公开(公告)号：US20240350450A1

公开(公告)日：2024-10-24

申请号：US18687601

申请日：2022-08-30

申请人： CYL GmbH

发明人： Reinhold Wintersteiger ,  Chrisitian Bücherl-Harrer

IPC分类号： A61K31/341 ,  A61K31/194 ,  A61P7/00

CPC分类号： A61K31/341 ,  A61K31/194 ,  A61P7/00

摘要： The present invention relates to a method of treating or preventing a disease or condition that benefits from an increased hemoglobin-mediated oxygen supply comprising administering to a subject a pharmaceutical composition comprising α-ketoglutaric acid (α-KG) and 5-hydroxymethyl-2-furfural (5-HFM).

公开(公告)号：US20240343786A1

公开(公告)日：2024-10-17

申请号：US18682310

申请日：2022-08-18

申请人： Alexion Pharmaceuticals, Inc.

发明人： Sung-Kwon Kim ,  Stephanie L. Beq

IPC分类号： C07K16/18 ,  A61K39/00 ,  A61P7/00 ,  C12N15/115

CPC分类号： C07K16/18 ,  A61P7/00 ,  C12N15/115 ,  A61K2039/505 ,  C07K2317/565 ,  C07K2317/76

摘要： The present disclosure relates to methods, uses, and compositions for the treatment of Sickle cell disease (SCD), beta thalassemia (BT), or sickle cell BT. More specifically, the disclosure concerns the treatment of patients having SCD. BT, or sickle cell BT using a complement C5 inhibitor, such as an anti-C5 antibody or fragment thereof, a nucleic acid molecule, a peptide, a small molecule, or an aptamer.

公开(公告)号：US20240309388A1

公开(公告)日：2024-09-19

申请号：US18441001

申请日：2024-02-14

申请人： Novo Nordisk A/S ,  Dicerna Pharmaceuticals, Inc.

发明人： Anna Linda Blois ,  Christina Marie Priest ,  Jihye Park ,  Henryk Dudek

IPC分类号： C12N15/113 ,  A61P7/00

CPC分类号： C12N15/1138 ,  A61P7/00 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/531

摘要： Oligonucleotides are provided herein that inhibit TMPRSS6 expression. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with hepcidin deficiency or suppression.

公开(公告)号：US20240301413A1

公开(公告)日：2024-09-12

申请号：US18550070

申请日：2022-03-11

申请人： THE CHILDREN'S HOSPITAL OF PHILADELPHIA

发明人： Wei Tong

IPC分类号： C12N15/113 ,  A61P7/00 ,  A61P7/06

CPC分类号： C12N15/113 ,  A61P7/00 ,  A61P7/06 ,  C12N2310/20 ,  C12N2310/531

摘要： Compositions and methods for the treatment of ribosomopathies and regeneration of hematopoietic stems cells are provided.

公开(公告)号：US12076342B2

公开(公告)日：2024-09-03

申请号：US16091266

申请日：2017-04-07

申请人： THE GOVERNING COUNCIL OF THE UNIVERSITY OF TORONTO

发明人： Peter W. Zandstra ,  Shreya Shukla

IPC分类号： A61K35/17 ,  C12N5/074 ,  C12N5/0783 ,  C12N5/0789 ,  A61P7/00 ,  A61P35/00

CPC分类号： A61K35/17 ,  C12N5/0607 ,  C12N5/0636 ,  C12N5/0647 ,  C12N5/0696 ,  A61P7/00 ,  A61P35/00 ,  C12N2500/38 ,  C12N2500/99 ,  C12N2501/115 ,  C12N2501/125 ,  C12N2501/145 ,  C12N2501/155 ,  C12N2501/165 ,  C12N2501/2307 ,  C12N2501/25 ,  C12N2501/42 ,  C12N2501/58 ,  C12N2501/998 ,  C12N2502/1171 ,  C12N2533/52 ,  C12N2533/90

摘要： The present disclosure provides a method for generating progenitor T cells from stem and/or progenitor cells comprising exposing the stem and/or progenitor cells to Notch ligand Delta-like-4 (DL4) and vascular adhesion molecule 1 (VCAM-1) under conditions suitable to generate progenitor T cells. The method provided is suitable for in vitro and in vivo pro-T cell generation. In vitro, the pro-T cells are generated under serum-free conditions. Cells produced using the method are provided as well as methods of using same.

公开(公告)号：US20240287192A1

公开(公告)日：2024-08-29

申请号：US18350267

申请日：2023-07-11

申请人： Horizon Therapeutics Ireland DAC

发明人： Katherine Ann VOUSDEN ,  Bo CHEN ,  Gary Patrick SIMS

IPC分类号： C07K16/28 ,  A61P7/00

CPC分类号： C07K16/283 ,  A61P7/00 ,  C07K2317/24 ,  C07K2317/33 ,  C07K2317/565 ,  C07K2317/76 ,  C07K2317/77 ,  C07K2317/92

摘要： The disclosure provides FcγRIIA-binding molecules, for example, humanized monoclonal antibodies capable of inhibiting FcγRIIA activity, and methods of using the FcγRIIA binding molecules, for example, in treating or preventing inflammatory, immune-mediated, or autoimmune diseases or disorders.

公开(公告)号：US12065441B2

公开(公告)日：2024-08-20

申请号：US16972605

申请日：2019-06-05

申请人： GENGLE THERAPEUTICS, INC.

发明人： Jianming Yin

IPC分类号： C07D487/04 ,  A61P1/00 ,  A61P7/00 ,  A61P17/00 ,  A61P17/04 ,  A61P19/02 ,  A61P19/08 ,  A61P29/00 ,  A61P35/00 ,  A61P37/00 ,  A61P37/06 ,  C07D471/04

CPC分类号： C07D487/04 ,  A61P1/00 ,  A61P7/00 ,  A61P17/00 ,  A61P17/04 ,  A61P19/02 ,  A61P19/08 ,  A61P29/00 ,  A61P35/00 ,  A61P37/00 ,  A61P37/06 ,  C07D471/04

摘要： Disclosed is a pyrazolopyrimidine derivative having a structure represented by general formula (I), a pharmaceutically acceptable salt, hydrate and metabolite formed by metabolism in any form thereof, and use in preparing drugs for preventing and/or treating indications associated with JAK kinase function, and a pharmaceutical composition prepared therefrom for preventing and/or treating indications associated with JAK kinase function. The present disclosure is a selective JAK kinase inhibitor, which has a pharmaceutical therapeutic effect on immunity and inflammatory responses by acting on JAK kinase.

公开(公告)号：US12059493B2

公开(公告)日：2024-08-13

申请号：US18492833

申请日：2023-10-24

申请人： Albert Einstein College of Medicine

发明人： Joel M. Friedman

IPC分类号： A61K31/12 ,  A61K9/00 ,  A61K9/06 ,  A61K31/05 ,  A61K31/121 ,  A61K31/352 ,  A61K31/366 ,  A61K31/4375 ,  A61K31/593 ,  A61K47/10 ,  A61K47/12 ,  A61P7/00 ,  A61P37/06

CPC分类号： A61K9/0014 ,  A61K9/06 ,  A61K31/05 ,  A61K31/12 ,  A61K31/121 ,  A61K31/352 ,  A61K31/366 ,  A61K31/4375 ,  A61K31/593 ,  A61K47/10 ,  A61K47/12 ,  A61P7/00 ,  A61P37/06

摘要： A transdermal formulation for the delivery of one or more active agents to a subject is provided. The formulation can be applied to the treatment of various diseases or conditions associated with an elevated CD38 level.

公开(公告)号：US12043843B2

公开(公告)日：2024-07-23

申请号：US15762700

申请日：2016-11-04

申请人： CRISPR Therapeutics AG

发明人： Matthew Hebden Porteus ,  Melanie Ruth Allen ,  Chad Albert Cowan ,  Ante Sven Lundberg ,  Michelle I-Ching Lin ,  Jeffrey Li ,  Thao Thi Nguyen

IPC分类号： C12N15/85 ,  A61P7/00 ,  C07K14/805 ,  C12N9/22 ,  C12N15/11 ,  C12N15/90 ,  A61K48/00 ,  C12N15/10 ,  C12N15/113 ,  C12N15/63

CPC分类号： C12N15/85 ,  A61P7/00 ,  C07K14/805 ,  C12N9/22 ,  C12N15/11 ,  C12N15/907 ,  A61K48/00 ,  C12N15/102 ,  C12N15/113 ,  C12N15/63 ,  C12N2310/20 ,  C12N2800/80

摘要： The present application provides materials and methods for treating hemoglobinopathies. More specifically, the application provides methods for producing progenitor cells that are genetically modified via genome editing to increase the production of fetal hemoglobin (HbF), as well as modified progenitor cells (including, for example, CD34+ human hematopoietic stem cells) producing increased levels of HbF, and methods of using such cells for treating hemoglobinopathies such as sickle cell anemia and β-thalassemia.

公开(公告)号：US20240238296A1

公开(公告)日：2024-07-18

申请号：US18557694

申请日：2022-04-28

申请人： Fulcrum Therapeutics, Inc.

发明人： Paul Bruno ,  Christopher Moxham ,  Billy Stuart

IPC分类号： A61K31/519 ,  A61K31/437 ,  A61K31/472 ,  A61P7/00 ,  C12Q1/6883 ,  G01N33/68

CPC分类号： A61K31/519 ,  A61K31/437 ,  A61K31/472 ,  A61P7/00 ,  C12Q1/6883 ,  G01N33/6893 ,  C12Q2600/158

摘要： Provided herein, in part, are methods of downregulating BCL11A expression and treatment of BCL11A mediated disorders. The methods may comprise the use of inhibitors such as EED, EHZ2, and/or PRC2 inhibitors.