公开(公告)号：US12129263B2

公开(公告)日：2024-10-29

申请号：US17511193

申请日：2021-10-26

申请人： Denali Therapeutics Inc.

发明人： Javier de Vicente Fidalgo ,  Anthony A. Estrada ,  Jianwen A. Feng ,  Brian Fox ,  Cinzia Maria Francini ,  Christopher R. H. Hale ,  Cheng Hu ,  Colin Philip Leslie ,  Maksim Osipov ,  Elena Serra ,  Zachary K. Sweeney ,  Arun Thottumkara

IPC分类号： C07D498/04 ,  A61K31/553 ,  A61P3/10 ,  A61P9/00 ,  A61P11/00 ,  A61P25/00 ,  A61P27/02 ,  A61P37/00 ,  A61P43/00 ,  C07D223/16 ,  C07D267/04 ,  C07D267/14 ,  C07D413/14 ,  C07D498/10 ,  C07D519/00

CPC分类号： C07D498/04 ,  C07D223/16 ,  C07D267/04 ,  C07D267/14 ,  C07D413/14 ,  C07D519/00

摘要： The present disclosure relates generally to compounds and compositions, intermediates, processes for their preparation, and their use as kinase inhibitors.

公开(公告)号：US12110491B2

公开(公告)日：2024-10-08

申请号：US17577832

申请日：2022-01-18

申请人： Ionis Pharmaceuticals, Inc.

发明人： C. Frank Bennett ,  Susan M. Freier ,  Sarah Greenlee ,  Eric E. Swayze

IPC分类号： C12N15/11 ,  C12N15/113

CPC分类号： C12N15/113 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2320/34 ,  C12N2320/35

摘要： Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate diseases, including neurodegenerative diseases, such as Huntington's Disease (HD).

公开(公告)号：US12071454B2

公开(公告)日：2024-08-27

申请号：US17061065

申请日：2020-10-01

申请人： Sanofi

发明人： Gary J. Nabel ,  Chih-Jen Wei ,  Kurt Swanson ,  Pradeep Dhal ,  Ram Dharanipragada ,  Magnus Besev

IPC分类号： C07K14/195

CPC分类号： C07K14/195 ,  C07K2319/30

摘要： Antigenic respiratory syncytial virus (RSV) polypeptides for use in eliciting antibodies against RSV are provided. Also provided are antigenic polypeptides comprising an RSV polypeptide and a ferritin protein.

公开(公告)号：US12128053B2

公开(公告)日：2024-10-29

申请号：US18171070

申请日：2023-02-17

申请人： Woolsey Pharmaceuticals, Inc.

发明人： Qicai Liu ,  Hemant N. Joshi ,  Thomas MacAllister

IPC分类号： A61K31/551 ,  A61K47/38

CPC分类号： A61K31/551 ,  A61K47/38

摘要： A taste-masking composition for pharmaceutical uses is provided comprising a rho kinase inhibitor, a pharmaceutically acceptable salt thereof, a hydrate thereof, a prodrug thereof, a substituted derivative thereof, or a metabolite thereof, or any combination thereof, the rho kinase inhibitor having a bitter taste and being present at a concentration above a taste threshold concentration. The rho kinase inhibitor can comprise fasudil. The composition further comprises a taste-masking agent that masks the bitter taste. The taste-masking agent can partially or fully mask the bitter taste of the rho kinase inhibitor. The taste-masking agent can comprise one or more agents. The composition can be formulated to treat a neurodegenerative disease, for example one or more symptoms of such diseases such as wandering. A method of treating a neurodegenerative disease using such a composition is also provided. Methods of manufacturing the compositions of the present disclosure are further provided.

公开(公告)号：US12121592B2

公开(公告)日：2024-10-22

申请号：US17832196

申请日：2022-06-03

申请人： Translate Bio, Inc.

发明人： Frank DeRosa ,  Braydon Charles Guild ,  Michael Heartlein

IPC分类号： A61K48/00 ,  A61K9/00 ,  A61K9/127 ,  A61K9/51 ,  A61K31/713 ,  A61K38/18 ,  A61K38/47 ,  A61K38/48 ,  A61K38/57 ,  C07K14/505 ,  C07K14/81 ,  C12N9/40 ,  C12N9/64 ,  C12N15/52 ,  C12N15/85

CPC分类号： A61K48/0008 ,  A61K9/0019 ,  A61K9/0073 ,  A61K9/1271 ,  A61K9/1272 ,  A61K9/5123 ,  A61K31/713 ,  A61K38/1816 ,  A61K38/47 ,  A61K38/4846 ,  A61K38/57 ,  A61K48/0075 ,  A61K48/0091 ,  C07K14/505 ,  C07K14/8125 ,  C12N9/2465 ,  C12N9/644 ,  C12N15/52 ,  C12N15/85 ,  C12Y302/01022 ,  C12Y304/21022 ,  A61K48/00

摘要： Disclosed herein are compositions and methods for modulating the production of a protein in a target cell. The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.

公开(公告)号：US12116396B2

公开(公告)日：2024-10-15

申请号：US18454526

申请日：2023-08-23

申请人： Immunomic Therapeutics

发明人： Teri Heiland

IPC分类号： C07K14/705 ,  A61K39/00 ,  A61P35/00

CPC分类号： C07K14/70596 ,  A61K39/00115 ,  A61P35/00 ,  A61K2039/55522 ,  A61K2039/57 ,  A61K2039/575 ,  A61K2039/6031 ,  C07K2319/02 ,  C07K2319/03 ,  C07K2319/40

摘要： The present invention provides improved LAMP Constructs comprising specific fragments of the LAMP lumenal domain to deliver antigens to immune cells for enhanced processing. These LAMP Constructs can be used for the treatment of disease and in particular, allergies, infectious disease, diabetes, hyperproliferative disorders and/or cancer. The improved LAMP Constructs allow for presentation of properly configured three dimensional epitopes for production of an immune response when administered to a subject. The improved LAMP Constructs can be multivalent molecules, and/or can be provided as part of a multivalent vaccine containing two or more LAMP Constructs. The improved LAMP Constructs as described herein can also be used to generate antibodies when administered to a non-human vertebrate.

公开(公告)号：US12129466B2

公开(公告)日：2024-10-29

申请号：US17576245

申请日：2022-01-14

申请人： Ionis Pharmaceuticals, Inc.

发明人： Paymaan Jafar-nejad

IPC分类号： C07H21/04 ,  C12N15/113

CPC分类号： C12N15/113 ,  C12N2310/111 ,  C12N2310/113 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/323 ,  C12N2310/3341 ,  C12N2310/341

摘要： Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of UBE3A-ATS, the endogenous antisense transcript of ubiquitin protein ligase E3A (UBE3A) in a cell or subject, and in certain instances increasing the expression of paternal UBE3A and the amount of UBE3A protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurogenetic disorder. Such symptoms and hallmarks include developmental delays, ataxia, speech impairment, sleep problems, seizures, and EEG abnormalities. Such neurogenetic disorders include Angelman Syndrome.

公开(公告)号：US12121562B2

公开(公告)日：2024-10-22

申请号：US18067940

申请日：2022-12-19

申请人： SANOFI

发明人： Gary J. Nabel ,  Chih-Jen Wei ,  Laura Nguyen ,  Kurt Swanson ,  Te-Hui Chou

IPC分类号： A61K39/245 ,  A61K38/16 ,  A61K39/12 ,  A61K47/00 ,  C07K14/05

CPC分类号： A61K38/162 ,  A61K47/00 ,  C07K14/05 ,  C07K2319/00

摘要： This disclosure relates to antigenic EBV polypeptides and their use in eliciting antibodies against EBV. Also disclosed are antigenic polypeptides comprising an EBV polypeptide and a ferritin protein.

公开(公告)号：US12115167B2

公开(公告)日：2024-10-15

申请号：US18444104

申请日：2024-02-16

申请人： Woolsey Pharmaceuticals, Inc.

发明人： Qicai Liu ,  Hemant N. Joshi ,  Thomas Macallister

IPC分类号： A61K31/551 ,  A61K9/00 ,  A61K9/16 ,  A61K47/58

CPC分类号： A61K31/551 ,  A61K9/0053 ,  A61K9/16 ,  A61K47/585

摘要： An oral pharmaceutical composition is provided that can comprise a rho kinase inhibitor, for example, fasudil, a pharmaceutically acceptable salt thereof, a hydrate thereof, a prodrug thereof, a substituted derivative thereof, or a metabolite thereof, or any combination thereof, the rho kinase inhibitor having a bitter taste; and an ion exchange resin. The ion exchange resin can partially or fully mask the bitter taste of the rho kinase inhibitor, making the composition more palatable. The composition can comprise a solid dosage form, and/or a liquid dosage form. The solid dosage form can comprise a powder, granules, a tablet, or a capsule, or any combination thereof. The composition can be present, for example, in a unit dose, in an amount sufficient to treat a neurodegenerative disease. A method of treating the neurodegenerative disease with the oral pharmaceutical composition is provided. The method can ameliorate a symptom of a neurodegenerative disease.

公开(公告)号：US12102720B2

公开(公告)日：2024-10-01

申请号：US17556316

申请日：2021-12-20

申请人： Translate Bio, Inc.

发明人： Braydon Charles Guild ,  Michael Heartlein ,  Frank DeRosa ,  Jerry Chi Zhang

IPC分类号： A61K9/19 ,  A61K9/127 ,  A61K31/7088 ,  A61K38/45 ,  A61K38/46 ,  A61K38/47 ,  A61K38/50 ,  C07C323/25 ,  C07C323/27 ,  C07C323/44 ,  C07D233/64 ,  C07J41/00 ,  C07J43/00 ,  C12N15/88 ,  A61K9/51

CPC分类号： A61K9/19 ,  A61K9/1271 ,  A61K9/1272 ,  A61K31/7088 ,  A61K38/45 ,  A61K38/465 ,  A61K38/47 ,  A61K38/50 ,  C07C323/25 ,  C07C323/27 ,  C07C323/44 ,  C07D233/64 ,  C07J41/0055 ,  C07J43/003 ,  C12N15/88 ,  A61K9/5123 ,  C12Y201/03003 ,  C12Y203/01 ,  C12Y207/08015 ,  C12Y301/06 ,  C12Y302/01022 ,  C12Y302/01035 ,  C12Y302/01046 ,  C12Y302/01076 ,  C12Y305/03001

摘要： Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g., as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent transfection of said target cells, and in certain embodiments are characterized as having one or more properties that afford such compounds advantages relative to other similarly classified lipids.