公开(公告)号：US20230330195A1

公开(公告)日：2023-10-19

申请号：US17972533

申请日：2022-10-24

Applicant: Sangamo Therapeutics, Inc. ,  Regents of the University of Minnesota

Inventor： Russell DeKelver ,  R. Scott McIvor ,  Li Ou ,  Thomas Wechsler ,  Chester B. Whitley ,  Kanut Laoharawee

IPC: A61K38/47 ,  A61K48/00 ,  C12N15/90 ,  C12N15/10 ,  A61K9/00 ,  A61K38/17 ,  A61K38/46 ,  C12N7/00 ,  C12N9/16 ,  C12N9/24

CPC classification number: A61K38/47 ,  A61K48/005 ,  C12N15/907 ,  C12N15/102 ,  A61K9/0019 ,  A61K38/1703 ,  A61K38/465 ,  A61K48/0058 ,  C12N7/00 ,  C12N9/16 ,  C12N9/2402 ,  C12Y301/06013 ,  C12Y302/01076 ,  A01K2227/105 ,  C12N2750/10041 ,  A01K2217/075 ,  A01K2267/0362 ,  C12N2750/14143

Abstract: Nucleases and methods of using these nucleases for inserting a sequence encoding a therapeutic IDUA or IDS protein such as an enzyme into a cell, thereby providing proteins or cell therapeutics for treatment and/or prevention of MPS I or MPS II disease.

公开(公告)号：US11788101B2

公开(公告)日：2023-10-17

申请号：US16778996

申请日：2020-01-31

Applicant: UNIVERSITY OF WASHINGTON

Inventor： George Stamatoyannopoulos ,  John Stamatoyannopoulos

IPC: C12N15/86 ,  C12N15/10 ,  C12N15/85 ,  C12N15/113 ,  A61K48/00 ,  C12Q1/6876

CPC classification number: C12N15/86 ,  A61K48/005 ,  C12N15/113 ,  C12Q1/6876 ,  C12N2310/141 ,  C12N2740/15043 ,  C12N2740/16043 ,  C12N2830/40

Abstract: Provided herein are methods for identifying high potency genomic insulator elements that can be used in a vector composition e.g., that are useful for preventing unwanted expression of neighboring genes, such as proto-oncogenes, when administered to a subject in need thereof. Also provided herein are methods for treating disease and methods for administering a nucleic acid to a subject using such vectors.

公开(公告)号：US11786610B2

公开(公告)日：2023-10-17

申请号：US18048253

申请日：2022-10-20

Applicant: TranscripTx, Inc.

Inventor： David J. Lockhart ,  Brandon Wustman ,  Mirko Hennig ,  Daniella Ishimaru

IPC: A61K48/00 ,  A61P11/00 ,  A61P11/06 ,  A61K9/127 ,  A61K31/7105 ,  A61K31/7115 ,  C12N15/113 ,  A61K47/69

CPC classification number: A61K48/005 ,  A61K9/1271 ,  A61K31/7105 ,  A61K31/7115 ,  A61K47/6925 ,  A61K48/0041 ,  A61P11/00 ,  A61P11/06 ,  C12N15/113

Abstract: Polynucleotides encoding peptides, proteins, enzymes, and functional fragments thereof are disclosed. The polynucleotides of the disclosure can be effectively delivered to an organ, such as the lung, and expressed within cells of the organ. The polyribonucleotides of the disclosure can be used to treat a disease or condition associated with cilia maintenance and function, impaired function of the axoneme, such as DNAI1 or DNAH5.

公开(公告)号：US20230323398A1

公开(公告)日：2023-10-12

申请号：US18160241

申请日：2023-01-26

Applicant: Novartis AG

Inventor： Chad Eric BIGELOW ,  Vivian CHOI ,  Thaddeus Peter DRYJA ,  Akshata Ninad GUJAR ,  Shawn Michael HANKS ,  Terri MCGEE ,  Seshidhar Reddy POLICE ,  Joanna VROUVLIANIS

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

CPC classification number: C12N15/86 ,  A61K38/1709 ,  A61K48/005 ,  A61K48/0058 ,  C07K14/47 ,  C12N7/00 ,  C12N15/79

Abstract: The present invention relates to viral vectors that are capable of delivering a heterologous gene to the retina and in particular delivering RLBP1 to RPE and Müller cells of the retina. The invention also relates nucleic acids useful for producing viral vectors, compositions comprising the viral vectors and uses of the compositions and viral vectors. The invention also relates to methods of delivering and/or expressing a heterologous gene to the retina, improving the rate of dark adaption in a subject and treating RLBP1-associated retinal dystrophy.

公开(公告)号：US20230321276A1

公开(公告)日：2023-10-12

申请号：US18040803

申请日：2021-09-29

Applicant: ALMA BIO THERAPEUTICS

Inventor： Binah BAUM ,  Irun COHEN ,  Raanan MARGALIT

IPC: A61K48/00 ,  A61K45/06 ,  A61P1/00 ,  A61K38/17

CPC classification number: A61K48/005 ,  A61K38/1709 ,  A61K45/06 ,  A61P1/00

Abstract: The invention is directed to nucleic acid therapy for modulating host microflora, useful in the management of dysbiosis. The invention in embodiments thereof provides compositions and methods for alleviating dysbiosis and conditions associated therewith. According to additional embodiments, compositions and methods of the invention may be used for treating or preventing gut barrier dysfunction in a subject in need thereof, and for reducing the risk of developing adverse events related to expansion of gastrointestinal bacteria in patients at risk for developing dysbiosis, for example in hospitalized patients and immune suppressed subjects.

公开(公告)号：US11779655B2

公开(公告)日：2023-10-10

申请号：US17557409

申请日：2021-12-21

Applicant: SwanBio Therapeutics Limited

Inventor： Sean Clark ,  Karen Kozarsky ,  Tugba Guven-Ozkan ,  Anna Tretiakova

IPC: A61K48/00 ,  A61K9/00 ,  C12N15/86

CPC classification number: A61K48/005 ,  A61K9/0019 ,  C12N15/86 ,  C12N2800/107

Abstract: The present disclosure generally relates to polynucleotides and AAV vectors that provide for the expression of ALD protein in target (e.g., neurons or glial) cells when administered to subjects in need thereof. The present disclosure further relates to compositions comprising such a polynucleotide or vector. These polynucleotides, vectors, and compositions may be used for the treatment and prevention of ALD or AMN in subjects in need thereof.

公开(公告)号：US11779654B2

公开(公告)日：2023-10-10

申请号：US16753117

申请日：2018-10-04

Applicant: 2seventy bio, Inc.

Inventor： Jordan Jarjour ,  Kyle Havens ,  Michael Certo ,  Max Echterling

IPC: A61K48/00 ,  A61P3/06 ,  C12N9/22 ,  C12N9/64 ,  C12N15/63 ,  C12N15/90

CPC classification number: A61K48/005 ,  A61K48/0041 ,  A61P3/06 ,  C12N9/22 ,  C12N9/6454 ,  C12N15/63 ,  C12N15/90 ,  C12Y304/21061

Abstract: The present disclosure provides improved genome editing compositions and methods for editing a PCSK9 gene. The disclosure further provides genome edited cells for the prevention, treatment, or amelioration of at least one symptom of hypercholesterolemia or a condition associated therewith.

公开(公告)号：US20230304031A1

公开(公告)日：2023-09-28

申请号：US17924080

申请日：2021-05-11

Applicant: Abintus Bio, Inc.

Inventor： Douglas J. Jolly ,  Derek G. Ostertag ,  Noriyuki Kasahara ,  Carlos Ibanez ,  Cornelia A. Bentley ,  Mohd Mushtaq Husain

IPC: C12N15/86 ,  C07K14/725 ,  C07K16/18 ,  C12N15/113 ,  C12N9/78 ,  C12N9/12 ,  A61K35/76 ,  A61P35/00 ,  A61K48/00

CPC classification number: C12N15/86 ,  A61K35/76 ,  A61K48/005 ,  A61P35/00 ,  C07K14/7051 ,  C07K16/18 ,  C12N9/1211 ,  C12N9/78 ,  C12N15/113

Abstract: The disclosure provides compositions and methods for inducing an immunity via cellular expression of an antigen receptor binding construct in vivo.

公开(公告)号：US20230303642A1

公开(公告)日：2023-09-28

申请号：US18000461

申请日：2021-06-06

Applicant: The Regents of the University of California

Inventor： Gregory M. Barton ,  Nicholas Anton Lind ,  Olivia Majer ,  Bo Liu

IPC: C07K14/47 ,  C12N15/85 ,  A61K48/00

CPC classification number: C07K14/4702 ,  A61K48/005 ,  C12N15/85

Abstract: Disclosed herein are Unc93bl mutations that modulated the trafficking and/or signaling of TLR7, and compositions and methods of using thereof.

公开(公告)号：US20230287374A1

公开(公告)日：2023-09-14

申请号：US18155097

申请日：2023-01-17

Applicant: Guangzhou Medical University ,  GMU New Drug Development Co., Ltd.

Inventor： Xi-Yong Yu ,  Yu-Guang Zhu ,  Ao Shen ,  Ling-Min Zhang ,  Ji-Shuo Chang

IPC: C12N9/22 ,  C12N15/85 ,  A61K48/00

CPC classification number: C12N9/22 ,  A61K48/005 ,  A61K48/0008 ,  C12N15/85 ,  C07K2319/09 ,  C12N2310/20

Abstract: A targeted DNA demethylation method (Mini-CRISD) and a fusion protein used in the method are provided. The Mini-CRISD method can targets delivery of demethylation activity through engineered miniature dCjCas9 to deliver engineered miniature ROS1 demethylation effector to specific DNA sequences and/or specific genomic locations (such as CpG islands) to achieve targeted demethylation of specific DNA.