公开(公告)号：US12127537B2

公开(公告)日：2024-10-29

申请号：US17316266

申请日：2021-05-10

Applicant: Regeneron Pharmaceuticals, Inc. ,  Yale University ,  Institute for Research in Biomedicine (IRB)

Inventor： Richard Flavell ,  Markus Manz ,  Anthony Rongvaux ,  Till Strowig ,  Tim Willinger ,  Andrew J. Murphy ,  Sean Stevens ,  George Yancopoulos

IPC: A01K67/0278 ,  A01K67/0271 ,  C12N15/87

CPC classification number: A01K67/0278 ,  A01K67/0271 ,  C12N15/87 ,  A01K2207/12 ,  A01K2207/15 ,  A01K2217/072 ,  A01K2217/15 ,  A01K2227/105 ,  A01K2267/0331 ,  A01K2267/0381 ,  A01K2267/0387

Abstract: The invention relates generally to genetically modified non-human animals expressing human polypeptides and their methods of use.

公开(公告)号：US12121594B2

公开(公告)日：2024-10-22

申请号：US17820243

申请日：2022-08-16

Applicant: International Centre for Genetic Engineering and Biotechnology (ICGEB)

Inventor： Mauro Giacca ,  Giulia Ruozi ,  Francesca Bortolotti

IPC: A61K48/00 ,  A61K38/17 ,  C07K14/47

CPC classification number: A61K48/005 ,  A61K38/1709 ,  C07K14/47 ,  A01K2207/20 ,  A01K2217/052 ,  A01K2227/105 ,  A01K2267/0393 ,  C12N2750/14143

Abstract: A protein selected from the group consisting of Chrdl1, Fam3c, Fam3b and a fragment thereof, or a polynucleotide encoding therefor, for use in treating or reducing the risk of heart disease.

公开(公告)号：US20240315220A1

公开(公告)日：2024-09-26

申请号：US18605786

申请日：2024-03-14

Applicant: The Jackson Laboratory ,  Indiana University Research and Technology Corporation

Inventor： Gareth Howell ,  Michael Sasner ,  Gregory Carter ,  Bruce Lamb

IPC: A01K67/0278 ,  C07K14/705 ,  C07K14/775 ,  G01N33/68

CPC classification number: A01K67/0278 ,  C07K14/70503 ,  C07K14/775 ,  G01N33/6896 ,  A01K2217/056 ,  A01K2217/072 ,  A01K2217/15 ,  A01K2227/105 ,  A01K2267/0312 ,  G01N2500/00

Abstract: Genetically modified mice characterized by one or more symptoms or signs associated with expression of human APOE4p and mouse Trem2p and relevant to non-familial late-onset Alzheimer's disease are provided wherein the genome of the mouse includes: 1) a DNA sequence encoding a human APOE4 protein (APOE4p) operably linked to a promoter; and 2) a DNA sequence encoding a mouse Trem2 protein having a mutation p.R47H (Trem2p) operably linked to a promoter, such that the mouse expresses human APOE4p and mouse Trem2p. Methods are provided for screening for a compound for use in the treatment of Alzheimer's disease using such genetically modified mice.

公开(公告)号：US20240306616A1

公开(公告)日：2024-09-19

申请号：US18442444

申请日：2024-02-15

Applicant: Regeneron Pharmaceuticals, Inc.

Inventor： Kara L. Olson ,  Eric Smith ,  Ka-Man Venus Lai ,  Andrew J. Murphy ,  Gavin Thurston ,  Dayong Guo

IPC: A01K67/0278 ,  A01K67/0276 ,  C07K14/725 ,  C12N5/071 ,  C12N15/85 ,  G01N33/50

CPC classification number: A01K67/0278 ,  A01K67/0276 ,  C07K14/7051 ,  C12N5/0602 ,  C12N15/8509 ,  G01N33/5088 ,  A01K2207/12 ,  A01K2207/15 ,  A01K2217/072 ,  A01K2217/075 ,  A01K2217/15 ,  A01K2227/105 ,  A01K2267/01 ,  A01K2267/03 ,  A01K2267/0337 ,  Y02A50/30

Abstract: Non-human animals, expressing humanized CD3 proteins are provided. Non-human animals, e.g., rodents, genetically modified to comprise in their genome humanized CD3 proteins are also provided. Additionally, provided are methods and compositions of making such non-human animals, as well as methods of using said non-human animals.

公开(公告)号：US12091675B2

公开(公告)日：2024-09-17

申请号：US17296944

申请日：2019-07-15

Applicant: Takeda Pharmaceutical Company Limited

Inventor： Hanspeter Rottensteiner ,  Werner Hoellriegl

IPC: C12N15/861 ,  A61K38/37 ,  A61K48/00 ,  C07K14/755

CPC classification number: C12N15/861 ,  A61K38/37 ,  A61K48/0083 ,  C07K14/755 ,  A01K2217/075 ,  A01K2227/105 ,  A01K2267/0381

Abstract: The present disclosure provides, among other aspects, codon-altered polynucleotides encoding Factor VIII variants for expression in mammalian cells. In some embodiments, the disclosure also provides mammalian gene therapy vectors and methods for treating hemophilia A. In some embodiments, the present disclosure provides methods for dosing a hemophilia A patient with a polynucleotide, e.g., a codon-altered polynucleotide, encoding a Factor VIII polypeptide.

公开(公告)号：US20240301017A1

公开(公告)日：2024-09-12

申请号：US18658138

申请日：2024-05-08

Applicant: The Regents of the University of California

Inventor： Stephanie Cherqui

IPC: C07K14/47 ,  A01K67/0276 ,  A01K67/0278 ,  A61K35/28 ,  A61P25/28 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86

CPC classification number: C07K14/47 ,  A01K67/0276 ,  A01K67/0278 ,  A61K35/28 ,  A61P25/28 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86 ,  A01K2207/15 ,  A01K2217/05 ,  A01K2217/075 ,  A01K2217/15 ,  A01K2227/105 ,  A01K2267/0318 ,  C07K2319/60 ,  C12N2310/20 ,  C12N2740/16043 ,  C12N2800/80

Abstract: Provided herein are methods for treating a disease or disorder associated with mitochondrial dysfunction through ex vivo introduction of a nucleic acid molecule into hematopoietic stem and progenitor cells (HSPCs) followed by transplantation of the HSPCs into a subject in need of treatment. The nucleic acid molecule may include a functional human frataxin (hFXN) or may include a gene editing system that when transfected into the cells removes a trinucleotide extension mutation of endogenous hFXN.

公开(公告)号：US20240298619A1

公开(公告)日：2024-09-12

申请号：US18316461

申请日：2023-05-12

Applicant: Regeneron Pharmaceuticals, Inc.

Inventor： Elena Burova ,  Alexander O. Mujica ,  Ka-Man Venus Lai ,  Andrew J. Murphy

IPC: A01K67/0278 ,  A61K49/00 ,  C07K14/435 ,  C07K14/47 ,  C07K16/28 ,  C12Q1/6886 ,  G01N33/50

CPC classification number: A01K67/0278 ,  A61K49/0008 ,  C07K14/4747 ,  C07K16/2803 ,  C12Q1/6886 ,  G01N33/5038 ,  A01K2207/15 ,  A01K2217/05 ,  A01K2217/072 ,  A01K2227/105 ,  A01K2267/0331 ,  C07K14/435 ,  C07K2319/00 ,  C07K2319/02 ,  C07K2319/03 ,  C12Q2600/106 ,  C12Q2600/158 ,  G01N2333/57 ,  G01N2333/70503 ,  G01N2500/10

Abstract: Non-human animals, and methods and compositions for making and using the same, are provided, wherein the non-human animals comprise a humanization of a Programmed cell death 1 (Pdcd1) gene. The non-human animals, in some embodiments, comprise a genetic modification to an endogenous Pdcd1 gene so that the non-human animals express a PD-1 polypeptide that includes a human portion and an endogenous portion (e.g., a non-human portion).

公开(公告)号：US20240298618A1

公开(公告)日：2024-09-12

申请号：US18654005

申请日：2024-05-03

Applicant: PUSAN NATIONAL UNIVERSITY INDUSTRY-UNIVERSITY COOPERATION FOUNDATION

Inventor： Dong Jun LEE ,  Soo Yeon WOO ,  Hee Seon CHOI

IPC: A01K67/0276 ,  C12N9/12 ,  G01N33/50

CPC classification number: A01K67/0276 ,  C12N9/1241 ,  G01N33/5017 ,  A01K2217/075 ,  A01K2227/105 ,  A01K2267/0331 ,  G01N2500/10

Abstract: The present disclosure relates to an upstream of mTORC2 (UT2) gene-deficient mouse model and a method of screening therapeutic agents for myeloid leukemia using the same, wherein it was found that the onset of myeloid leukemia and proliferation of myeloid leukemia cells were more facilitated in UT2 gene-deficient mice and UT2 genetic scissor (Cripsr/Cas9)-deficient myeloid leukemia cell lines. In addition, it was found that the survival rate of patients with low expression of UT2 was low in myeloid leukemia patients, while expression of UT2 was low in cells of actual myeloid leukemia patients.

公开(公告)号：US12082565B2

公开(公告)日：2024-09-10

申请号：US17348907

申请日：2021-06-16

Applicant: Regeneron Pharmaceuticals, Inc.

Inventor： Susannah Brydges ,  Yajun Tang ,  Yang Liu ,  Jingtai Cao ,  Carmelo Romano

IPC: C12N5/00 ,  A01K67/0276 ,  A01K67/0278 ,  C07K14/47 ,  C12N15/85 ,  G01N33/50 ,  G01N33/68

CPC classification number: A01K67/0278 ,  A01K67/0276 ,  C07K14/47 ,  C12N15/8509 ,  G01N33/5088 ,  G01N33/6893 ,  A01K2217/054 ,  A01K2217/072 ,  A01K2217/075 ,  A01K2217/15 ,  A01K2217/206 ,  A01K2227/105 ,  A01K2267/0306 ,  A01K2267/0393 ,  C12N2015/8527 ,  C12N2015/8536 ,  C12N2510/00 ,  C12N2800/30 ,  C12N2830/008

Abstract: Non-human animals suitable for use as animal models for Retinoschisis are provided. In some embodiments, provided non-human animals are characterized by a disruption in a Retinoschisin-1 locus. In some embodiments, provided non-human animals are characterized by a mutant Retinoschisin-1 gene. The non-human animals may be described, in some embodiments, as having a phenotype that includes the development of one or more symptoms or phenotypes associated with Retinoschisis. Methods of identifying therapeutic candidates that may be used to prevent, delay or treat Retinoschisis or eye-related diseases, disorders or conditions are also provided.

公开(公告)号：US12070021B2

公开(公告)日：2024-08-27

申请号：US16650814

申请日：2018-09-25

Applicant: National University of Singapore ,  Agency for Science, Technology and Research

Inventor： Linfa Wang ,  Qingfeng Chen ,  Han Jia Justin Ng ,  Kylie Su Mei Yong

IPC: A01K67/0271

CPC classification number: A01K67/0271 ,  A01K2207/12 ,  A01K2227/105 ,  A01K2267/0337 ,  A01K2267/0387

Abstract: Generation of chimeric non-human animals hosting bat donor cells involves chimeric mice having bat cells that may be stably tolerated to provide a new platform technology in the general field of biology, and having application in the field of immunology related to virus-host interaction, cancer biology, autoimmunity, and the development of new drugs.