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公开(公告)号：US12150960B2
公开(公告)日：2024-11-26
申请号：US17108076
申请日：2020-12-01
Inventor： Lei Xiao , Zhiyuan Cao , Chengfei Pu , He Sun
Abstract: The present disclosure relates to compositions and methods for enhancing T cell response and/or CAR cell expansion and/or maintenance in vivo and/or in vitro. For example, a method of enhancing T cell-based therapy comprises administering genetically modified T cells comprising a first chimeric antigen receptor (CAR) and a second CAR, wherein a binding domain of the first CAR binds a first antigen, and a binding domain of the second CAR binds a second antigen. The first antigen is different from the second antigen. In embodiments, the first CAR binds a surface molecule or antigen of a white blood cell.
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92.发明申请
公开(公告)号：US20240384297A1
公开(公告)日：2024-11-21
申请号：US18690068
申请日：2022-05-27
Applicant: BEIJING GENECRADLE PHARMACEUTICAL CO., LTD.
Inventor： Xiaobing WU
IPC: C12N15/86 , A61K38/47 , A61K48/00 , C12N9/24 , C12N15/113
Abstract: Provided are a constitutive promoter CAR-Mut, an expression construct comprising the promoter and a GAA coding nucleotide sequence functionally linked thereto, a recombinant vector and a host cell. Also provided are a composition and method for delivering a GAA coding polynucleotide to a mammalian cell or an individual using the recombinant vector, and for treating a subject with Pompe disease or acid glucosidase deficiency.
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公开(公告)号：US20240384293A1
公开(公告)日：2024-11-21
申请号：US18288345
申请日：2022-04-27
Applicant: Novartis AG
Inventor： Astrid Bosse , Benoit Bossuge , Laurence Croute , Lars Ellenrieder , Laurence Guianvarch , David Schmitt , Eleonora Toffoli
IPC: C12N15/86
Abstract: The disclosure provides, at least in part, to a method for producing high titer lentiviral vectors, and for producing lentiviral particles carrying a transgene of interest and under satisfactory safety conditions. The disclosure also provides at least in part, methods of purification of such lentiviral particle, e.g., from a cell culture. The disclosure also provides a formulation to lentiviral preparations that maintain structural integrity of the viral vector during purification, storage, and gene transfer events.
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公开(公告)号：US20240384271A1
公开(公告)日：2024-11-21
申请号：US18626784
申请日：2024-04-04
Applicant: Genzyme Corporation
Inventor： Seng Cheng , Sarah Melissa Jacobo , Takako Moriguchi , Catherine O'Riordan , Guoxiang Ruan
IPC: C12N15/113 , A61K45/06 , A61P21/00 , C12N15/86
Abstract: Provided herein are RNAi molecules for treating myotonic dystrophy type 1 (DM1). Further provided herein are expression cassettes, vectors (e.g., rAAV), viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat DM1.
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95.发明申请
公开(公告)号：US20240384258A1
公开(公告)日：2024-11-21
申请号：US18667037
申请日：2024-05-17
Inventor： Melina Claussnitzer , Liang He , Manolis Kellis , Xinchen Wang
Abstract: Embodiments disclosed herein provide a general, scalable, high-throughput, and high-resolution approach for experimental dissection of regulatory regions and driver nucleotides in the context of human biology and disease. Applicants present HiDRA, a novel high-resolution global screen for transcriptional regulatory activity in accessible chromatin regions, enabling high-efficiency, high-throughput, and high-resolution inference of regulatory activity.
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公开(公告)号：US20240384244A1
公开(公告)日：2024-11-21
申请号：US18620632
申请日：2024-03-28
Applicant: Timothy Ley
Inventor： Timothy Ley
IPC: C12N9/10 , A61K31/706 , A61K38/07 , A61K48/00 , C12N15/86
Abstract: Treatment and prevention of DNMT3A deficiency-associated diseases are provided. Compositions for treatment or prevention include at least one cDNA vector and/or at least one therapeutic agent capable of restoring DNMT3L activity, restoring DNMT3L expression, increasing DNMT3L activity, increasing DNMT3L expression, and/or increasing DNMT3A enzymatic activity. Methods for treatment or prevention include administering to a subject a composition including the at least one cDNA vector and/or at least one therapeutic agent. Methods of increasing DNMT3A activity in a subject having a DNMT3A mutation, methods of reversing a hypomethylation phenotype in bone marrow cells of a subject having a DNMT3A mutation, and methods of promoting cancer cell death in a subject having Acute Myeloid Leukemia (AML) are also provided.
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公开(公告)号：US20240384242A1
公开(公告)日：2024-11-21
申请号：US18688691
申请日：2022-09-02
Applicant: BIOMARIN PHARMACEUTICAL INC.
Inventor： Peter COLOSI , Vincent LEONARD , Silvia RAMIREZ , Justin ISHIDA , Yu-Shan TSENG , Teague STERLING
IPC: C12N7/00 , A61K48/00 , C07K14/005 , C12N15/86
Abstract: The disclosure provides various compositions comprising novel adeno-associated virus (AAV) capsid sequences and functional fragments thereof. Also provided, are methods of delivery, treatment and manufacture using the compositions provided by the disclosure.
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公开(公告)号：US20240382521A1
公开(公告)日：2024-11-21
申请号：US18555333
申请日：2022-04-21
Applicant: Baylor College of Medicine
Inventor： Maksim Mamonkin , Norihiro Watanabe , Feiyan Mo
IPC: A61K35/17 , A61K39/00 , A61P35/02 , C07K16/28 , C12N5/0781 , C12N5/0783 , C12N15/86
Abstract: Embodiments of the disclosure include methods and compositions related to targeting of antigen-expressing cells with particular engineered antigen receptors expressed by immune cells. In specific embodiments, immune cells specifically engineered to express particular antigen receptor constructs are cultured in the presence of kinase inhibitors and exhibit reduced fratricidal activity compared to immune cells cultured in the absence of kinase inhibitors. In some embodiments, the genetically engineered immune cells having reduced fratricidal activity are used to treat diseases in subjects, and the fratricidal activity of the genetically engineered immune cells is restored in vivo after substantial elimination of the diseased cells, resulting in elimination of the genetically engineered immune cells.
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公开(公告)号：US12146169B2
公开(公告)日：2024-11-19
申请号：US17059723
申请日：2019-05-27
Inventor： Maria Fátima Bosch Tubert , Victor Sanchez Clares , Albert Ribera Sanchez , Virginia A. Haurigot
Abstract: The present invention provides new polynucleotide sequences, adeno-associated virus-derived vectors and pharmaceutical compositions containing the same for the treatment of lysosomal storage disorders and specially, for the treatment of mucopolysaccharidosis type IVA or Morquio A syndrome.
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公开(公告)号：US20240376497A1
公开(公告)日：2024-11-14
申请号：US18692672
申请日：2022-09-16
Applicant: Astellas Gene Therapies, Inc.
Inventor： Dwaipayan SEN , John T. GRAY , Joshua C. CHANG
Abstract: The invention provides compositions and methods for stimulating the expression of the human frataxin gene. The compositions described herein can be used, for instance, to produce genes and RNA equivalents optimized for expression in a particular cell type. The compositions and methods that can be used for treating Frederich ataxia. Using the compositions and methods of the disclosure, a patient (e.g., a mammalian patient, such as a human patient) having Frederich ataxia may be administered a plasmid (e.g., a viral vector) that contains a human frataxin gene (hFXN) or an RNA equivalent thereof.
