公开(公告)号：US20240325571A1

公开(公告)日：2024-10-03

申请号：US18739865

申请日：2024-06-11

申请人： Christopher Key

发明人： Christopher Key

IPC分类号： A61K48/00 ,  A61K35/76 ,  A61K35/761 ,  A61K38/17

CPC分类号： A61K48/0066 ,  A61K35/76 ,  A61K35/761 ,  A61K38/177

摘要： Compositions and methods are provided that mitigate iron imbalance resulting from inflammation and/or mitigate the effects of inflammatory disease by correcting dysregulation of iron internalization. Such correction is provided by modulating cellular content of TFR2, and/or hepcidin activity in an individual in need of treatment. TRF2 content can be modulated by correcting a mutation in an endogenous TRF2 gene of the individual.

公开(公告)号：US20240247061A1

公开(公告)日：2024-07-25

申请号：US18520030

申请日：2023-11-27

申请人： AKAMIS BIO LIMITED

发明人： Brian Robert CHAMPION ,  Alice Claire Noel BROWN

IPC分类号： C07K16/28 ,  A61K35/761 ,  A61K35/768 ,  A61K38/17 ,  A61K39/00 ,  A61P35/00 ,  C07K14/00 ,  C07K14/005 ,  C07K14/725 ,  C07K16/30 ,  C12N7/00 ,  C12N15/86

CPC分类号： C07K16/2809 ,  A61K35/761 ,  A61K35/768 ,  A61K38/1774 ,  A61P35/00 ,  C07K14/00 ,  C07K14/005 ,  C07K14/7051 ,  C07K16/2803 ,  C07K16/30 ,  C12N7/00 ,  C12N15/86 ,  A61K2039/505 ,  C07K2317/622 ,  C07K2317/73 ,  C12N2710/10032 ,  C12N2710/10041

摘要： The present disclosure relates to a replication deficient oncolytic viral vector or replication capable group B oncolytic adenovirus selected from the group consisting of Ad11 and enadenotucirev, wherein the virus encodes an antibody or a binding fragment thereof for expression on the surface of a cancer cell, wherein said antibody or binding fragment is specific to a CD3 protein of a T-cell receptor complex (TCR), wherein the virus does not encode a B7 protein or an active fragment thereof, pharmaceutical compositions comprising the same, and use of any one of the same in treatment, particularly in the treatment of cancer.

公开(公告)号：US12011489B2

公开(公告)日：2024-06-18

申请号：US17708911

申请日：2022-03-30

申请人： Christopher Key

发明人： Christopher Key

IPC分类号： A61K48/00 ,  A61K35/76 ,  A61K35/761 ,  A61K38/00 ,  A61K38/17

CPC分类号： A61K48/0066 ,  A61K35/76 ,  A61K35/761 ,  A61K38/177

摘要： Compositions and methods are provided that mitigate iron imbalance resulting from inflammation and/or mitigate the effects of inflammatory disease by correcting dysregulation of iron internalization. Such correction is provided by modulating expression of TFR2, transferrin, and/or hepcidin activity in an individual in need of treatment.

公开(公告)号：US20240150792A1

公开(公告)日：2024-05-09

申请号：US18547139

申请日：2022-02-24

申请人： NorthShore University HealthSystem

发明人： Prem Seth

IPC分类号： C12N15/86 ,  A61K35/761 ,  A61K45/06 ,  A61P35/00 ,  C07K14/535 ,  C07K14/71

CPC分类号： C12N15/86 ,  A61K35/761 ,  A61K45/06 ,  A61P35/00 ,  C07K14/535 ,  C07K14/71 ,  C07K2319/30 ,  C12N2710/10032 ,  C12N2710/10041 ,  C12N2810/6018 ,  C12N2820/007

摘要： The present disclosure provides an oncolytic virus comprising a nucleic acid(s) encoding a granulocyte-macrophage colony-stimulating factor (GM-CSF) and a soluble form of the TGF-B receptor-II (sTGF-BRII). The present disclosure also provides compositions comprising the oncolytic virus and treatment methods using the oncolytic virus. The treatment methods include local and/or systemic administration of the oncolytic virus for treating cancers, such as solid tumors.

公开(公告)号：US20240141386A1

公开(公告)日：2024-05-02

申请号：US18161806

申请日：2023-01-30

申请人： EDITAS MEDICINE, INC.

发明人： Morgan Lee MAEDER ,  Rina J. MEPANI ,  Michael STEFANIDAKIS

IPC分类号： C12N15/90 ,  A61K9/00 ,  A61K35/761 ,  C12N9/22 ,  C12N15/11 ,  C12N15/113 ,  C12N15/86

CPC分类号： C12N15/907 ,  A61K9/0048 ,  A61K35/761 ,  C12N9/22 ,  C12N15/11 ,  C12N15/113 ,  C12N15/86 ,  C12N2310/20 ,  C12N2320/32 ,  C12N2330/50 ,  C12N2330/51 ,  C12N2750/14143 ,  C12N2800/80

摘要： Nucleic acids and viral vectors, particularly adeno-associated virus (AAV) vectors are provided that encode Cas9 and paired guide RNAs. The nucleic acids and vectors, and compositions that comprise them, can be used in methods to treat subjects, to alter cells in subjects who may suffer from an inherited retinal dystrophy such as CEP290 associated disease or who may be in need of alteration of a cell or a cellular nucleic acid sequence associated with an inherited retinal dystrophy such as the CEP290 gene, and/or to treat inherited retinal dystrophies including CEP290 associated disease.

公开(公告)号：US20240123003A1

公开(公告)日：2024-04-18

申请号：US18392002

申请日：2023-12-21

申请人： Prevail Therapeutics, Inc.

发明人： Asa ABELIOVICH ,  Laura HECKMAN ,  Herve RHINN ,  Franz HEFTI

IPC分类号： A61K35/761 ,  A61K9/00 ,  A61K38/16 ,  A61P25/28 ,  A61P43/00 ,  C12N15/86

CPC分类号： A61K35/761 ,  A61K9/0085 ,  A61K38/162 ,  A61P25/28 ,  A61P43/00 ,  C12N15/86 ,  A61K9/0019 ,  C12Y302/01045

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 expression constructs comprising a transgene encoding beta-glucocerebrosidase, an inhibitory RNA targeting alpha-Synuclein, or a combination of the foregoing. The disclosure further provides methods of treating Gaucher disease, Parkinson's disease or other synucleinopathies by administering such expression constructs to a subject in need thereof.

公开(公告)号：US20240082330A1

公开(公告)日：2024-03-14

申请号：US18389536

申请日：2023-11-14

申请人： Mayo Foundation for Medical Education and Research

发明人： Kah-Whye Peng ,  Stephen James Russell ,  Mitesh J. Borad ,  Yumei Zhou ,  Amber C. Miller

IPC分类号： A61K35/768 ,  C12N7/00 ,  G01N33/574

CPC分类号： A61K35/768 ,  C12N7/00 ,  G01N33/57438 ,  A61K35/761

摘要： This document relates to methods and materials involved in treating cancer. For example, methods and materials for using one or more oncolytic viruses (e.g., one or more replicating oncolytic viruses) to treat cancer in a mammal (e.g., a human) identified as having a cancer likely to respond to oncolytic virotherapy are provided.

公开(公告)号：US20240067985A1

公开(公告)日：2024-02-29

申请号：US18152699

申请日：2023-01-10

申请人： Gritstone bio, Inc.

发明人： Wade Blair ,  Brendan Bulik-Sullivan ,  Jennifer Busby ,  Adnan Derti ,  Leonid Gitlin ,  Karin Jooss ,  Ciaran Daniel Scallan ,  Roman Yelensky ,  Gijsbert Grotenbreg

IPC分类号： C12N15/86 ,  A61K35/761 ,  A61K39/00 ,  A61K39/395 ,  A61P35/00 ,  C07K14/74 ,  C07K16/28 ,  C12N7/00

CPC分类号： C12N15/86 ,  A61K35/761 ,  A61K39/0011 ,  A61K39/39541 ,  A61P35/00 ,  C07K14/70539 ,  C07K16/2818 ,  C12N7/00 ,  A61K2039/505 ,  A61K2039/5256 ,  C12N2710/10034 ,  C12N2710/10043 ,  C12N2710/10071

摘要： Disclosed herein are chimpanzee adenoviral vectors that include neoantigen-encoding nucleic acid sequences derived from a tumor of a subject. Also disclosed are nucleotides, cells, and methods associated with the vectors including their use as vaccines.

公开(公告)号：US20240024437A1

公开(公告)日：2024-01-25

申请号：US18147627

申请日：2022-12-28

申请人： Transgene SA

发明人： Philippe SLOS ,  Julie Hortelano ,  Karola Rittner ,  Xavier Preville

IPC分类号： A61K39/00 ,  C07K16/28 ,  A61K38/20 ,  A61K38/17 ,  A61K38/19 ,  A61K39/12 ,  A61K35/761 ,  A61K39/395 ,  C07K16/30 ,  C12N7/00

CPC分类号： A61K39/0011 ,  C07K16/2818 ,  A61K39/00 ,  A61K38/2013 ,  A61K38/1709 ,  A61K38/193 ,  A61K39/12 ,  A61K35/761 ,  A61K39/00117 ,  A61K39/39558 ,  C07K16/3046 ,  C12N7/00 ,  A61K2039/5256 ,  C12N2710/24143 ,  C12N2710/24171 ,  A61K2039/505 ,  C07K2317/76 ,  C07K2317/73 ,  A61K2039/55533 ,  A61K2039/575

摘要： The present invention relates to a combination product, composition(s) and kit of parts comprising at least (i) a therapeutic vaccine and (ii) one or more immune checkpoint modulator(s). The present invention also concerns a method for treating a proliferative or an infectious disease as well as a method for eliciting or stimulating and/or re-orienting an immune response, wherein said methods comprise administering to a subject in need thereof said combination product or said composition(s).

公开(公告)号：US20240016889A1

公开(公告)日：2024-01-18

申请号：US18472574

申请日：2023-09-22

申请人： The Trustees of Columbia University in the City of New York

发明人： Umrao MONANI ,  Jeong KI-KIM

IPC分类号： A61K38/17 ,  A61P21/00 ,  A61K9/00 ,  A61K35/761 ,  A61K38/46 ,  C12N15/113

CPC分类号： A61K38/1709 ,  A61P21/00 ,  A61K9/0019 ,  A61K9/0085 ,  A61K35/761 ,  A61K38/465 ,  C12N15/113 ,  A61K48/00

摘要： The present disclosure provides compositions and methods of treating motor neuron diseases, including spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS). A modulator of a heat shock protein, such as an Hsp70 family member protein, may be used in the present method. Alternatively, a mutant heat shock protein may be used.