公开(公告)号：US20240358852A1

公开(公告)日：2024-10-31

申请号：US18556251

申请日：2022-04-19

Applicant: Anjarium Biosciences AG

Inventor： Joel de Beer ,  Monique Maurer ,  Nicolas Meier ,  Lavaniya Kunalingam ,  Marcello Clerici

IPC: A61K48/00 ,  A61P3/00 ,  C12N9/10 ,  C12N9/44

CPC classification number: A61K48/005 ,  A61K48/0041 ,  A61P3/00 ,  C12N9/1051 ,  C12N9/2451 ,  C12Y204/01025 ,  C12Y302/01033

Abstract: Provided herein are double strand DNA molecules comprising inverted repeats, expression cassette and one or more restriction sites for nicking endonucleases, the methods of use thereof, and the methods of making therefor.

公开(公告)号：US20240350584A1

公开(公告)日：2024-10-24

申请号：US18616511

申请日：2024-03-26

Applicant: Genzyme Corporation

Inventor： Marco A. PASSINI ,  Lamya Shihabuddin ,  Seng H. Cheng

IPC: A61K38/17 ,  A61K31/7088 ,  A61K48/00 ,  C07K14/47 ,  C12N7/00 ,  C12N15/86

CPC classification number: A61K38/1709 ,  A61K31/7088 ,  A61K48/00 ,  A61K48/0008 ,  A61K48/005 ,  A61K48/0075 ,  C07K14/4702 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14121 ,  C12N2750/14133 ,  C12N2750/14143 ,  C12N2750/14171

Abstract: Compositions and methods for treating disorders affecting motor function, such as motor function affected by disease or injury to the brain and/or spinal cord, are disclosed.

公开(公告)号：US20240343768A1

公开(公告)日：2024-10-17

申请号：US18602622

申请日：2024-03-12

Applicant: The General Hospital Corporation

Inventor： Xandra BREAKEFIELD ,  Casey MAGUIRE ,  Shilpa PRABHAKAR ,  David YELLEN

IPC: C07K14/47 ,  A61K9/00 ,  A61K31/436 ,  A61K38/17 ,  A61K48/00 ,  A61P35/00 ,  C12N7/00 ,  C12N15/86

CPC classification number: C07K14/4705 ,  A61K9/0019 ,  A61K9/007 ,  A61K9/0085 ,  A61K31/436 ,  A61K38/1709 ,  A61K48/005 ,  A61P35/00 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2750/14171 ,  C12N2830/50

Abstract: The invention provides compositions and methods for treating tuberous sclerosis complex (TSC). In particular, provided are condensed tuberins (cTuberins), cTuberin nucleic acids, and recombinant adeno-associated viruses (rAAVs) carrying a cTuberin nucleic acid for treating a patient with TSC.

公开(公告)号：US20240325569A1

公开(公告)日：2024-10-03

申请号：US18268731

申请日：2019-12-18

Applicant: CELL AND GENE THERAPY LTD ,  OBSCHESTVO S OGRANICHENNOI OTVETSTVENNOSTJU "PRORYVNYE INNOVATSIONNYE TEKHNOLOGII"

Inventor： Natalia SAVELIEVA

IPC: A61K48/00 ,  A61K38/18 ,  A61K38/30 ,  C12N15/10 ,  C12N15/85

CPC classification number: A61K48/005 ,  A61K38/185 ,  A61K38/1866 ,  A61K38/30 ,  C12N15/1096 ,  C12N15/85

Abstract: The invention refers to genetic engineering and can be used in biotechnology, medicine, and agriculture for the manufacture of gene therapy products. Gene therapy DNA vector based on the gene therapy DNA vector VTvaf 17 carrying the therapeutic gene selected from the group of BDNF, VEGFA, BFGF, NGF, GDNF, NT3, CNTF, and IGF1 genes was constructed in order to increase the expression level of this therapeutic gene in humans and animals, while gene therapy DNA vector VTvaf17-BDNF, or VTvaf 17-VEGFA, or VTvaf17-BFGF, or VTvaf17-NGF, or VTvaf17-GDNF, or VTvaf17-NT3, or VTvaf17-CNTF, or VTvaf17-IGFl, has the nucleotide sequence SEQ ID No. 1, or SEQ ID No. 2, or SEQ ID No. 3, or SEQ ID No. 4, or SEQ ID No. 5, or SEQ ID No. 6, or SEQ ID No. 7, or SEQ ID No. 8, respectively.

公开(公告)号：US20240325506A1

公开(公告)日：2024-10-03

申请号：US18573086

申请日：2022-06-23

Applicant: AvroBio, Inc.

Inventor： Mark DEANDRADE ,  Chris MASON ,  Chris MASON

IPC: A61K38/47 ,  A61K38/17 ,  A61K48/00 ,  A61P3/00 ,  C12N15/113

CPC classification number: A61K38/47 ,  A61K38/1774 ,  A61K48/0033 ,  A61K48/005 ,  A61P3/00 ,  C12N15/1137 ,  C12N15/1138 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/531

Abstract: Described herein are methods for treating a subject having or at risk of developing Gaucher disease, by administering one or more agents that increase expression and/or activity of glucocerebrosidase (GBA) and/or scavenger receptor class B member 2 (SCARB2), such as pluripotent cells that express GBA and/or SCARB2, and to the subject. Also disclosed are compositions comprising one or more agents that increase the expression and/or activity of GBA and/or SCARB2.

公开(公告)号：US20240316218A1

公开(公告)日：2024-09-26

申请号：US18544247

申请日：2023-12-18

Applicant: Northwestern University

Inventor： Rishi ARORA

IPC: A61K48/00 ,  A61K38/00 ,  A61P9/06 ,  C07K14/47 ,  C12N15/113

CPC classification number: A61K48/005 ,  A61P9/06 ,  C07K14/4703 ,  C12N15/1137 ,  A61K38/00

Abstract: Provided herein are compositions, methods, and devices for the treatment and prevention of atrial fibrillation (AF) using gene therapy techniques. In particular, oxidative stress (OS) and parasympathetic nervous system signaling are inhibited to prevent and/or reverse the electrical remodeling that underlies AF.

公开(公告)号：US20240316165A1

公开(公告)日：2024-09-26

申请号：US18578052

申请日：2022-07-12

Applicant: ModernaTX, Inc.

Inventor： Husain Attarwala ,  Lei Jiang ,  Matthew Lumley

IPC: A61K38/53 ,  A61K9/51 ,  A61K31/167 ,  A61K31/192 ,  A61K47/24 ,  A61K48/00 ,  A61P3/00

CPC classification number: A61K38/53 ,  A61K9/5123 ,  A61K31/167 ,  A61K31/192 ,  A61K47/24 ,  A61K48/005 ,  A61P3/00

Abstract: This disclosure relates to mRNA therapy for the treatment of propionic acidemia. mRNAs for use in the invention, when administered in vivo, encode propionyl-CoA carboxy lase alpha (PCCA) and propionyl-CoA carboxy lase beta (PCCB). mRNA therapies of the disclosure increase and/or restore deficient levels of PCCA and/or PCCB expression and/or activity in subjects.

公开(公告)号：US12097267B2

公开(公告)日：2024-09-24

申请号：US17099421

申请日：2020-11-16

Applicant: The Trustees of the University of Pennsylvania

Inventor： Jeannette Bennicelli ,  Jean Bennett ,  Junwei Sun

IPC: A61K48/00 ,  A61K38/44 ,  A61P27/02 ,  C12N9/02 ,  C12N15/86

CPC classification number: A61K48/005 ,  A61K38/44 ,  A61K48/00 ,  A61K48/0075 ,  A61P27/02 ,  C12N9/0036 ,  C12N9/0051 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2800/22 ,  C12N2830/001

Abstract: Codon optimized nucleic acid sequences for the long form and short form of RdCVF are provided, as well as recombinant viral vectors, such as AAV, expression cassettes, proviral plasmids or other plasmids containing the codon optimized sequences. Recombinant vectors are provided that express the codon optimized RdCVFL and RdCVF individually, or express two copies of a codon optimized RdCVF or RdCVFL nucleic acid sequence, or both RdCVFL and RdCVF in a single vector or virus. Compositions containing these codon optimized sequences are useful in methods for treating, retarding or halting certain blinding diseases resulting from the absence or inappropriate expression of RdCVF and RdCVFL.

公开(公告)号：US20240285801A1

公开(公告)日：2024-08-29

申请号：US18588657

申请日：2024-02-27

Applicant: UNIVERSITY OF PITTSBURGH - OF THE COMMONWEALTH SYSTEM OF HIGHER EDUCATION

Inventor： Dwi U. Kemaladewi ,  Robert David Nicholls ,  Jia Qi Cheng Zhang

IPC: A61K48/00 ,  A61K38/39 ,  A61P21/00 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86

CPC classification number: A61K48/005 ,  A61K38/39 ,  A61P21/00 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86 ,  C12N2310/20 ,  C12N2750/14143

Abstract: This document provides methods and materials for treating a mammal (e.g., a human) having, or at risk of developing, muscular dystrophy (e.g., a laminin α2-related dystrophy (LAMA2-RD)). For example, an adeno-associated virus (AAV) designed for targeted gene activation of nucleic acid encoding a Lama1 polypeptide (e.g., the endogenous Lama1 gene) can be administered to a mammal (e.g., a human) having, or at risk of developing, muscular dystrophy (e.g., a LAMA2-RD) to treat the mammal.

公开(公告)号：US20240285775A1

公开(公告)日：2024-08-29

申请号：US18527005

申请日：2023-12-01

Applicant: Yale University

Inventor： Elias QUIJANO ,  Peter GLAZER

IPC: A61K47/54 ,  A61K39/00 ,  A61K47/68 ,  A61K48/00 ,  A61P35/00 ,  C07K14/47 ,  C07K14/52 ,  C07K16/44 ,  C12N15/11 ,  C12N15/113 ,  C12N15/87

CPC classification number: A61K47/549 ,  A61K47/6807 ,  A61K47/6851 ,  A61K48/0025 ,  A61K48/005 ,  A61P35/00 ,  C07K14/4747 ,  C07K14/52 ,  C07K16/44 ,  C12N15/111 ,  C12N15/113 ,  C12N15/1135 ,  C12N15/87 ,  A61K2039/505 ,  A61K48/00 ,  C07K2317/24 ,  C07K2317/31 ,  C07K2317/565 ,  C07K2317/622 ,  C07K2317/77 ,  C07K2319/33 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/3181 ,  C12N2310/3513 ,  C12N2320/32

Abstract: Compositions and methods of use thereof for delivering nucleic acid cargo into cells are provided. The compositions typically include (a) a 3E10 monoclonal antibody or an antigen binding, cell-penetrating fragment thereof; a monovalent, divalent, or multivalent single chain variable fragment (scFv); or a diabody; or humanized form or variant thereof, and (b) a nucleic acid cargo including, for example, a nucleic acid encoding a polypeptide, a functional nucleic acid, a nucleic acid encoding a functional nucleic acid, or a combination thereof. Elements (a) and (b) are typically non-covalently linked to form a complex.