公开(公告)号：US11964052B2

公开(公告)日：2024-04-23

申请号：US18163117

申请日：2023-02-01

申请人： SUZHOU ABOGEN BIOSCIENCES CO., LTD.

发明人： Bo Ying ,  Xiulian Wang

IPC分类号： A61K9/16 ,  C07C217/28 ,  C07C219/06 ,  C07C229/16 ,  C07C229/22 ,  C07C235/06 ,  C07D207/24 ,  C07D295/13

CPC分类号： A61K9/1617 ,  C07C217/28 ,  C07C219/06 ,  C07C229/16 ,  C07C229/22 ,  C07C235/06 ,  C07D207/24 ,  C07D295/13 ,  C07C2601/04 ,  C07C2601/08 ,  C07C2601/14 ,  C07C2601/18

摘要： Provided herein are lipid compounds that can be used in combination with other lipid components, such as neutral lipids, cholesterol and polymer conjugated lipids, to form lipid nanoparticles for delivery of therapeutic agents (e.g., nucleic acid molecules) for therapeutic or prophylactic purposes, including vaccination. Also provided herein are lipid nanoparticle compositions comprising said lipid compounds.

公开(公告)号：US11840497B2

公开(公告)日：2023-12-12

申请号：US17249171

申请日：2021-02-22

申请人： PTC Therapeutics, Inc.

发明人： Orion D. Jankowski ,  Kieron E. Wesson ,  Paul Mollard ,  William D. Shrader

IPC分类号： C07C235/80 ,  C07C235/32 ,  C07C235/78 ,  C07C317/28 ,  C07D207/27 ,  C07D211/46 ,  C07D213/40 ,  C07D233/61 ,  C07D295/13 ,  C07D295/192 ,  C07D265/30 ,  C07D311/66 ,  C07D207/26 ,  C07D213/50 ,  C07D295/116 ,  C07D295/185 ,  A61P21/00 ,  A61P25/00 ,  A61P25/16 ,  A61P27/00 ,  C07D213/51

CPC分类号： C07C235/80 ,  A61P21/00 ,  A61P25/00 ,  A61P25/16 ,  A61P27/00 ,  C07C235/32 ,  C07C235/78 ,  C07C317/28 ,  C07D207/26 ,  C07D207/27 ,  C07D211/46 ,  C07D213/40 ,  C07D213/51 ,  C07D233/61 ,  C07D265/30 ,  C07D295/116 ,  C07D295/13 ,  C07D295/185 ,  C07D295/192 ,  C07D311/66 ,  C07C2601/02 ,  C07C2601/16

摘要： Methods of treating or suppressing mitochondrial diseases, such as Friedreich's ataxia (FRDA), Leber's Hereditary Optic Neuropathy (LHON), mitochondrial myopathy, encephalopathy, lactacidosis, and stroke (MELAS), Kearns-Sayre Syndrome (KSS), are disclosed, as well as compounds useful in the methods of the invention, such as 4-(p-quinolyl)-2-hydroxybutanamide derivatives. Methods and compounds useful in treating other disorders such as amyotrophic lateral sclerosis (ALS), Huntington's disease, Parkinson's disease, and pervasive developmental disorders such as autism are also disclosed. Energy biomarkers useful in assessing the metabolic state of a subject and the efficacy of treatment are also disclosed. Methods of modulating, normalizing, or enhancing energy biomarkers, as well as compounds useful for such methods, are also disclosed.

公开(公告)号：US11484530B2

公开(公告)日：2022-11-01

申请号：US16553219

申请日：2019-08-28

申请人： Bjoern Colin Kahrs

发明人： Bjoern Colin Kahrs

IPC分类号： C07D295/15 ,  C07D295/03 ,  C07D295/13 ,  C07D401/12 ,  C07D211/62 ,  A61K31/496 ,  A61K31/495 ,  A61K31/445 ,  A61K31/45 ,  A61P35/00 ,  A61K31/4439 ,  A61K31/05 ,  A61K31/12 ,  A61K31/16 ,  A61K31/19 ,  A61K31/216 ,  A61K31/26 ,  A61K31/385 ,  A61K31/47 ,  A61K31/225 ,  A61K31/426 ,  A61K9/00

摘要： The invention relates to pharmaceutical compositions comprising PPAR agonists and Nrf2 activators and methods of using combinations of PPAR agonists and Nrf2 activators for treating diseases such as psoriasis, asthma, multiple sclerosis, inflammatory bowel disease, and arthritis.

公开(公告)号：US11420934B2

公开(公告)日：2022-08-23

申请号：US16715711

申请日：2019-12-16

申请人： Salk Institute for Biological Studies ,  Mitobridge, Inc.

发明人： Ronald M. Evans ,  Michael Downes ,  Thomas J. Baiga ,  Joseph P. Noel ,  Emi Kanakubo Embler ,  Weiwei Fan ,  John F. W. Keana ,  Mark G. Bock ,  Authur F. Kluge ,  Mike A. Patane

IPC分类号： C07C233/87 ,  A61K31/381 ,  A61K31/5377 ,  A61K31/44 ,  A61K31/4025 ,  A61K31/422 ,  A61K31/341 ,  A61K31/427 ,  A61K31/164 ,  A61K31/4178 ,  C07C255/57 ,  C07C317/44 ,  C07C233/73 ,  C07C235/42 ,  C07C235/48 ,  C07C235/84 ,  C07C237/22 ,  C07C237/32 ,  C07D309/06 ,  C07D405/04 ,  C07D405/10 ,  C07D407/12 ,  C07D413/04 ,  C07D413/10 ,  C07D413/12 ,  C07D417/04 ,  C07D417/10 ,  C07D417/12 ,  C07D207/04 ,  C07D271/12 ,  C07D209/34 ,  C07D295/13 ,  C07D305/06 ,  C07D307/83 ,  C07D231/12 ,  C07D333/24 ,  C07D405/12 ,  C07D213/56 ,  C07D307/54 ,  C07C237/48 ,  C07C259/06

摘要： Provided herein are compounds and compositions useful in increasing PPARδ activity. The compounds and compositions provided herein are useful for the treatment of PPARδ related diseases (e.g., muscular diseases, vascular disease, demyelinating disease, and metabolic diseases).

公开(公告)号：US20220249693A1

公开(公告)日：2022-08-11

申请号：US17579115

申请日：2022-01-19

申请人： Beam Therapeutics Inc.

发明人： Cory Dane Sago ,  Gregory Lawrence Hamilton

IPC分类号： A61K47/69 ,  C07C219/06 ,  C07D295/13 ,  A61K47/24 ,  A61K47/26 ,  A61K31/7105 ,  A61K31/711 ,  A61P1/16

摘要： The present disclosure describes compositions, preparations, nanoparticles (such as lipid nanoparticles), and/or nanomaterials and methods of their use.

公开(公告)号：US20220218622A1

公开(公告)日：2022-07-14

申请号：US17500486

申请日：2021-10-13

申请人： George Mason Research Foundation, Inc.

发明人： Michael Daro Buschmann ,  Mikell Paige ,  Suman Alishetty ,  Manuel Carrasco ,  Mohamad Gabriel Alameh ,  Drew Weissman

IPC分类号： A61K9/51 ,  C07C229/16 ,  C07D295/13 ,  C07D207/09 ,  C07D211/26 ,  A61K31/7105 ,  A61K47/28

摘要： The invention encompasses novel ionizable lipids compounds and their use in lipid nanoparticles delivery systems that are useful in the delivery of nucleic acids to a mammalian subject that can be included for use, for example, as cancer vaccines, gene editing therapeutics, delivery of nucleic acid (e.g., mRNA) encoding antibodies, vaccines for infectious disease, and protein replacement therapeutics. Additionally, the invention encompasses compositions and therapeutics comprising the ionizable lipids in the lipid nanoparticles and the use of the composition and therapeutics for the preparation of a pharmaceutical composition, especially a vaccine, (e.g., for use in the prophylaxis or treatment of infectious diseases, tumor or cancer diseases, rare diseases, allergies, or autoimmune diseases). The invention encompasses methods of treatment or prophylaxis of the aforementioned diseases.

公开(公告)号：US11293060B2

公开(公告)日：2022-04-05

申请号：US16596539

申请日：2019-10-08

申请人： Roche Sequencing Solutions, Inc.

发明人： Frank Bergmann ,  Ian Quang Chau ,  Peter Crisalli ,  Hannah Marie Kallewaard-Lum ,  Hannes Kuchelmeister ,  Sebastian Johannes Pomplun ,  Hasham Saqib ,  Christian Wellner

IPC分类号： C12Q1/68 ,  C12Q1/6869 ,  C07D295/13 ,  C07D487/04 ,  C07H21/02 ,  C07H21/04 ,  A61K47/68

摘要： The present invention relates to improving the processing rate of a sequencing reaction, for example in a nanopore sequencing reaction, by means of using improved nucleoside-tags. The tags are linked to the nucleoside phosphate via a Pictet Spengler reaction. Exemplary sequencing reactions that are improved by the present methods include nanopore-based nucleic acid sequencing-by-synthesis reactions.

公开(公告)号：US11260053B2

公开(公告)日：2022-03-01

申请号：US16276540

申请日：2019-02-14

申请人： Satiogen Pharmaceuticals, Inc.

发明人： Bronislava Gedulin ,  Andrew A. Young ,  Howard E. Greene

IPC分类号： A61K31/575 ,  A61K31/496 ,  A61K9/00 ,  A61K9/02 ,  A61K9/28 ,  A61K31/155 ,  A61K31/16 ,  A61K31/18 ,  A61K31/19 ,  A61K31/191 ,  A61K31/216 ,  A61K31/38 ,  A61K31/40 ,  A61K31/42 ,  A61K31/452 ,  A61K31/454 ,  A61K31/495 ,  A61K31/4985 ,  A61K31/4995 ,  A61K31/513 ,  A61K31/5377 ,  A61K31/55 ,  A61K31/554 ,  A61K31/655 ,  A61K31/7042 ,  A61K45/06 ,  C07C279/14 ,  C07D295/13 ,  C07D401/12 ,  C07D487/08

摘要： Provided herein are methods of utilizing bile acid transport inhibitors and/or enteroendocrine peptide enhancing agents for the treatment of obesity, diabetes, and inflammatory gastrointestinal conditions.

公开(公告)号：US11236386B2

公开(公告)日：2022-02-01

申请号：US16598685

申请日：2019-10-10

申请人： Roche Diagnostics Operations, Inc.

发明人： Frank Bergmann ,  Tobias Oelschlaegel ,  Sebastian Johannes Pomplun

IPC分类号： C12Q1/6869 ,  C07D295/13 ,  C07D487/04 ,  C07H21/02 ,  C07H21/04 ,  A61K47/68

摘要： The present description relates to a method for binding to a target molecule having an aldehyde compound derived from N-(2-aminoethyl)pyrrole, which compound also has a moiety of interest, to compounds (conjugates) obtained by this method, having both the target molecule and the moiety of interest and to novel substances derived from N-(2-aminoethyl)pyrrole. In one embodiment, the compound has the formula of Formula II wherein R1, R2 and R3 independently are H, substituted or unsubstituted alkyl, substituted or unsubstituted alkenyl, substituted or unsubstituted heteroalkyl, substituted or unsubstituted aryl, or substituted or unsubstituted heteroaryl, wherein R4, R5, R6, R7 and R8 independently are H, substituted or unsubstituted alkyl, substituted or unsubstituted heteroalkyl, substituted or unsubstituted aryl, substituted or unsubstituted heteroaryl, or -LM, wherein L is absent or is a linker and M is a moiety of interest selected from a nucleotide, an oligonucleotide, a peptide, a label, a cytotoxic agent, a partner of a binding pair and a functional group, wherein two of R4, R5, R6, R7, and R8 optionally are linked to form a substituted or unsubstituted cycloalkyl or a substituted or unsubstituted heterocycloalkyl, and T is a target molecule selected from the group consisting of a solid phase, a polypeptide, a protein, a carbohydrate, a nucleotide and a nucleic acid, with the proviso that at least one of R4, R5, R6, R7 or R8 is -LM.

公开(公告)号：US11168051B2

公开(公告)日：2021-11-09

申请号：US16265234

申请日：2019-02-01

申请人： Acuitas Therapeutics, Inc.

发明人： Xinyao Du ,  Steven M. Ansell

IPC分类号： C07C233/36 ,  C07D295/13 ,  C07C211/09 ,  C07C235/10 ,  A61K9/51 ,  A61K31/713 ,  A61K9/14 ,  A61K31/7105 ,  A61K47/18 ,  A61K47/24 ,  C07C233/38 ,  C07D207/09 ,  C12N15/113

摘要： Compounds are provided having the following structure: or a pharmaceutically acceptable salt, tautomer or stereoisomer thereof, wherein R1a, R1b, R2a, R2b, R3a, R3b, R4a, R4b, R5, R6, R7, R8, R9, L1, L2, G1, G2, G3, a, b, c and d are as defined herein. Use of the compounds as a component of lipid nanoparticle formulations for delivery of a therapeutic agent, compositions comprising the compounds and methods for their use and preparation are also provided.