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1. 发明公开

US20240197860A1 SELF-REPLICATING RNA AND USES THEREOF 审中-公开
公开(公告)号：US20240197860A1
公开(公告)日：2024-06-20
申请号：US18512730
申请日：2023-11-17
申请人： Trustees of Boston University
发明人： Joshua Edward MCGEE , Jack Rainier KIRSCH , Eric BRESSLER , Mark W. GRINSTAFF , Wilson WONG , Lidya Yidnekachew Sertse , Kexin Li
IPC分类号： A61K39/215 , A61K39/00 , A61P37/04 , C07K14/005 , C07K14/575 , C07K16/28 , C07K16/32 , C12N15/86
CPC分类号： A61K39/215 , A61K39/4631 , A61P37/04 , C07K14/005 , C07K14/575 , C07K16/2803 , C07K16/32 , C12N15/86 , A61K2039/53 , A61K2239/23 , C12N2770/20022 , C12N2770/36143 , C12N2830/002 , C12N2830/50 , C12N2840/203
摘要： The technology described herein is directed to compositions and methods for modifying and controlling the activity of cells by expression of proteins from self-amplifying RNA (saRNA). Also described herein are compositions and methods for modifying and controlling the activity of cells by expression of proteins from self-amplifying RNA that is substituted with chemically modified nucleotides.

2. 发明公开

US20240102001A1 COUPLING ENDONUCLEASES WITH END-PROCESSING ENZYMES DRIVES HIGH EFFICIENCY GENE DISRUPTION 审中-公开
公开(公告)号：US20240102001A1
公开(公告)日：2024-03-28
申请号：US18526930
申请日：2023-12-01
申请人： SEATTLE CHILDREN'S HOSPITAL (dba SEATTLE CHILDREN'S RESEARCH INSTITUTE)
发明人： Andrew M. SCHARENBERG , Michael T. CERTO , Kamila Sabina GWIAZDA
IPC分类号： C12N15/10 , A61K9/00 , A61K35/28 , A61K38/45 , A61K38/46 , A61K38/52 , C12N9/12 , C12N9/22 , C12N9/90 , C12N15/62
CPC分类号： C12N15/102 , A61K9/0019 , A61K35/28 , A61K38/45 , A61K38/465 , A61K38/52 , C12N9/1252 , C12N9/22 , C12N9/90 , C12N15/62 , A61K2035/124 , C07K2319/60 , C12N2800/80 , C12N2840/203
摘要： The present disclosure relates to the co-expression of an endonuclease with an end-processing enzyme for the purpose of enhanced processing of the polynucleotide ends generated by endonuclease cleavage.

3. 发明公开

US20240093202A1 Suppression-Replacement Gene Therapy 审中-公开
公开(公告)号：US20240093202A1
公开(公告)日：2024-03-21
申请号：US18270014
申请日：2021-12-30
申请人： Mayo Foundation for Medical Education and Research
发明人： Michael J. ACKERMAN , Steven M. DOTZLER , William GENDRON , Sahej BAINS , Changsung John KIM , David J. TESTER
IPC分类号： C12N15/113 , A61K38/17 , A61K48/00 , A61P9/00 , C07K14/705 , C12N15/86
CPC分类号： C12N15/1138 , A61K38/177 , A61K48/005 , A61P9/00 , C07K14/705 , C12N15/86 , C12N2310/14 , C12N2320/34 , C12N2740/16043 , C12N2750/14143 , C12N2840/203
摘要： Methods and materials for treating a mammal having a congenital disease (e.g., a congenital heart disease such as congenital long QT syndrome) are provided herein. For example, this document provides methods and materials for generating and using nucleic acids to treat a mammal having a congenital disease, where the nucleic acids can suppress expression of mutant disease-related alleles in the mammal while providing a replacement cDNA that does not contain the disease-related mutation(s).

4. 发明公开

US20230340536A1 METHODS AND COMPOSITIONS FOR TRANSDUCING LYMPHOCYTES AND REGULATING THE ACTIVITY THEREOF 审中-公开
公开(公告)号：US20230340536A1
公开(公告)日：2023-10-26
申请号：US18322531
申请日：2023-05-23
申请人： Exuma Biotech Corp.
发明人： Gregory Ian FROST , James Joseph ONUFFER, Jr. , Ghiabe H. Guibinga , Farzad HAERIZADEH , Anirban KUNDU
IPC分类号： C12N15/86 , A61K39/00 , C12N5/0783 , C07K14/005 , C07K14/715 , C07K14/705 , C07K16/30 , C07K14/725 , C12N15/113 , C12N15/10 , C07K16/32 , C07K14/54 , C07K16/28
CPC分类号： C12N15/86 , A61K39/0011 , C12N5/0646 , C07K14/005 , C07K14/7155 , C07K14/70596 , C07K16/30 , C07K14/7051 , C07K14/70578 , C07K14/70517 , C12N15/1138 , C12N15/1048 , C07K16/32 , C07K14/5418 , C07K16/2809 , C07K14/70521 , C12N5/0636 , C12N15/113 , C07K16/2803 , C07K16/2818 , C07K16/2863 , C07K16/283 , C12N2310/141 , C12N2760/18422 , C12N2740/10045 , C12N2840/203 , C12N2740/10043 , C12N2310/12 , C07K2319/02 , C12N2740/16043 , C12N2830/008 , C07K2319/31 , C12N2310/531 , C12N2510/00 , C12N2310/121 , C07K2319/33 , A61K35/17
摘要： The present disclosure provides methods for genetically modifying lymphocytes and methods for performing adoptive cellular therapy that include transducing T cells and/or NK cells. The methods can include inhibitory RNA molecule(s) and/or engineered signaling polypeptides that can include a lymphoproliferative element, and/or a chimeric antigen receptor (CAR), for example a microenvironment restricted biologic CAR (MRB-CAR). Additional elements of such engineered signaling polypeptides are provided herein, such as those that drive proliferation and regulatory elements therefor, as well as replication incompetent recombinant retroviral particles and packaging cell lines and methods of making the same. Numerous elements and methods for regulating transduced and/or genetically modified T cells and/or NK cells are provided, such as, for example, those including riboswitches, MRB-CARs, recognition domains, and/or pH-modulating agents.

5. 发明公开

US20230310654A1 GENE THERAPIES FOR LYSOSOMAL DISORDERS 审中-公开
公开(公告)号：US20230310654A1
公开(公告)日：2023-10-05
申请号：US18020575
申请日：2021-08-10
申请人： Prevail Therapeutics, Inc.
发明人： Asa ABELIOVICH , Jeffrey SEVIGNY , Travis LEWIS , Olga USPENSKAYA
IPC分类号： A61K48/00 , C12N15/113 , A61K9/00 , A61P25/16 , A61K31/573 , A61K39/395 , A61K31/436 , A61P37/06 , C12N15/86 , C12N9/24
CPC分类号： A61K48/005 , C12N15/113 , A61K9/0019 , A61P25/16 , A61K31/573 , A61K39/3955 , A61K31/436 , A61P37/06 , C12N15/86 , C12Y302/01045 , C12N9/2402 , C12N2750/14143 , C12N2840/203 , C12N2310/11
摘要： The disclosure relates to compositions and methods for treatment of diseases associated with aberrant lysosomal function, such as Parkinson's disease and Gaucher disease. The disclosure provides methods of treating Gaucher disease, Parkinson's disease or other synucleinopathies by administering expression constructs comprising a transgene encoding beta-glucocerebrosidase, an inhibitory RNA targeting alpha-Synuclein, or a combination of the foregoing to a subject in need thereof.

6. 发明公开

US20230295651A1 RECOMBINANT NUCLEIC ACID CONSTRUCT AND USE THEREOF 审中-公开
公开(公告)号：US20230295651A1
公开(公告)日：2023-09-21
申请号：US17885088
申请日：2022-08-10
申请人： Pantherna Therapeutics GmbH
发明人： Joerg KAUFMANN , Oliver KEIL , Daniel TONDERA
IPC分类号： C12N15/85 , A61K9/51 , C07K14/47 , C12N9/12
CPC分类号： C12N15/85 , A61K9/5123 , C07K14/4705 , C12N9/12 , A61K48/00 , C12N2800/107 , C12N2830/50 , C12N2840/203
摘要： The present invention is related to a recombinant nucleic acid construct comprising in 5′-> 3′ direction

a 5′ UTR,
a coding region coding for an effector molecule, and
a 3′ UTR,

wherein the 5′ UTR is selected from the group consisting of a 5′ UTR of a gene coding for MCP-1 or a derivative thereof having a nucleotide identity of at least 85%, a 5′ UTR of a gene coding for RPL12s.c. or a derivative thereof having a nucleotide identity of at least 85%, a 5′ UTR of a gene coding for Ang-2 or a derivative thereof having a nucleotide identity of at least 85%, a 5′ UTR of a gene coding for HSP70 or a derivative thereof having a nucleotide identity of at least 85%, a 5′ UTR of a gene coding for H3.3. or a derivative thereof having a nucleotide identity of at least 85%, a 5′ UTR of a gene coding for Galectin-9 or a derivative thereof having a nucleotide identity of at least 85%, a 5′ UTR of a gene coding for GADD34 or a derivative thereof having a nucleotide identity of at least 85%, a 5′ UTR of a gene coding for EDN1 or a derivative thereof having a nucleotide identity of at least 85%, a 5′ UTR of a gene coding for HSP70m5 or a derivative thereof having a nucleotide identity of at least 85%, a 5′ UTR of a gene coding for E-selectin or a derivative thereof having a nucleotide identity of at least 85% a 5′ UTR of a gene coding for ICAM-1 or a derivative thereof having a nucleotide identity of at least 85%, a 5′ UTR of a gene coding for IL-6 or a derivative thereof having a nucleotide identity of at least 85% and a 5′ UTR of a gene coding for vWF or a derivative thereof having a nucleotide identity of at least 85 %;
wherein 3′ UTR is selected from the group consisting of a 3′ UTR of a gene coding for vWF or a derivative thereof having a nucleotide identity of at least 85%, a 3′ UTR of a gene coding for MCP-1 or a derivative thereof having a nucleotide identity of at least 85%,, a 3′ UTR of a gene coding for RPL12s.c. or a derivative thereof having a nucleotide identity of at least 85%, a 3′ UTR of a gene coding for HSP70 or a derivative thereof having a nucleotide identity of at least 85%, a 3′ UTR of a gene coding for H3.3. or a derivative thereof having a nucleotide identity of at least 85%, a 3′ UTR of a gene coding for GADD34 or a derivative thereof having a nucleotide identity of at least 85%, a 3′ UTR of a gene coding for EDN1 or a derivative thereof having a nucleotide identity of at least 85%, and a 3′ UTR of a gene coding for IL-6 or a derivative thereof having a nucleotide identity of at least 85%,
wherein the effector molecule is effective in restoring a cellular function of a cell or is effective in exercising a therapeutic effect in or on a cell, and
wherein the recombinant nucleic acid construct is different from a wild type mRNA coding for the effector molecule.

7. 发明公开

US20230272419A1 CANINE AND FELINE INDUCIBLE EXPRESSION CONSTRUCTS FOR GENE THERAPY APPLICATIONS 审中-公开
公开(公告)号：US20230272419A1
公开(公告)日：2023-08-31
申请号：US18006900
申请日：2021-07-26
申请人： The Trustees of the University of Pennsylvania
发明人： James M. Wilson , Christian Hinderer , Makoto Horiuchi
IPC分类号： C12N15/86 , C07K14/47
CPC分类号： C12N15/86 , C07K14/4702 , C07K2319/81 , C12N2750/14143 , C07K2319/71 , C07K2319/33 , C12N2840/203
摘要： Provided herein are nucleic acid molecules, vectors, and recombinant AAV comprising an inducible gene expression system. The system includes a transgene encoding a gene product operably linked to expression control sequences comprising a promoter; an activation domain comprising a canine or feline transactivation domain and a FKBP12-rapamycin binding (FRB) domain of canine or feline FKBP12-rapamycin-associated protein (FRAP); a DNA binding domain comprising a zinc finger homeodomain (ZFHD) and one, two or three FK506 binding protein domain (FKBP) subunit genes; and at least 8 copies of the binding site for ZFHD (8XZFHD) followed by a minimal IL2 promoter. The presence of an effective amount of a rapamycin or a rapalog induces expression of the transgene in a host cell.

8. 发明公开

US20230212635A1 METHODS OF PRODUCING RECOMBINANT COMPLEMENT PROTEINS, VECTORS AND THERAPEUTIC USES THEREOF 审中-公开
公开(公告)号：US20230212635A1
公开(公告)日：2023-07-06
申请号：US17928798
申请日：2021-11-19
申请人： Gyroscope Therapeutics Limited
发明人： Kevin Marchbank , David Kavanagh , Claire Harris , Thomas Cox
IPC分类号： C12P21/02 , C12N15/86 , C12N9/50 , C12N5/071
CPC分类号： C12P21/02 , C12N15/86 , C12N9/50 , C12Y304/21075 , C12N5/0687 , C12N2840/203 , C12N2750/14143
摘要： Aspects of the present invention relate to the recombinant production of a mature complement system protein. Certain embodiments of the present invention relate to recombinant production of fully mature human complement Factor I protein (CFI). Included herein are details of an expression vector with which to recombinantly express fully mature human CFI from mammalian cells. Further disclosed are chromatography steps with which to purify recombinantly expressed CFI. Certain aspects of the present invention relate to the use of an expression system in gene therapy and the like. Certain embodiments of the present invention relate to use of said vector as a medicament, for example for use in the treatment of complement-mediated disorders.

9. 发明授权

US11692193B2 Expression from transposon-based vectors and uses 有权
公开(公告)号：US11692193B2
公开(公告)日：2023-07-04
申请号：US17190954
申请日：2021-03-03
申请人： Just-Evotec Biologics, Inc.
发明人： Jeffrey T. McGrew , Pauline S. Smidt , E-Ching Ong
IPC分类号： C12N15/00 , C12N15/52 , C12N15/65 , C12N15/113 , C12N15/85 , C12N15/67
CPC分类号： C12N15/52 , C12N15/113 , C12N15/65 , C12N15/67 , C12N15/85 , C12N2015/8518 , C12N2710/22031 , C12N2800/107 , C12N2800/90 , C12N2830/001 , C12N2830/003 , C12N2830/60 , C12N2840/203
摘要： Recombinant expression vectors are disclosed that include a control sequence for recombinant expression of proteins of interest; the control sequence combines a mCMV enhancer sequence with a rat EF-1alpha intron sequence. Some of the vectors are useful for tetracycline-inducible expression. Some of the vectors contain a 5′ PiggyBac ITR and a 3′ PiggyBac ITR to promote genomic integration into a host cell chromosome. A method of selecting a stable production cell line for manufacturing a protein of interest is also disclosed. Also disclosed are mammalian host cells comprising the inventive recombinant expression vectors and a method of producing a protein of interest, in vitro, involving the mammalian host cell.

10. 发明公开

US20230193311A1 IMMUNOGENIC COMPOSITIONS AND USES THEREOF 审中-公开
公开(公告)号：US20230193311A1
公开(公告)日：2023-06-22
申请号：US17925966
申请日：2021-05-20
申请人： Flagship Pioneering Innovations VI, LLC
发明人： Avak KAHVEJIAN , Alexandra Sophie DE BOER , Yann Paul Guy Régis ECHELARD , Nicholas McCartney PLUGIS , Erica Gabrielle WEINSTEIN , Jennifer A. NELSON
IPC分类号： C12N15/85 , A61P37/04 , A61K39/215 , A61P31/14 , A61K39/145 , A61P31/16 , A61K39/12 , A61P31/20 , A61K39/00 , A61P35/00 , A61K39/39
CPC分类号： C12N15/85 , A61P37/04 , A61K39/215 , A61P31/14 , A61K39/145 , A61P31/16 , A61K39/12 , A61P31/20 , A61K39/0011 , A61P35/00 , A61K39/39 , C12N2840/203 , C12N2760/16122 , C12N2760/16134 , C12N2760/16171 , C12N2770/20022 , C12N2770/20034 , C12N2770/20071 , C12N2710/20022 , C12N2710/20034 , A61K2039/55505 , A61K2039/53 , A61K2039/70 , A61K2039/55555
摘要： This disclosure provides compositions, pharmaceutical preparations, and uses of polyribonucleotides encoding one or more immunogenic polypeptides. In particular, this disclosure features circular polyribonucleotide encoding one or more immunogenic polypeptides.

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