公开(公告)号：US20230405085A1

公开(公告)日：2023-12-21

申请号：US18313124

申请日：2023-05-05

申请人： INSTITUT PASTEUR ,  Centre National de la Recherche Scientifique (CNRS) ,  Sorbonne Universite ,  Universite Clermont Auvergne

发明人： Christine PETIT ,  Paul AVAN ,  Sedigheh DELMAGHANI ,  Jean DEFOURNY ,  Asadollah AGHAIE ,  Saaid SAFIEDDINE ,  Alice EMPTOZ

IPC分类号： A61K38/17 ,  A61K8/64 ,  A61K9/00 ,  A61Q19/02 ,  A61Q19/08 ,  A61K48/00 ,  A61K35/761 ,  C12N15/86 ,  C12N7/00 ,  C12N15/113

CPC分类号： A61K38/1709 ,  A61K8/64 ,  A61K9/0046 ,  A61Q19/02 ,  A61Q19/08 ,  A61K48/00 ,  A61K35/761 ,  A61K38/17 ,  A61K48/005 ,  C12N15/86 ,  A61K48/0075 ,  A61K9/0019 ,  C12N7/00 ,  C12N15/113 ,  A61K2800/91 ,  A01K2217/075 ,  A01K2267/0306 ,  C12N2750/14143 ,  A01K2227/105 ,  C12N2310/14 ,  C12N2310/531 ,  C12N2710/10021 ,  C12N2710/10043

摘要： The present invention relates to the use of gasdermin, in particular of gasdermin A, gasdermin B, gasdermin C, gas-dermin D, DFNA5 or DFNB59 (or pejvakin), and more particularly pejvakin for modulating cellular redox homeostasis. A particularly preferred use of gasdermin, in particular of gasdermin A, gasdermin B, gasdermin C, gasdennin D, DFNA5 or DFNB59 (or pejvakin), and more particularly pejvakin in the context of the present invention is as an antioxidant. The present invention also concerns a virally-mediated gene therapy for restoring genetically-impaired auditory and vestibular functions in subjects suffering from an Usher syndrome. More precisely, this gene therapy takes advantage of an AA V2/8 vector expressing at least one USH1 gene product, preferably SANS.

公开(公告)号：US20230392149A1

公开(公告)日：2023-12-07

申请号：US18169779

申请日：2023-02-15

申请人： Genzyme Corporation

发明人： Catherine R. O'RIORDAN ,  Adam Palermo ,  Brenda Richards ,  Lisa M. Stanek

IPC分类号： C12N15/113 ,  A61P25/28 ,  A61K35/761 ,  C12N7/00 ,  C12N15/86

CPC分类号： C12N15/113 ,  A61P25/28 ,  A61K35/761 ,  C12N7/00 ,  C12N15/86 ,  A61K48/00

摘要： Provided herein are RNAi molecules for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, 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 Huntington's disease.

公开(公告)号：US11827906B2

公开(公告)日：2023-11-28

申请号：US16487674

申请日：2018-02-27

申请人： The Trustees of the University of Pennsylvania

发明人： James M. Wilson ,  Qiang Wang ,  April Tepe ,  Kevin Turner

IPC分类号： C12N7/00 ,  C07K14/005 ,  C07K16/32 ,  A61K35/761

CPC分类号： C12N7/00 ,  A61K35/761 ,  C07K14/005 ,  C07K16/32 ,  C12N2750/14121 ,  C12N2750/14122 ,  C12N2750/14143

摘要： A recombinant adeno-associated virus (rAAV) vector comprising an AAVhu68 capsid produced in a production system comprising a nucleotide sequence of SEQ ID NO: 1, or a sequence at least 75% identical thereto which encodes SEQ ID NO:2. The AAVhu68 capsid comprises subpopulations of highly deamidated asparagine residues in asparagine-glycine pairs in the amino acid sequence of SEQ ID NO: 2. Also provided are compositions containing the rAAV and uses thereof. Additionally, rAAV having an engineered AAV capsid comprising at least one subpopulation of vp1 or vp2 proteins having a Val at amino acid position 157 with reference to the AAVhu68 vp1 numbering are provided.

公开(公告)号：US20230374545A1

公开(公告)日：2023-11-23

申请号：US18150477

申请日：2023-01-05

申请人： University of Massachusetts ,  Sichuan University

发明人： Guangping Gao ,  Guangchao Xu ,  Phillip Tai ,  Yuquan Wei ,  Li Luo

IPC分类号： C12N15/86 ,  C12N15/861 ,  C07K14/005 ,  C07K14/015 ,  A61K35/761 ,  A01K67/027 ,  A61K48/00

CPC分类号： C12N15/86 ,  C12N15/861 ,  C07K14/005 ,  C07K14/015 ,  A61K35/761 ,  A01K67/0275 ,  C12N2750/14122 ,  C12N2750/14123 ,  A01K2267/03 ,  A01K2217/05 ,  A61K48/00 ,  C12N2750/14143 ,  C12N2750/14145

摘要： The disclosure in some aspects relates to recombinant adeno-associated viruses having distinct tissue targeting capabilities. In some aspects, the disclosure relates to gene transfer methods using the recombinant adeno-associated viruses. In some aspects, the disclosure relates to isolated AAV capsid proteins and isolated nucleic acids encoding the same.

公开(公告)号：US20230340529A1

公开(公告)日：2023-10-26

申请号：US18024359

申请日：2021-09-02

申请人： University of Massachusetts

发明人： Guangping Gao ,  Philip Tai ,  Claudio Punzo ,  Haijiang Lin

IPC分类号： C12N15/86 ,  A61K35/761

CPC分类号： C12N15/86 ,  A61K35/761 ,  C12N2750/14143 ,  C12N2750/14171 ,  C12N2750/14122

摘要： Aspects of the disclosure relate to compositions and methods for expressing anti-Vascular endothelial cell growth factor (VEGF) agent in a cell or subject. In some embodiments, the disclosure provides rAAVs comprising a capsid protein (e.g., AAV2 variants, AAV2/3 hybrid variants, AAV8 variants, etc.), and a transgene encoding an anti-VEGF agent (e.g., KH902) and one or more regulatory sequences. In some embodiments, compositions described herein are useful for treating subjects having diseases associated with angiogenesis or aberrant VEGF activity/signaling.

公开(公告)号：US11744894B2

公开(公告)日：2023-09-05

申请号：US16960054

申请日：2019-01-15

申请人： NANKAI UNIVERSITY

发明人： Yao Chen ,  Zhenjie Zhang ,  Yifan Feng

IPC分类号： A61K47/24 ,  A61K35/761 ,  A61K39/395 ,  A61K47/02

CPC分类号： A61K47/24 ,  A61K35/761 ,  A61K39/3955 ,  A61K47/02

摘要： A novel composite biological agent based on a porous frame material, comprising porous frame materials and biomolecules. The porous frame materials cover a biological product, wherein the porous frame materials are metal-organic frame material (MOFs), covalent organic frame materials (COFs), and hydrogen-bonding organic frame materials (HOFs), and the biomolecules are any one or a combination of antibodies, enzymes, peptides, vaccines, nucleotides, and virus species. The composite biological agent uses the porous frame materials and biomolecules to form a porous frame material/biomolecule complex, and the biomolecules are coated to achieve the protection effect. Under the premise of remaining biomolecule activity, the system can achieve efficient separation and recovery of the porous materials and the biomolecules, so that the technical problems of synthesis, storage, release, etc. are solved, a good technical effect is achieved, and the biomolecules are effectively protected. The system is applied to the storage and transportation of biological agents and preparation of novel agents.

公开(公告)号：US20230250411A1

公开(公告)日：2023-08-10

申请号：US18161467

申请日：2023-01-30

申请人： MIAMI UNIVERSITY

发明人： PAUL A. HARDING ,  KAYLA CARTWRIGHT ,  SEAN R. TAYLOR ,  ABBY SFERRELLA

IPC分类号： C12N9/64 ,  C12N15/86 ,  C12N5/077 ,  A61K35/761

CPC分类号： C12N9/6416 ,  C12N15/86 ,  C12N5/0653 ,  A61K35/761 ,  C12N2710/10041

摘要： Obesity and related conditions and diseases pose ever increasing issues for the developed world. There is a need for interventions that can treat/prevent the underlying causes of e.g., diabetes and obesity. Accordingly compositions and methods for altering at least one expression level related to cellular metabolism are disclosed herein.

公开(公告)号：US20230220355A1

公开(公告)日：2023-07-13

申请号：US18151093

申请日：2023-01-06

申请人： Salk Institute for Biological Studies

发明人： William Partlo ,  Clodagh O'Shea

IPC分类号： C12N7/00 ,  C07K14/005 ,  A61P35/00 ,  A61K35/761 ,  A61K31/65

CPC分类号： C12N7/00 ,  C07K14/005 ,  A61P35/00 ,  A61K35/761 ,  A61K31/65 ,  C12N2710/10021 ,  C12N2710/10071 ,  C12N2710/10032 ,  C12N2710/10022

摘要： Recombinant adenovirus genomes that include a synthetic transcriptional circuit are described. Synthetic adenoviruses positively regulated using two-step transcriptional amplification (TSTA) are further described. Selection of the heterologous promoter is based on the desired replication characteristics of the synthetic virus. For example, the heterologous promoter can be a constitutive promoter, a tumor-specific promoter or a tissue-specific promoter.

公开(公告)号：US11690913B2

公开(公告)日：2023-07-04

申请号：US17141463

申请日：2021-01-05

申请人： TARGOVAX OY

发明人： Tuuli Ranki ,  Sari Anneli Pesonen ,  Magnus Jäderberg ,  Elina Haavisto ,  Lukasz Kuryk ,  Antti Vuolanto

IPC分类号： A61K35/761 ,  A61K39/395 ,  A61K39/00 ,  A61P35/00 ,  C07K16/28 ,  C07K14/535

CPC分类号： A61K39/3955 ,  A61K35/761 ,  A61P35/00 ,  C07K14/535 ,  C07K16/2818 ,  C07K16/2827 ,  A61K2039/505 ,  A61K2039/545 ,  A61K39/3955 ,  A61K2300/00

摘要： The invention relates to the combination therapy comprising oncolytic adenovirus vector and a checkpoint inhibitor or checkpoint inhibitors. More specifically, the invention relates to oncolytic adenovirus vector and checkpoint inhibitor or checkpoint inhibitors for use in a cancer therapy.

公开(公告)号：US20190247452A1

公开(公告)日：2019-08-15

申请号：US15771110

申请日：2016-10-28

申请人： Board of Supervisors of Louisiana State University and Agricultural and Mechanical College

发明人： Michael Lan ,  Mary Breslin

IPC分类号： A61K35/761 ,  C12N15/86 ,  A61P35/00 ,  C12Q1/6886

CPC分类号： A61K35/761 ,  A61K49/0097 ,  A61P35/00 ,  C12N15/86 ,  C12N2710/10332 ,  C12N2710/10343 ,  C12N2840/007 ,  C12Q1/6886 ,  C12Q2600/158 ,  G01N33/50

摘要： A conditionally replicating adenovirus were generated that can specifically replicate and express therapeutic genes in neuroendocrine tumors. The promoter-specific expression of the adenoviruses is regulated upstream by an INSM1 (insulinoma-associated-1) promoter that is silent in normal adult tissues but active in developing neuroendocrine cells and neuroendocrine tumors. By placing the INSM1-promoter with an insulator and two copies of neuronal restrictive silencer elements in an adenoviral vector, the construct can retain tumor specificity and drive expression of a mutated adenovirus E1A gene (424E1A) and the herpes simplex virus thymidine kinase gene. The INSM1-promoter-driven viruses could replicate specifically in the INSM1-positive cells and I NSM1-specific HSV-tk expression in combination with ganciclovir treatment displayed dose-dependent tumor cell-specific killing in insulinomas. When the INSM1-promoter driven HSV-tk was combined with 424E1A and INSM 1 p-HSV-tk viruses, the co-infected insulinoma expressed higher levels of HSV-tk and more efficient tumor suppression as compared to the INSM1 p-HSV-tk virus alone.