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公开(公告)号：US20240124896A1
公开(公告)日：2024-04-18
申请号：US18344732
申请日：2023-06-29
申请人： Seattle Children's Hospital (d/b/a Seattle Children's Research Institute) , Fred Hutchinson Cancer Center
发明人： Andrew Scharenberg , Kyle Jacoby , Hans-Peter Kiem , David J. Rawlings , Christopher Lux , Sowmya Pattabhi , Olivier M. Humbert
CPC分类号： C12N15/907 , A61K35/28 , A61P9/00 , C07K14/805 , C12N15/11 , C12N15/86 , C12N2310/20 , C12N2750/14143 , C12N2800/80
摘要： The present disclosure provides improved compositions for the homology directed repair of the human globin locus for the prevention, treatment, or amelioration of at least one symptom of a hemoglobinopathy.
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公开(公告)号：US12110499B2
公开(公告)日：2024-10-08
申请号：US16608182
申请日：2018-04-24
发明人： Andrew Scharenberg , Kyle Jacoby , Hans-Peter Kiem , David J. Rawlings , Christopher Lux , Sowmya Pattabhi , Olivier M. Humbert
CPC分类号： C12N15/907 , A61K35/28 , A61P9/00 , C07K14/805 , C12N15/11 , C12N15/86 , C12N2310/20 , C12N2750/14143 , C12N2800/80
摘要： The present disclosure provides improved compositions for the homology directed repair of the human globin locus for the prevention, treatment, or amelioration of at least one symptom of a hemoglobinopathy.
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公开(公告)号：US20240238438A1
公开(公告)日：2024-07-18
申请号：US18558014
申请日：2022-04-29
IPC分类号： A61K47/68 , A61K31/7088 , A61K38/46 , A61K47/69 , A61P7/06 , C12N9/22 , C12N15/11 , C12N15/88
CPC分类号： A61K47/6849 , A61K31/7088 , A61K38/465 , A61K47/6925 , A61P7/06 , C12N9/22 , C12N15/11 , C12N15/88 , C12N2310/20
摘要： The present invention relates to compositions and methods for effective delivery of an agent to a stem cell using a delivery vehicle, such as a lipid nanoparticle (LNP), comprising a CD90 targeting domain.
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4.发明公开公开(公告)号：US20240091265A1
公开(公告)日：2024-03-21
申请号：US18208490
申请日：2023-06-12
发明人： Jarrod Dudakov , Marcel van den Brink , Enrico Velardi , Hans-Peter Kiem , Stefan Radtke , Scott James
IPC分类号： A61K35/28 , A61K35/12 , A61P35/00 , C12N5/0789 , C12N15/86
CPC分类号： A61K35/28 , A61K35/12 , A61P35/00 , C12N5/0647 , C12N15/86
摘要： The use of luteinizing hormone receptor (LHR) binding agents and luteinizing hormone (LH) agonists to enrich for primitive hematopoietic stem cell (pHSC) populations, to target pHSC for ablation, and/or to expand pHSC populations are described. The methods can be used to prepare therapeutic hematopoietic stem cell (HSC) populations, to prepare patients for therapeutic HSC transplants, and/or to treat malignancies, such as those associated with hyperproliferative HSC.
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5.发明公开公开(公告)号：US20230159895A1
公开(公告)日：2023-05-25
申请号：US18057098
申请日：2022-11-18
IPC分类号： C12N5/0789 , A61K35/28 , G01N33/569
CPC分类号： C12N5/0647 , A61K35/28 , G01N33/56972 , C12N2501/125 , C12N2501/145 , C12N2501/26 , C12N2510/00 , G01N2333/70589 , G01N2333/70596
摘要： Strategies to assess and/or produce cell populations with predictive engraftment potential are described. The cell populations can be used for a variety of therapeutic and research purposes.
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公开(公告)号：US20230313224A1
公开(公告)日：2023-10-05
申请号：US17995671
申请日：2021-04-12
发明人： Andre Lieber , Hans-Peter Kiem , Hongjie Wang
IPC分类号： C12N15/86 , C07K14/075 , C07K14/755 , C12N5/0789 , A61P7/00
CPC分类号： C12N15/86 , A61P7/00 , C07K14/075 , C07K14/755 , C12N5/0647 , C12N2710/10343 , C12N2800/90
摘要： The current disclosure provides recombinant adenoviral vectors and adenoviral genomes that can accommodate or that contain a large transposon payload, for instance a transposon payload of up to 40 kb. The adenoviral vectors and genomes can deliver the large transposon payload into a target genome, for instance for gene therapy.
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公开(公告)号：US20220380776A1
公开(公告)日：2022-12-01
申请号：US17771128
申请日：2020-10-22
发明人： Olivier Humbert , Hans-Peter Kiem , Roland B. Walter , Andre Lieber , Chang Li
IPC分类号： C12N15/113 , C12N5/00 , C12N5/0789 , C12N9/22
摘要： Systems and methods to selectively protect therapeutic cells by reducing CD33 expression in the therapeutic cells using base editors and targeting non-therapeutic cells with an anti-CD33 therapy are described. The selective protection results in the enrichment of the therapeutic cells while simultaneously targeting any diseased, malignant and/or non-therapeutic CD33 expressing cells within a subject.
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公开(公告)号：US11786557B2
公开(公告)日：2023-10-17
申请号：US16753277
申请日：2018-10-02
发明人： Jarrod Dudakov , Marcel van den Brink , Enrico Velardi , Hans-Peter Kiem , Stefan Radtke , Scott James
IPC分类号： A61K35/28 , A61P35/00 , C12N5/0789 , C12N15/86 , A61K35/12
CPC分类号： A61K35/28 , A61K35/12 , A61P35/00 , C12N5/0647 , C12N15/86
摘要： The use of luteinizing hormone receptor (LHR) binding agents and luteinizing hormone (LH) agonists to enrich for primitive hematopoietic stem cell (pHSC) populations, to target pHSC for ablation, and/or to expand pHSC populations are described. The methods can be used to prepare therapeutic hematopoietic stem cell (HSC) populations, to prepare patients for therapeutic HSC transplants, and/or to treat malignancies, such as those associated with hyperproliferative HSC.
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公开(公告)号：US20230133243A1
公开(公告)日：2023-05-04
申请号：US17995687
申请日：2021-04-12
发明人： Andre Lieber , Hans-Peter Kiem
IPC分类号： A61K48/00 , A61K38/20 , A61K39/395 , A61K31/436 , A61K31/573 , A61K38/19 , A61K31/395 , A61P37/06 , A61P7/06
摘要： The present disclosure provides, among other things, immune suppression regimens for in vivo gene therapy and uses thereof. In various embodiments of the present disclosure, in vivo gene therapy includes delivery of at least one exogenous coding nucleic acid sequence to a stem cell of the subject. Success of in vivo gene therapy can be inhibited or reduced by immunotoxicity. The present disclosure provides compositions and methods, including among other things immune suppression regimens, that reduce immunotoxicity of in vivo gene therapy, e.g., in vivo gene therapy including administration of a viral gene therapy vector to a subject.
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公开(公告)号：US20200009266A1
公开(公告)日：2020-01-09
申请号：US16486442
申请日：2018-02-15
申请人： Fred Hutchinson Cancer Research Center , Seattle Children's Hospital d/b/a Seattle Children's Research Institute
发明人： Frieda Chan , Olivier Humbert , Hans-Peter Kiem , Jennifer E. Adair , David Rawlings , Andrew Scharenberg , Troy Torgerson
IPC分类号： A61K48/00 , A61K38/19 , A61K31/395 , A61P37/00
摘要： In vivo gene therapies for immune deficiencies are described. The in vivo gene therapies utilize a foamy viral vector including a PGK promoter with a therapeutic gene. The foamy viral vector can be beneficially administered with cell mobilization into the peripheral blood.
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