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1.发明公开公开(公告)号：US20240271111A1
公开(公告)日：2024-08-15
申请号：US18424277
申请日：2024-01-26
发明人： Derek Jantz , James Jefferson Smith
CPC分类号： C12N9/22 , A61K9/51 , A61K38/43 , A61P31/20 , C12N15/52 , C12N15/86 , A61K38/00 , C12N2730/10122 , C12N2750/14141
摘要： The present invention encompasses engineered meganucleases which recognize and cleave a recognition sequence within an open reading frame (ORF) of the genome of at least two genotypes of the Hepatitis B virus (HBV). The present invention also encompasses methods of using such engineered meganucleases in a pharmaceutical composition and in methods for treating or reducing the symptoms of a HBV infection, or treating hepatocellular carcinoma (HCC). Further, the invention encompasses pharmaceutical compositions comprising engineered meganuclease proteins, nucleic acids encoding engineered meganucleases, and the use of such compositions for treating HBV infections or HCC.
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公开(公告)号：US20240156919A1
公开(公告)日：2024-05-16
申请号：US18490484
申请日：2023-10-19
CPC分类号： A61K38/465 , A61K38/54 , C12N7/00 , C12N9/22 , C12N2750/14143
摘要： The invention relates to the field of molecular biology and recombinant nucleic acid technology. In particular, the invention relates to a method of treating a patient with Duchenne Muscular Dystrophy comprising the removal of at least one exon from the dystrophin gene using engineered nucleases.
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3.发明公开公开(公告)号：US20240002823A1
公开(公告)日：2024-01-04
申请号：US18360517
申请日：2023-07-27
发明人： Gary Owens , Janel Lape , James Jefferson Smith , John Morris , Caitlin Turner , Whitney Lewis , Derek Jantz
CPC分类号： C12N9/22 , C12N15/86 , C12N2750/14143
摘要： The present disclosure encompasses engineered meganucleases that bind and cleave recognition sequences within a dystrophin gene. The present disclosure also encompasses methods of using such engineered meganucleases to make genetically modified cells. Further, the disclosure encompasses pharmaceutical compositions comprising engineered meganuclease proteins, or polynucleotides encoding engineered meganucleases of the disclosure, and the use of such compositions for the modification of a dystrophin gene in a subject, or for treatment of Duchenne Muscular Dystrophy.
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公开(公告)号：US11753630B2
公开(公告)日：2023-09-12
申请号：US17931896
申请日：2022-09-13
发明人： Gary Owens , Janel Lape , James Jefferson Smith , John Morris , Caitlin Turner , Whitney Lewis , Derek Jantz
CPC分类号： C12N9/22 , C12N15/86 , C12N2750/14143
摘要： The present disclosure encompasses engineered meganucleases that bind and cleave recognition sequences within a dystrophin gene. The present disclosure also encompasses methods of using such engineered meganucleases to make genetically modified cells. Further, the disclosure encompasses pharmaceutical compositions comprising engineered meganuclease proteins, or polynucleotides encoding engineered meganucleases of the disclosure, and the use of such compositions for the modification of a dystrophin gene in a subject, or for treatment of Duchenne Muscular Dystrophy.
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公开(公告)号：US20230190780A1
公开(公告)日：2023-06-22
申请号：US17925277
申请日：2021-05-14
发明人： Christopher Ryan Heery , Daniel H. Fowler , Alan F. List , Aaron Martin , Daniel T. MacLeod , Derek Jantz
IPC分类号： A61K31/7056 , A61K31/675 , C07K16/28 , C07K14/725 , A61K31/436 , A61P35/00
CPC分类号： A61K31/7056 , A61K31/675 , C07K16/2893 , C07K14/7051 , A61K31/436 , A61P35/00 , A61K2039/545
摘要： The present invention encompasses methods for reducing the number of target cells in a subject, such as cancer cells. The methods include administration of genetically-modified human immune cells expressing a chimeric antigen receptor or exogenous T cell receptor, which have specificity for an antigen on the target cells Administration of the genetically-modified immune cells can be preceded by the administration of a lymphodepletion region and/or an immunosuppression regimen, to improve efficacy of the therapy and persistence of the cells in vivo.
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公开(公告)号：US20230088311A1
公开(公告)日：2023-03-23
申请号：US17812400
申请日：2022-07-13
发明人： Derek Jantz , James Jefferson Smith
IPC分类号： C12Q1/6811 , C12N9/22 , G06Q99/00
摘要： Methods of cleaving double-stranded DNA that can be recognized and cleaved by a rationally-designed, I-CreI-derived meganuclease are provided. Also provided are recombinant nucleic acids, cells, and organisms containing such recombinant nucleic acids, as well as cells and organisms produced using such meganucleases. Also provided are methods of conducting a custom-designed, I-CreI-derived meganuclease business.
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公开(公告)号：US20230036065A1
公开(公告)日：2023-02-02
申请号：US17782607
申请日：2020-12-03
IPC分类号： A61K35/17 , A61K39/395 , A61K31/664 , A61K31/7076 , C07K16/28 , A61K38/17 , A61P35/00 , A61P35/02
摘要： The present invention encompasses methods of cancer immunotherapy, and particularly methods of allogeneic cellular immunotherapy, using particular lymphodepletion regimens in combination with particular populations of chimeric antigen receptor T cells expressing anti CD19 CAR PBCAR0191, anti CD20 CAR PBCAR20A or anti BCMA CAR PBCAR269A.
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8.发明申请公开(公告)号：US20220333137A1
公开(公告)日：2022-10-20
申请号：US17585694
申请日：2022-01-27
摘要： Methods of inserting genes into defined locations in the chromosomal DNA of cultured mammalian cell lines which are subject to gene amplification are disclosed. In particular, sequences of interest (e.g., genes encoding biotherapeutic proteins) are inserted proximal to selectable genes in amplifiable loci, and the transformed cells are subjected to selection to induce co-amplification of the selectable gene and the sequence of interest. The invention also relates to meganucleases, vectors and engineered cell lines necessary for performing the methods, to cell lines resulting from the application of the methods, and use of the cell lines to produce protein products of interest.
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9.发明申请公开(公告)号：US20220243187A1
公开(公告)日：2022-08-04
申请号：US17665265
申请日：2022-02-04
发明人： Derek Jantz , James Jefferson Smith
摘要： The present invention encompasses engineered meganucleases which recognize and cleave a recognition sequence within an open reading frame (ORF) of the genome of at least two genotypes of the Hepatitis B virus (HBV). The present invention also encompasses methods of using such engineered meganucleases in a pharmaceutical composition and in methods for treating or reducing the symptoms of a HBV infection, or treating hepatocellular carcinoma (HCC). Further, the invention encompasses pharmaceutical compositions comprising engineered meganuclease proteins, nucleic acids encoding engineered meganucleases, and the use of such compositions for treating HBV infections or HCC.
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公开(公告)号：US11286291B2
公开(公告)日：2022-03-29
申请号：US16339699
申请日：2017-10-04
发明人： Derek Jantz , Aaron Martin , Jeffrey Sunman , Daniel T. MacLeod
IPC分类号： C07K14/715 , C07K14/725 , C07K14/705 , C07K16/28 , A61P35/00 , A61K35/17 , C12N5/0783 , C12N7/00 , A61K38/00 , A61K39/00 , C12N9/90
摘要： The present disclosure provides novel co-stimulatory domains useful in genetically-modified cells to promote cell proliferation and/or promote cytokine secretion after antigen recognition. For example, disclosed herein are genetically-modified cells comprising a chimeric antigen receptor or an inducible regulatory construct incorporating the co-stimulatory domains disclosed herein. Also disclosed herein are plasmids and viral vectors comprising a nucleic acid sequence encoding the co-stimulatory domains, and methods of administering compositions comprising the novel co-stimulatory domains to subjects in order to reduce the symptoms, progression, or occurrence of disease, such as cancer.
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