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21.发明公开公开(公告)号：US20240342207A1
公开(公告)日：2024-10-17
申请号：US18294680
申请日：2022-08-02
发明人： Liuqing YANG , Chunru LIN , Sergey D. EGRANOV , Yajuan LI
IPC分类号： A61K31/7105 , A61K48/00 , A61P3/00 , C12N15/11
CPC分类号： A61K31/7105 , A61K48/0075 , A61P3/00 , C12N15/11 , C12N2310/315 , C12N2310/322 , C12N2310/3517
摘要： This disclosure provides modified nucleic acids and compositions thereof that are able to mimic the human HULC lncRNA and/or the mouse Pair lncRNA and enhance phenylalanine hydroxylase enzyme (PAH)-substrate binding affinity, PAH-cofactor binding affinity, and/or PAH enzymatic conversion of phenylalanine to tyrosine. Also provided are methods of using the modified nucleic acids and compositions thereof to treat subjects having or suspected of having phenylketonuria and hyperphenylalaninemia.
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公开(公告)号：US12117448B2
公开(公告)日：2024-10-15
申请号：US16867560
申请日：2020-05-05
发明人： Randolph Watnick
IPC分类号： G01N33/574 , A61K38/00 , A61K38/17 , A61K45/06 , A61K48/00 , C07K14/475
CPC分类号： G01N33/574 , A61K38/1709 , A61K45/06 , C07K14/475 , A61K38/00 , A61K48/00 , G01N2800/56
摘要： The invention relates to isolated peptides and chimeric polypeptides derived from Saposin A that have anti-angiogenic activity. These peptides are small, consisting essentially of at least 10 consecutive amino acid residues from the 31st-50th amino acid residue of Saposin A. The invention also relates to the use of these isolated peptides and chimeric polypeptides in compositions for the treatment, prevention, and inhibition of angiogenesis-related diseases and disorders such as cancer and cancer metastasis.
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公开(公告)号：US12116604B2
公开(公告)日：2024-10-15
申请号：US18049944
申请日：2022-10-26
摘要： Transposase polypeptides and polynucleotides are provided, which have a high activity in mammalian cells. Methods for engineering cells, such as chimeric antigen T-cells, with the transposes are also provided.
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公开(公告)号：US12115230B2
公开(公告)日：2024-10-15
申请号：US17918092
申请日：2021-04-09
发明人： Alexandra Chadwick , Kallanthottathil G. Rajeev , Ellen Rohde , Christopher Cheng , Caroline Reiss
IPC分类号： C12N15/11 , A61K9/127 , A61K31/7105 , A61K38/46 , A61K48/00 , C07K14/515 , C12N9/22 , C12N15/113 , C12N15/90
CPC分类号： A61K48/0066 , A61K9/127 , A61K31/7105 , A61K38/465 , C07K14/515 , C12N9/22 , C12N15/11 , C12N15/113 , C12N15/907 , C07K2319/09 , C07K2319/80 , C12N2310/20 , C12N2310/315 , C12N2310/321 , C12N2310/331 , C12N2310/335 , C12N2310/344 , C12N2310/3521 , C12N2800/80
摘要： Provided herein are compositions for gene modification or editing and methods of using same to treat or prevent certain conditions. Specific compositions and methods capable of safely and effectively editing gene targets expressed in the liver to durably lower LDL-C thereby treating a leading cause of cardiovascular disease are disclosed.
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公开(公告)号：US20240335566A1
公开(公告)日：2024-10-10
申请号：US18718023
申请日：2022-12-09
CPC分类号： A61K48/0058 , A61K38/1709 , C07K14/47 , C12N15/86 , C12N2750/14143 , C12N2830/008 , C12N2830/48 , C12N2830/50
摘要： Provided herein is a gene therapy for TNNC1 (Troponin C)-related cardiomyopathy, e.g. using an adeno-associated virus (AAV) vector. The promoter of the vector may be a MHCK7 promoter or a cardiac troponin T (hTNNT2) promoter. The capsid may be an AAV9 or AAVrh.74 capsid or a functional variant thereof. Other promoters or capsids may be used. Further provided are methods of treatment, such as by intravenous, intracoronary, intracarotid or intracardiac administration of the rAAV vector, and other compositions and methods.
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公开(公告)号：US20240335563A1
公开(公告)日：2024-10-10
申请号：US18294722
申请日：2022-08-11
CPC分类号： A61K48/0058 , A61K31/7068 , A61P3/10 , C12N9/0071 , C12N9/22 , C12N15/11 , C12N15/90 , C12Y114/11 , C12N2310/20
摘要： Disclosed herein are compositions and in vitro and in vivo methods for reducing diabetes-induced epigenetic barriers in cells to enhance the efficacy of treatments of diabetes.
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公开(公告)号：US20240335561A1
公开(公告)日：2024-10-10
申请号：US18578960
申请日：2022-07-22
CPC分类号： A61K48/005 , A61K31/395 , A61K38/193 , A61K38/42 , A61K48/0075 , A61P7/06 , C12N9/22 , C12N15/111 , C12N2310/20
摘要： Disclosures herein are directed to compositions and methods for increasing gamma globinexpression in a cell or subject by suppressing expression of Klf1. Also described are methods of treating or ameliorating-hemoglobinopathies. The compositions are administered to a subject as a therapeutic amount of a gene editing composition, including an RNA-guided endonuclease and one or more gRNA or sgRNA, or one or more transcription activator-like effector nucleases (TALENs), that targets an intron of a Klf1 gene locus in at least one cell in the subject to effect a deletion in intron, thereby reducing expression of Klf1.
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公开(公告)号：US12109275B2
公开(公告)日：2024-10-08
申请号：US16848991
申请日：2020-04-15
申请人： CureVac SE
发明人： Andreas Thess , Thomas Schlake , Jochen Probst
IPC分类号： A61K48/00 , A61K39/00 , C07K14/435 , C12N15/67
CPC分类号： A61K48/0066 , A61K39/0011 , C07K14/435 , C12N15/67 , A61K2039/53 , A61K2039/575
摘要： The present application describes a coding nucleic acid sequence, particularly a messenger RNA (mRNA), comprising or coding for a histone stem-loop and a poly(A) sequence or a polyadenylation signal and the use thereof for increasing the expression of an encoded protein. It also discloses its use for the preparation of a pharmaceutical composition, especially a vaccine e.g. for the use in the treatment of tumours and cancer diseases, cardiovascular diseases, infectious diseases, autoimmune diseases or genetic diseases, or in gene therapy. The present invention further describes an in vitro transcription method, in vitro methods for increasing the expression of a protein using the nucleic acid comprising or coding for a histone stem-loop and a poly(A) sequence or a polyadenylation signal and an ex vivo and in vivo method.
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公开(公告)号：US12109251B2
公开(公告)日：2024-10-08
申请号：US17366825
申请日：2021-07-02
发明人： Sergei Zolotukhin , Andres Acosta
IPC分类号： A61K38/22 , A61K9/00 , A61K9/68 , A61K48/00 , C07K14/575
CPC分类号： A61K38/22 , A61K9/0019 , A61K9/0056 , A61K9/0058 , A61K9/006 , A61K48/005 , A61K48/0075 , C07K14/575 , C12N2799/025
摘要： Disclosed herein are compositions and methods for treating obesity involving satiation gut peptide administration to the mouth of a subject for a predetermined dose and frequency. In other embodiments, materials and methods of treating certain psychological disorders are disclosed involving satiation gut peptides. In exemplary embodiments, the satiation gut peptide pertains to PYY.
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30.发明公开公开(公告)号：US20240325568A1
公开(公告)日：2024-10-03
申请号：US18580716
申请日：2022-07-20
发明人： Benjamin Deverman , Qin Huang , Ken Chan
IPC分类号： A61K48/00 , A61K31/7105 , A61K38/46 , A61K47/64 , C12N15/86
CPC分类号： A61K48/0033 , A61K31/7105 , A61K38/465 , A61K47/64 , C12N15/86 , C12N2750/14122 , C12N2750/14143 , C12N2750/14145
摘要： Highly selective targeting moieties and compositions comprising the targeting moieties are described herein to efficiently transduce endothelial cell of the central nervous system vasculature. Embodiments include use and delivery of the targeting moieties and compositions to selectively direct delivery of cargo.
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