anti-FAM19A5 antibody, e.g., anti-FAM19A5 scFv.

Adeno-associated virus (AAV) delivery of anti-FAM19A5 antibodies

Novel intron fragments are provided. The intron fragments can increase gene expression to levels equal to or higher than those achieved by the full-length intron while maintaining their ability to increase gene expression even when combined with various types of promoters and splicing donors. Particularly, the intron fragments enable loading of larger transgenes when used in genetic information delivery systems whose size is limited, for example, adeno-associated viruses (AAVs) and rhabdoviruses. Therefore, the use of the intron fragments is expected to extend the range of therapeutic genes.

Intron fragments

The present invention relates to a hybrid Hepatocyte Growth Factor (HGF) gene which is prepared by inserting an inherent or foreign intron between exons 4 and 5 in HGF cDNA, which has a base sequence of SEQ ID NO: 2. The gene has high expression efficiency and simultaneously expresses two heterotypes of HGF and dHGF (deleted variant HGF). Further the gene may be used for treating or preventing ischemic or liver diseases.

Hybrid hepatocyte growth factor gene having high expression efficiency of two heterotypes of hepatocyte growth factor

The present invention provides for a method of treating or preventing ischemic or liver disease in a subject by administering a composition reconstituted from a lyophilized hepatocyte growth factor (HGF) DNA formulation, where the DNA formulation comprises an HGF plasmid DNA, salt and a carbohydrate. The invention further provides for a method of making such a lyophilized DNA formulation that preserves or enhances gene expression both in vitro and in vivo, thus maintaining or stimulating the biological activity of the expressed protein. The invention also provides for the DNA formulation, or the lyophilized DNA formulation according to the methods disclosed.

Lyophilized DNA formulations for enhanced expression of plasmid DNA

The present invention relates to compositions and methods for fusion protein separation utilizing a peptide linker comprising a novel thrombin cleavage site.

Compositions and methods for fusion protein separation

The present invention relates to the compositions for a gene therapy of rheumatoid arthritis including a gene encoding an anti-angiogenic protein or parts thereof. More specifically, the present invention provides a gene therapy of rheumatoid arthritis by preparing a recombinant vector that expresses a gene encoding an anti-angiogenic protein such as angiostatin or parts thereof, and transplanting the recombinant vector or a cell that is transfected or transduced with the recombinant vector into the affected area of a patient, and also provides the compositions for the gene therapy. The compositions for the gene therapy according to the present invention can be used effectively for the treatment of rheumatoid arthritis, for which effective treating methods have not been developed until now, by providing the anti-angiogenic proteins into the knee of a patient continuously to prevent the synovial tissue hyperplasia and the resulting inflammation.

Compositions for gene therapy of rheumatoid arthritis including a gene encoding an anti-angiogenic protein or parts thereof

The present invention provides a retroviral vector containing an iduronate-2-sulfatase (IDS) gene but without selective marker gene, constructed on, as a backbone, a minimum sized MLV-based retroviral vector which does not contain any viral coding sequences but can still produce a high viral titer and drive a high level of gene expression.

Construction of high efficiency retroviral vector for gene therapy of hunter syndrome

The present invention relates to improved retroviral vectors for gene therapy. In this invention, retroviral vectors with higher safety and efficiency are constructed from MLV-based starting vectors, MON and MIN. The improved vectors have the following features: 1) sequences corresponding to MLV-derived pol gene are completely deleted in the vectors, avoiding homologous recombination which has been a baffling problem in conventional retroviral vectors, 2) a heterologous intron, splicing acceptor and/or non-coding sequence are/is inserted into the upstream position of a cloning site, maximizing the expression of a foreign gene through efficient splicing, 3) the vectors contain either the full-length U3 sequence of 5&prime; LTR or a strong heterologous promoter instead, permitting the abundant production of RNA, 4) either IRES (Internal Ribosomal Entry Site) or internal SV40 minimal promoter is inserted into the downstream position of cloning site, enabling the simultaneous expression of two or more foreign genes. Since the improved retroviral vectors of this invention turn out to be safe and to express the foreign gene efficiently, they are useful for gene therapy and the like.

High efficiency retroviral vectors that contain none of viral coding sequences

Growing community of inventors

Seoul, South Korea

Jong-Mook Kim