公开(公告)号：US12233140B2

公开(公告)日：2025-02-25

申请号：US18514738

申请日：2023-11-20

Applicant: Yale University

Inventor： Yang Deng ,  Asiri Ediriwickrema ,  W. Mark Saltzman

IPC: A61K8/02 ,  A61K8/90 ,  A61K9/00 ,  A61K9/16 ,  A61K9/50 ,  A61K9/51 ,  A61K31/4178 ,  A61K31/427 ,  A61K31/4745 ,  A61K39/00 ,  A61K47/34 ,  A61Q17/04 ,  C07C59/06 ,  C07C59/08 ,  C08G83/00

Abstract: Core-shell particles and methods of making and using thereof are described herein. The core is formed of or contains one or more hydrophobic materials or more hydrophobic materials. The shell is formed of or contains hyperbranched polyglycerol (HPG). The HPG coating can be modified to adjust the properties of the particles. Unmodified HPG coatings impart stealth properties to the particles which resist non-specific protein absorption and increase circulation in the blood. The hydroxyl groups on the HPG coating can be chemically modified to form functional groups that react with functional groups and adhere the particles to tissue, cells, or extracellular materials, such as proteins.

公开(公告)号：US12226400B2

公开(公告)日：2025-02-18

申请号：US17264982

申请日：2019-07-31

Applicant: UNIVERSITY OF PITTSBURGH-OF THE COMMONWEALTH SYSTEM OF HIGHER EDUCATION

Inventor： Elias Aizenman ,  Carlos Jaime Camacho

IPC: A61K31/4155 ,  A61K31/4178 ,  A61K31/428 ,  A61K31/4409 ,  A61K31/506 ,  A61K31/53 ,  A61P25/28

Abstract: Disclosed herein are small molecule compounds capable of disrupting Kv2.1-syntaxin binding. The compounds are useful for treating a variety of neurological disorders, diseases, and injuries.

公开(公告)号：US12213988B2

公开(公告)日：2025-02-04

申请号：US17347230

申请日：2021-06-14

Applicant: ASTRAZENECA AB

Inventor： Anna Maria Langkilde

IPC: A61K31/7004 ,  A61K9/00 ,  A61K31/351 ,  A61K31/401 ,  A61K31/41 ,  A61K31/4178 ,  A61K45/06 ,  A61P13/12

Abstract: The present disclosure is directed to methods of treating patients with chronic kidney disease (CKD), with and without Type 2 diabetes, with an SGLT2 inhibitor, such as dapagliflozin.

公开(公告)号：US12213961B2

公开(公告)日：2025-02-04

申请号：US18513181

申请日：2023-11-17

Applicant: VICORE PHARMA AB

Inventor： Carl-Johan Dalsgaard ,  Rohit Batta

IPC: A61K31/4178 ,  A61K9/48 ,  A61P1/00 ,  A61P11/00

Abstract: According to the invention there is provided a method of improving (e.g. restoring) function in a patient having idiopathic pulmonary fibrosis, which method comprises perorally administering a therapeutically-effective amount of N-butyloxycarbonyl-3-(4-imidazol-1-ylmethylphenyl)-5-iso-butylthiophene-2-sulfonamide (C21), or a pharmaceutically acceptable salt thereof, to said patient. Said treatment is capable of treating said IPF in a therapeutic, including a curative, fashion.

公开(公告)号：US20250025439A1

公开(公告)日：2025-01-23

申请号：US18708662

申请日：2022-11-30

Applicant: INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE ,  CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE ,  UNIVERSITE COTE D'AZUR ,  CENTRE HOSPITALIER RÉGIONAL ,  ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES (A.F.M.)

Inventor： Véronique PAQUIS-FLUCKLINGER ,  Stéphane AZOULAY ,  Marc-Alexandre D'ELIA ,  Baptiste ROPERT

IPC: A61K31/341 ,  A61K31/4178 ,  A61K31/422 ,  A61K31/5377 ,  A61P21/00 ,  A61P25/28

Abstract: Inventors have succeeded in identifying compounds comprising a nitrofuranyl moiety having the advantage of compensating mitochondrial dysfunction associated with both primary and secondary MICOS stability defects. These compounds are thus useful in preventing mitochondrial dysfunction associated with CHCHD10 mutations. Accordingly, the present invention relates to the use of nifuroxazide and its derivatives in the treatment of disorders associated with the disorganisation of the MICOS complex (MItochondrial contact site and Cristae Organizing System) and mitochondrial dysfunction such as mitochondrial disorders, in particular myopathy and cardiomyopathy, and neurodegenerative diseases, in particular motor neuron disease (including amyotrophic lateral sclerosis (ALS)) and frontotemporal dementia (FTD).

公开(公告)号：US12201616B2

公开(公告)日：2025-01-21

申请号：US17877564

申请日：2022-07-29

Applicant: Opus Genetics, Inc.

Inventor： Mina Sooch ,  Alan R. Meyer ,  Konstantinos Charizanis ,  Bernhard Hoffmann ,  William H. Pitlick

IPC: A61K31/417 ,  A61K9/00 ,  A61K9/08 ,  A61K31/4178 ,  A61K47/12 ,  A61K47/26 ,  A61P27/08 ,  A61P27/10

Abstract: The invention provides methods, compositions, and kits containing an alpha-adrenergic antagonist, such as phentolamine, for use in monotherapy or as part of a combination therapy to treat patients suffering from presbyopia, mydriasis, and/or other ocular disorders.

公开(公告)号：US12201615B2

公开(公告)日：2025-01-21

申请号：US17877558

申请日：2022-07-29

Applicant: Opus Genetics, Inc.

Inventor： Mina Sooch ,  Alan R. Meyer ,  Konstantinos Charizanis ,  Bernhard Hoffmann ,  William H. Pitlick

IPC: A61K31/417 ,  A61K9/00 ,  A61K9/08 ,  A61K31/4178 ,  A61K47/12 ,  A61K47/26 ,  A61P27/08 ,  A61P27/10

Abstract: The invention provides methods, compositions, and kits containing an alpha-adrenergic antagonist, such as phentolamine, for use in monotherapy or as part of a combination therapy to treat patients suffering from presbyopia, mydriasis, and/or other ocular disorders.

公开(公告)号：US12195450B2

公开(公告)日：2025-01-14

申请号：US17422670

申请日：2020-01-29

Applicant: The Johns Hopkins University

Inventor： Takashi Tsukamoto ,  Niyada Hin ,  Ondrej Stepanek ,  Barbara Slusher ,  Camilo Rojas ,  Ajit G. Thomas

IPC: C07D409/04 ,  A61K31/4164 ,  A61K31/4178 ,  A61K31/4184 ,  A61K31/437 ,  A61K31/4439 ,  A61P25/00 ,  A61P25/28 ,  C07D233/64 ,  C07D235/18 ,  C07D401/04 ,  C07D405/14 ,  C07D409/14 ,  C07D471/04

Abstract: Small molecule inhibitors of neutral sphingomyelinase 2 (nSMase2) and their use for treating neurodegenerative diseases, such as, neurodegenerative diseases associated with high levels of ceramide, including, but not limited to Alzheimer's disease (AD), HIV-associated neurocognitive disorder (HAND), multiple sclerosis (MS), and amyotrophic lateral sclerosis (ALS), and, in other aspects, for treating cancer or HIV-1, are provided.

公开(公告)号：US20250009717A1

公开(公告)日：2025-01-09

申请号：US18672743

申请日：2024-05-23

Applicant: Stealth BioTherapeutics Inc.

Inventor： Dennis Keefe ,  Guozhu Zheng ,  Vania Broccoli

IPC: A61K31/4245 ,  A61K31/135 ,  A61K31/197 ,  A61K31/4152 ,  A61K31/4178 ,  A61K31/4184 ,  A61K31/5513 ,  A61K38/30 ,  A61P25/28

Abstract: The present disclosure provides novel methods for treating or preventing amyotrophic lateral sclerosis (ALS), methods for delaying the onset of neurological symptoms associated with ALS, increasing survival in subjects afflicted with ALS, and attenuating the decline of muscle strength associated with ALS in a subject in need thereof. The present disclosure also provides methods for treating or preventing α-synucleinopathy or TDP-43 proteinopathy. The methods comprise administering to the subject an effective amount of a mitochondria-targeting peptidomimetic compound, such as (R)-2-amino-N-((S)-1-(((S)-5-amino-1-(3-benzyl-1,2,4-oxadiazol-5-yl)pentyl)amino)-3-(4-hydroxy-2,6-dimethylphenyl)-1-oxopropan-2-yl)-5-guanidinopentanamide, or a pharmaceutically acceptable salt, stereoisomer, tautomer, hydrate, and/or solvate thereof.

公开(公告)号：US12178912B2

公开(公告)日：2024-12-31

申请号：US18074699

申请日：2022-12-05

Applicant: R.P. Scherer Technologies, LLC ,  UNIVERSITY OF MISSISSIPPI

Inventor： Sampada Bhaskar Upadhye ,  Ronald S. Vladyka ,  Michael Andrew Repka ,  Jun-Bom Park ,  Roshan Vijay Tiwari ,  Hemlata Gunga Patil ,  Joseph Thomas Morott, Jr. ,  Wenli Lu

IPC: A61K9/16 ,  A61K9/00 ,  A61K9/20 ,  A61K31/4178 ,  A61K31/519 ,  B01J2/10

Abstract: A dry granulation process using a twin-screw extruder for granulating a powder mixture which includes at least one active ingredient and at least one carrier. The process includes steps of kneading the powder mixture in the screw barrel of the twin-screw extruder at a barrel temperature below a melting point of the at least one active ingredient and a melting point or a glass transition temperature of the at least one carrier to provide a kneaded powder mixture, and extruding the kneaded powder mixture to form granules. Granules and tablets produced using the dry granulation process in the twin-screw extruder are also provided.