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1. 发明申请

WO2022245919A1 METHODS AND COMPOSITIONS FOR TREATING OCULAR DISEASES AND DISORDERS 审中-公开
公开(公告)号：WO2022245919A1
公开(公告)日：2022-11-24
申请号：PCT/US2022/029797
申请日：2022-05-18
申请人： ABEONA THERAPEUTICS INC. , PADEGIMAS, Linas , KEVANY, Brian
发明人： PADEGIMAS, Linas , KEVANY, Brian
IPC分类号： A61K35/76 , A61P27/02 , C12N15/35 , C12N15/864
摘要： Provided herein are recombinant AAV vectors, AAV viral vectors, capsid proteins, and administration methods for improved gene therapy, and methods for their manufacture and use.

2. 发明申请

WO2022015788A1 RECOMBINANT ADENO-ASSOCIATED VIRAL VECTORS FOR MULTIPARTITE GENE DELIVERY 审中-公开
公开(公告)号：WO2022015788A1
公开(公告)日：2022-01-20
申请号：PCT/US2021/041527
申请日：2021-07-14
申请人： ABEONA THERAPEUTICS INC. , PADEGIMAS, Linas , BARRETT, Brianna
发明人： PADEGIMAS, Linas , BARRETT, Brianna
IPC分类号： C12N15/85
摘要： Provided herein are recombinant AAV vectors, AAV viral vectors, and capsid proteins for improved gene therapy, and methods for their manufacture and use in a multipartite (e.g., bipartite) delivery system. The vectors provide portions of transgene and direct their recombination in a cell to provide complete transgenes.

3. 发明申请

WO2009036280A1 LONG-TERM IN VIVO TRANSGENE EXPRESSION 审中-公开
标题翻译： VIVO TRANSGENE EXPRESSION的长期使用
公开(公告)号：WO2009036280A1
公开(公告)日：2009-03-19
申请号：PCT/US2008/076177
申请日：2008-09-12
申请人： COPERNICUS THERAPEUTICS, INC. , COOPER, Mark J. , PADEGIMAS, Linas
发明人： COOPER, Mark J. , PADEGIMAS, Linas
IPC分类号： C12N15/85
CPC分类号： C07K14/4712 , A61K48/0066 , C12N15/85 , C12N2799/04 , C12N2830/42 , C12N2830/50 , C12N2830/85 , C12N2840/00 , C12N2840/85
摘要： Efficient and prolonged hCFTR expression is one of the major obstacles for cystic fibrosis lung therapy. hCFTR mRNA expression levels depend on eukaryotic expression cassette components, prokaryotic backbone elements, and the gene transfer method may also influence transcriptional silencing mechanisms. A codon-optimized and CpG-reduced human CFTR gene (CO-CFTR) was made. Various vector modifications were tested to facilitate extended duration of CO-CFTR expression. Insertion of an extended 3'BGH transcribed sequence (712 bp) in an inverted orientation produced prolonged expression of CO-CFTR expression at biologically relevant levels. Further studies revealed that prolonged CO-CFTR expression is dependant on the orientation of the extended BGH 3' BGH transcribed sequence and its transcription, is not specific to the UbC promoter, and is less dependent on other vector backbone elements.
摘要翻译：有效和长期的hCFTR表达是囊性纤维化肺部治疗的主要障碍之一。 hCFTR mRNA表达水平取决于真核表达盒组分，原核骨架元素，基因转移方法也可能影响转录沉默机制。制备了密码子优化和CpG还原的人CFTR基因（CO-CFTR）。测试各种载体修饰以促进CO-CFTR表达的延长的持续时间。以倒置方向插入延伸的3'BGH转录序列（712bp）产生CO-CFTR表达在生物相关水平上的延长表达。进一步的研究表明，延长的CO-CFTR表达依赖于扩展的BGH 3'BGH转录序列的方向及其转录，对UbC启动子不是特异性的，并且较少依赖于其它载体骨架元素。

4. 发明申请

WO2008045548A3 CODON OPTIMIZED CFTR 审中-公开
公开(公告)号：WO2008045548A3
公开(公告)日：2008-04-17
申请号：PCT/US2007/021862
申请日：2007-10-12
申请人： COPERNICUS THERAPEUTICS INC. , COOPER, Mark, J. , PADEGIMAS, Linas
发明人： COOPER, Mark, J. , PADEGIMAS, Linas
IPC分类号： C07K14/47 , A61K48/00
摘要： A synthetic hCFTR DNA sequence has been developed that produces remarkably high levels of hCFTR mRNA and protein in dosed murine lungs and human cells in culture compared to the natural hCFTR cDNA. This synthetic DNA addresses.problems inherent in some natural cDNAs, such as premature transcriptional truncation sites introduced during cDNA synthesis. Introns are initially present in mRNA until the mRNA is processed. cDNA made from processed mRNA is devoid of introns. Thus DNA sequences (exon junctions) are present in a cDNA molecule which are not present in cells in nature. These exon junctions may affect transcription. Methods for improving expression of CFTR are based on sequence changes in cDNA molecules. The improvement methods may be applied to other cDNA molecules which are refractory to in vivo expression efforts. Compositions embodying the sequence changes increase the production of both transgenic mRNA and protein from cDNA molecules.

5. 发明申请

WO2020117898A1 RECOMBINANT ADENO-ASSOCIATED VIRAL VECTOR FOR GENE DELIVERY 审中-公开
公开(公告)号：WO2020117898A1
公开(公告)日：2020-06-11
申请号：PCT/US2019/064396
申请日：2019-12-04
申请人： ABEONA THERAPEUTICS INC.
发明人： MILLER, Timothy J. , PADEGIMAS, Linas
IPC分类号： A61K48/00 , C12Q1/70 , C07K14/005 , C07K14/015 , C12N15/86 , C12N15/864
摘要： Provided herein are recombinant AAV vectors, AAV viral vectors, and capsid proteins for improved gene therapy, and methods for their manufacture and use.

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