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公开(公告)号:US20240148771A1
公开(公告)日:2024-05-09
申请号:US18317447
申请日:2023-05-15
IPC分类号: A61K31/7105 , A61K31/711 , A61K31/7115 , A61K31/712 , A61K31/7125 , A61K31/713 , A61K38/00 , C07K19/00 , C12N9/22 , C12N15/00 , C12N15/10 , C12N15/62 , C12N15/82 , C12N15/90
CPC分类号: A61K31/7105 , A61K31/711 , A61K31/7115 , A61K31/712 , A61K31/7125 , A61K31/713 , A61K38/00 , C07K19/00 , C12N9/22 , C12N15/00 , C12N15/102 , C12N15/62 , C12N15/8213 , C12N15/825 , C12N15/8251 , C12N15/902 , C12N15/907 , A61K48/00 , C07K2319/01
摘要: Provided herein are compositions and methods for modifying a predetermined nucleic acid sequence. A programmable nucleoprotein molecular complex containing a polypeptide moiety and a specificity conferring nucleic acid (SCNA) which assembles in-vivo, in a target cell, and is capable of interacting with the predetermined target nucleic acid sequence is provided. The programmable nucleoprotein molecular complex is capable of specifically modifying and/or editing a target site within the target nucleic acid sequence and/or modifying the function of the target nucleic acid sequence.
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公开(公告)号:US20240139294A1
公开(公告)日:2024-05-02
申请号:US18298913
申请日:2023-04-11
发明人: Kamel KHALILI , Wenhui Hu
CPC分类号: A61K38/465 , A61K9/0034 , A61K35/12 , A61K45/06 , A61K48/00 , A61K48/005 , C12N7/00 , C12N9/22 , C12N15/111 , C12N2310/20 , C12N2320/30 , C12N2740/16063 , C12Y301/21
摘要: A method of preventing transmission of a retrovirus from a mother to her offspring, by administering to the mother a therapeutically effective amount of a composition comprising a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease, and the two or more different multiplex gRNAs, wherein each of the at least two gRNAs is complementary to a different target nucleic acid sequence in a long terminal repeat (LTR) of proviral DNA of the virus that is unique from the genome of the host cell, cleaving a double strand of the proviral DNA at a first target protospacer sequence with the CRISPR-associated endonuclease, cleaving a double strand of the proviral DNA at a second target protospacer sequence with the CRISPR-associated endonuclease, excising an entire HIV-1 proviral genome, eradicating the HIV-1 proviral DNA from the host cell, and preventing transmission of the proviral DNA to the offspring.
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公开(公告)号:US11965000B2
公开(公告)日:2024-04-23
申请号:US18463276
申请日:2023-09-07
申请人: CureVac SE
发明人: Thomas Kramps , Margit Schnee , Daniel Voss , Benjamin Petsch
IPC分类号: C07K14/005 , A61K39/12 , A61K39/155 , C07K16/10 , C12N7/00 , A61K39/00 , A61K48/00
CPC分类号: C07K14/005 , A61K39/12 , A61K39/155 , C07K16/1027 , C12N7/00 , A61K2039/505 , A61K2039/53 , A61K2039/6031 , A61K48/00 , C07K2317/24 , C12N2760/18534
摘要: The present invention relates to an mRNA sequence, comprising a coding region, encoding at least one antigenic peptide or protein of RSV infections Respiratory syncytial virus (RSV) or a fragment, variant or derivative thereof. Additionally the present invention relates to a composition comprising a plurality of mRNA sequences comprising a coding region, encoding at least one antigenic peptide or protein of RSV infections Respiratory syncytial virus (RSV) or a fragment, variant or derivative thereof. Furthermore it also discloses the use of the mRNA sequence or the composition comprising a plurality of mRNA sequences for the preparation of a pharmaceutical composition, especially a vaccine, e.g. for use in the prophylaxis or treatment of RSV infections Respiratory syncytial virus (RSV) infections. The present invention further describes a method of treatment or prophylaxis of RSV infections using the mRNA sequence.
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公开(公告)号:US20240123035A1
公开(公告)日:2024-04-18
申请号:US18177455
申请日:2023-03-02
申请人: ModernaTX, Inc.
发明人: Antonin DE FOUGEROLLES , Kristy M. Wood , Sayda M. ELBASHIR , Noubar B. Afeyan , Pedro VALENCIA , Jason P. SCHRUM
IPC分类号: A61K38/19 , A61K9/00 , A61K9/127 , A61K9/14 , A61K9/16 , A61K31/7088 , A61K31/7105 , A61K38/48 , A61K48/00 , C07K2/00 , C07K14/535 , C12N15/00 , C12N15/117 , C12N15/88 , C12P21/00
CPC分类号: A61K38/193 , A61K9/0019 , A61K9/0024 , A61K9/0048 , A61K9/1271 , A61K9/1272 , A61K9/14 , A61K9/16 , A61K9/1647 , A61K31/7088 , A61K31/7105 , A61K38/4833 , A61K48/00 , A61K48/0033 , A61K48/0041 , A61K48/005 , A61K48/0066 , C07K2/00 , C07K14/535 , C12N15/00 , C12N15/117 , C12N15/88 , C12P21/00
摘要: The present disclosure provides, inter alia, formulation compositions comprising modified nucleic acid molecules which may encode a protein, a protein precursor, or a partially or fully processed form of the protein or a protein precursor. The formulation composition may further include a modified nucleic acid molecule and a delivery agent. The present invention further provides nucleic acids useful for encoding polypeptides capable of modulating a cell's function and/or activity.
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公开(公告)号:US11957765B2
公开(公告)日:2024-04-16
申请号:US16808206
申请日:2020-03-03
申请人: Genzyme Corporation
发明人: Marco A. Passini , James Dodge
IPC分类号: A61K48/00 , C07K14/47 , C12N15/86 , C12N15/864
CPC分类号: A61K48/0075 , A61K48/00 , C07K14/47 , C12N15/86 , C12N15/8645 , C12N2750/14041 , C12N2750/14143 , C12N2799/025
摘要: The disclosure pertains to methods and compositions for treating disorders affecting the central nervous system (CNS). These disorders include neurometabolic disorders such as lysosomal storage diseases that affect the central nervous system, e.g., Niemann-Pick A disease. They also include disorders such as Alzheimer's disease. The disclosed methods involve contacting an axonal ending of a neuron with a composition containing high titer AAV carrying a therapeutic transgene so that the AAV vector is axonally transported in a retrograde fashion and transgene product is expressed distally to the administration site.
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公开(公告)号:US20240115598A1
公开(公告)日:2024-04-11
申请号:US18446743
申请日:2023-08-09
申请人: Sanofi
发明人: Timothy Wright
IPC分类号: A61K31/712 , A61K9/00 , A61K48/00 , A61P13/12 , C12N15/113
CPC分类号: A61K31/712 , A61K9/0019 , A61K48/00 , A61P13/12 , C12N15/113 , C12N2310/11 , C12N2310/141
摘要: Provided herein are methods for the treatment of Alport syndrome, using a modified oligonucleotide targeted to miR-21. In certain embodiments, the modified oligonucleotide targeted to miR-21 improves kidney function and/or reduces fibrosis in subjects having Alport syndrome. In certain embodiments, administration of the modified oligonucleotide targeted to miR-21 delays the onset of end-stage renal disease in a subject having Alport syndrome. In certain embodiments, the modified oligonucleotide targeted to miR-21 delays the need for dialysis or kidney transplant in a subject having Alport syndrome.
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公开(公告)号:US20240101607A1
公开(公告)日:2024-03-28
申请号:US18314773
申请日:2023-05-09
发明人: Douglas Grant McFadden , Alfred S. Lewin , Alexandra Rose Lucas , Cristhian J. Ildefonso , Mohammed Masmudur Rahman
CPC分类号: C07K14/005 , A61K9/0048 , A61K38/162 , A61K48/005 , C12N7/00 , C12N15/86 , A61K48/00 , C12N2710/24022 , C12N2750/14143
摘要: Disclosed are methods and compositions for preventing, treating, and/or ameliorating one or more symptoms of inflammation in a mammal. In particular, viral vectors and medicaments containing them are disclosed, which are useful in the prophylaxis, therapy, or amelioration of symptoms of one or more inflammatory-mediated mammalian diseases, such as age-related macular degeneration (AMD), arthritis, Bechet's disease, Best macular dystrophy, corneal inflammation, diabetic retinopathy, drusen formation, dry AMD, dry eye, geographic atrophy, glaucomaocular neovascularization, Lupus erythematosus, macular degeneration, Mallatia Leventinese and Doyne honeycomb retinal dystrophy, nephritis, ocular hypertension, ocular inflammation, recurrent uveitis, Sorsby fundus dystrophy, vasculitis, vitreoretinopathy, wet AMD, or related disorders. In exemplary methods, administration of a pharmaceutical composition comprising a recombinant viral vector that delivers a secretable and cell-penetrating M013 protein or peptide to a subject in need thereof facilitated treatment of particular human disorders such as AMD, ocular neovascularization, uveitis, and related inflammatory ocular disease.
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38.
公开(公告)号:US20240093227A1
公开(公告)日:2024-03-21
申请号:US18265756
申请日:2021-12-08
发明人: Masatora FUKUDA , Makoto KOIZUMI , Shinzo IWASHITA
CPC分类号: C12N15/85 , C12N9/78 , C12N15/11 , C12Y305/04 , A61K48/00 , C12N2310/315 , C12N2310/321 , C12N2800/107
摘要: Provided is an oligonucleotide which may induce an editing activity of ADRC in cell and has excellent stability in a living body. The oligonucleotide includes a first oligonucleotide identifying a target RNA and a second oligonucleotide linked to the 5′-side of the first oligonucleotide. The first oligonucleotide consists of a target-corresponding nucleotide residue, an oligonucleotide of 10 to 24 residues at the 3′-side, and an oligonucleotide of 3 to 6 residues at the 5′-side. The second oligonucleotide has no nucleotide residue corresponding to a nucleotide residue of the target RNA or has a nucleotide residue which does not form a complementary pair at the 3′-end thereof and the number of residue is 3 to 6. The residue at the 3′-side of the target-corresponding nucleotide residue is a 2′-deoxynucleotide residue, and the third nucleotide residue counted in the 3′-direction from the target-corresponding nucleotide in the oligonucleotide at the 3′-side of the target-corresponding nucleotide residue is a 2′-deoxy-2′-fluoronucleotide residue.
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公开(公告)号:US11931402B2
公开(公告)日:2024-03-19
申请号:US17580692
申请日:2022-01-21
IPC分类号: A61K38/17 , A61K31/519 , A61K31/7048 , A61K31/713 , A61K38/00 , A61K38/45 , A61K48/00 , C12N15/113
CPC分类号: A61K38/1709 , A61K31/519 , A61K31/7048 , A61K31/713 , A61K38/005 , A61K38/45 , A61K48/00 , A61K48/005 , C12N15/1137 , C12Y207/11001 , C12N2310/14 , C12N2750/14143
摘要: The present invention provides a method of protecting the heart from damage, by administering to a patient at risk of such damage, a pharmaceutically effective amount of a composition which inhibits the interaction of RSK3 and mAKAPβ, or the expression or activity of one or both of those molecules. This composition may be in the form of a peptide that specifically inhibits mAKAPβ binding to RSK3 or in the form of an siRNA construct which inhibits the expression of RSK3.
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40.
公开(公告)号:US20240084293A1
公开(公告)日:2024-03-14
申请号:US18502499
申请日:2023-11-06
发明人: Marine Prissette , Matthew Koss , Wen Fury , Brian Zambrowicz
IPC分类号: C12N15/10 , C12N5/00 , C12N5/071 , C12N9/22 , C12N15/113 , G01N21/64 , G01N33/68 , G16B25/00
CPC分类号: C12N15/1082 , C12N5/0018 , C12N5/0686 , C12N9/22 , C12N15/1086 , C12N15/1089 , C12N15/113 , G01N21/6428 , G01N33/6896 , G16B25/00 , A61K48/00 , C12N2310/20 , C12N2800/80
摘要: Cas-protein-ready tau biosensor cells, CRISPR/Cas synergistic activation mediator (SAM)-ready tau biosensor cells, and methods of making and using such cells to screen for genetic modifiers of tau seeding or aggregation are provided. Reagents and methods for sensitizing such cells to tau seeding activity or tau aggregation or for causing tau aggregation are also provided.
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