公开(公告)号：US20250064792A1

公开(公告)日：2025-02-27

申请号：US18822966

申请日：2024-09-03

Applicant: The Research Foundation for The State University of New York

Inventor： Addolorata PISCONTI

IPC: A61K31/444 ,  A61K31/00 ,  A61K31/222 ,  A61K31/26 ,  A61K31/505 ,  A61K38/00 ,  A61K45/06 ,  A61P21/00 ,  A61P25/14

Abstract: The invention provides anelastase inhibitor for use in the promotion of muscle regeneration in the treatment of a myopa-thy, as well as a method for promoting muscle regeneration in a subject with a myopathy, the method comprising providing the subject with a therapeutically effective amount of an elastase inhibitor. Further provided is a pharmaceutical composition comprising anelastase inhibitor for use in the promotion of muscle regeneration in the treatment of a myopathy. Elastase inhibitors may have a protective effect on muscle progenitor cells and their regenerative potential, which aids muscle cell regeneration. By protecting regenerative potential of muscle progenitor cells, elastase inhibitors enable or enhance the grown of new or existing muscle fibres.

公开(公告)号：US20240415943A1

公开(公告)日：2024-12-19

申请号：US18624753

申请日：2024-04-02

Applicant: Voyager Therapeutics, Inc.

Inventor： Robert Kotin ,  Adrian Philip Kells ,  Bernard Ravina

IPC: A61K38/51 ,  A61K35/76 ,  A61K48/00 ,  A61P25/14 ,  C12N7/00 ,  C12N9/88 ,  C12N15/86

Abstract: The disclosure relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides encoding AADC for the treatment of Parkinson's Disease.

公开(公告)号：US20240390327A1

公开(公告)日：2024-11-28

申请号：US18278998

申请日：2022-02-26

Applicant: Johns Hopkins University

Inventor： Shenandoah Robinson ,  Lauren Jantzie

IPC: A61K31/4045 ,  A61K31/472 ,  A61K38/18 ,  A61P25/00 ,  A61P25/14 ,  A61P25/28

Abstract: In one aspect, methods and compositions are provided for treating a subject suffering from or susceptible to prenatal opioid exposure. In a further aspect, methods and compositions are provided for treating a subject suffering from or susceptible traumatic brain injury, intracranial hemorrhage, acquired hydrocephalus, demyelinating diseases including multiple sclerosis, neurological symptoms from systemic lupus erythematosus, and neurodegenerative diseases. In an additional aspect, methods and compositions are provided for treating subject having a COVID-19 infection, exhibiting symptoms of a COVID-19 infection, having suspected exposure to COVID-19, or suspected of suffering from long COVID or post-acute sequelae of COVID-19 (PASC).

公开(公告)号：US20240360187A1

公开(公告)日：2024-10-31

申请号：US18545392

申请日：2023-12-19

Applicant: UNIVERSITÄT BERN

Inventor： Achim STOCKER

IPC: C07K14/47 ,  A61K9/14 ,  A61K9/51 ,  A61K38/00 ,  A61K38/17 ,  A61K47/50 ,  A61P3/02 ,  A61P19/02 ,  A61P21/00 ,  A61P25/14 ,  A61P29/00 ,  B82Y5/00 ,  B82Y40/00

CPC classification number: C07K14/47 ,  A61K9/146 ,  A61K9/51 ,  A61K38/1709 ,  A61K47/50 ,  A61P3/02 ,  A61P19/02 ,  A61P21/00 ,  A61P25/14 ,  A61P29/00 ,  B82Y5/00 ,  B82Y40/00 ,  A61K38/00

Abstract: The present invention relates to a nanosphere comprising an equal number of a human SEC14-like protein and a cognate ligand of said SEC14-like protein as well as to methods of producing the same and uses of said nanospheres.

公开(公告)号：US12049448B2

公开(公告)日：2024-07-30

申请号：US17871065

申请日：2022-07-22

Applicant: PRAXIS PRECISION MEDICINES, INC.

Inventor： Kiran Reddy ,  Margaret S. Lee ,  Gabriel Maurice Belfort ,  Sapna Makhija Garad ,  Mahesh Padval ,  Randall Wagner ,  Marion Wittmann

IPC: C07D211/34 ,  A61K9/00 ,  A61K9/20 ,  A61K9/48 ,  A61K31/445 ,  A61K47/38 ,  A61P25/14 ,  C07D211/26

CPC classification number: C07D211/34 ,  A61K9/0053 ,  A61K9/2009 ,  A61K9/2013 ,  A61K9/2018 ,  A61K9/2054 ,  A61K9/4825 ,  A61K9/4858 ,  A61K9/4866 ,  A61K31/445 ,  A61K47/38 ,  A61P25/14 ,  C07D211/26 ,  C07B2200/13

Abstract: Described herein, in part, are dosage forms and compositions useful for preventing and/or treating a disease or condition relating to aberrant function of a T-type calcium channel, such as epilepsy and epilepsy syndromes (e.g., absence seizures, juvenile myoclonic epilepsy, or a genetic epilepsy), tremor (e.g., essential tremor), and psychiatric disorder (e.g., mood disorders (e.g., major depressive disorder)). The present invention further comprises methods for modulating the function of a T-type calcium channel.

公开(公告)号：US20240247272A1

公开(公告)日：2024-07-25

申请号：US18444881

申请日：2024-02-19

Applicant: KOREA ADVANCED INSTITUTE OF SCIENCE AND TECHNOLOGY

Inventor： Dae Soo KIM ,  Jin Kuk KIM ,  Ye Won LEE ,  Eun Ji JUNG ,  Min Sung PARK ,  Sin Jeong LEE ,  Su Jin CHAE

IPC: C12N15/113 ,  A61P25/08 ,  A61P25/14 ,  A61P25/22 ,  A61P25/24 ,  A61P25/26

CPC classification number: C12N15/1138 ,  A61P25/08 ,  A61P25/14 ,  A61P25/22 ,  A61P25/24 ,  A61P25/26 ,  C12N2310/11 ,  C12N2310/3125 ,  C12N2310/313 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/341

Abstract: The present invention provides antisense oligonucleotides for the treatment of various T-type calcium channel α1G subunit-related disorders, and more specifically, antisense oligonucleotides for inhibiting expression of the gene encoding Cav3.1, targeting SEQ ID NO: 1, for the treatment of various neuropsychiatric disorders, including Parkinson's disease, epilepsy, essential tremor, depression, anxiety disorders, and unconsciousness.

公开(公告)号：US12042507B2

公开(公告)日：2024-07-23

申请号：US17181921

申请日：2021-02-22

Applicant: University of Georgia Research Foundation, Inc.

Inventor： Hang Yin ,  Liwei Xie ,  Amelia Yao-Ye Yin

IPC: A61K31/7105 ,  A61K9/00 ,  A61K31/015 ,  A61K31/277 ,  A61P21/04 ,  A61P25/14 ,  C12Q1/6876

CPC classification number: A61K31/7105 ,  A61K9/0019 ,  A61K31/015 ,  A61K31/277 ,  A61P21/04 ,  A61P25/14 ,  C12Q1/6876

Abstract: Compositions and methods for modulating HIF-2α to meditate of hypoxia signaling in satellite cells and applications thereof for improving skeletal muscle generation and repair are provided. For example, methods of enhancing, increasing, accelerating or/and otherwise improving skeletal muscle generation or regeneration in a subject in need thereof are disclosed. In some embodiments, the methods include administering the subject an effective amount of HIF-2α inhibitor. The HIF-2α inhibitor can be effective to, for example, increase muscle satellite cell proliferation, differentiation, or a combination thereof in a subject. Composition and methods for improving respiration, and reducing or preventing the development or progression of fibrosis are also provided. The disclosed compositions and methods are particularly useful for treating muscular dystrophies, myopathies, and other muscle-related diseases and disorders.

公开(公告)号：US12024509B2

公开(公告)日：2024-07-02

申请号：US16814328

申请日：2020-03-10

Applicant: C4X Discovery Limited

Inventor： Emma Louise Blaney ,  Barrie Phillip Martin ,  Thorsten Nowak ,  Martin John Watson

IPC: C07D471/04 ,  A61K31/4192 ,  A61K31/435 ,  A61K31/4439 ,  A61K31/47 ,  A61P3/04 ,  A61P19/10 ,  A61P25/00 ,  A61P25/04 ,  A61P25/06 ,  A61P25/14 ,  A61P25/16 ,  A61P25/18 ,  A61P25/20 ,  A61P25/22 ,  A61P25/24 ,  A61P25/28 ,  A61P25/30 ,  A61P25/32 ,  A61P25/34 ,  A61P25/36 ,  A61P43/00 ,  C07C233/69 ,  C07C233/78 ,  C07C275/28 ,  C07C275/40 ,  C07D213/63 ,  C07D213/64 ,  C07D213/74 ,  C07D231/38 ,  C07D235/26 ,  C07D235/30 ,  C07D239/34 ,  C07D239/42 ,  C07D239/80 ,  C07D239/84 ,  C07D401/04 ,  C07D401/12 ,  C07D401/14 ,  C07D403/12 ,  C07D413/12 ,  C07D413/14 ,  C07D417/12 ,  C07D417/14

CPC classification number: C07D471/04 ,  C07C233/69 ,  C07C233/78 ,  C07C275/28 ,  C07C275/40 ,  C07D213/63 ,  C07D213/64 ,  C07D213/74 ,  C07D231/38 ,  C07D235/26 ,  C07D235/30 ,  C07D239/34 ,  C07D239/42 ,  C07D239/80 ,  C07D239/84 ,  C07D401/12 ,  C07D401/14 ,  C07D403/12 ,  C07D413/12 ,  C07D413/14 ,  C07D417/12 ,  C07D417/14

Abstract: The present invention relates to compounds that are inhibitors of the orexin-1 receptor. The compounds have the structural formula I defined herein. The present invention also relates to processes for the preparation of these compounds, to pharmaceutical compositions comprising them, and to their use in the treatment of diseases or disorders associated with orexin-1 receptor activity.

公开(公告)号：US20240099987A1

公开(公告)日：2024-03-28

申请号：US18380680

申请日：2023-10-17

Applicant: BUZZELET DEVELOPMENT AND TECHNOLOGIES LTD.

Inventor： AHARON M. EYAL ,  NOA RAZ

IPC: A61K31/015 ,  A61K31/01 ,  A61K31/05 ,  A61K31/352 ,  A61K47/06 ,  A61K47/10 ,  A61K47/38 ,  A61P11/06 ,  A61P21/04 ,  A61P25/00 ,  A61P25/08 ,  A61P25/14 ,  A61P25/16 ,  A61P25/18 ,  A61P25/22 ,  A61P25/24 ,  A61P25/28 ,  A61P25/30 ,  A61P35/00

CPC classification number: A61K31/015 ,  A61K31/01 ,  A61K31/05 ,  A61K31/352 ,  A61K47/06 ,  A61K47/10 ,  A61K47/38 ,  A61P11/06 ,  A61P21/04 ,  A61P25/00 ,  A61P25/08 ,  A61P25/14 ,  A61P25/16 ,  A61P25/18 ,  A61P25/22 ,  A61P25/24 ,  A61P25/28 ,  A61P25/30 ,  A61P35/00

Abstract: A composition of at least one cannabinoid, at least one terpene of pinene, limonene, linalool, caryophyllene, myrcene, humulene, terpineol, nerolidol, bisabolol, geraniol, borneol, eucalyptol in a concentration of at least about 20 wt % of terpenes in the composition, or at least two terpenes in a combined amount of at least about 40 wt % of terpenes in the composition, at least 5% by weight of a non-cannabinoid, non-terpene carrier, less than 5% by weight glycol, and less than 20% by weight water, and where the non-cannabinoid, non-terpene carrier comprises a) at least 5% by weight cellulose and the total terpenes to total cannabinoids weight/weight ratio in the composition is from 0.1 to 1.0, or b) less than 5% by weight cellulose and the total terpenes to total cannabinoids weight/weight ratio is from 0.05 to 1.0, obtained by providing and blending the cannabinoid and terpene in amounts configured to provide the ratio.

公开(公告)号：US20240000795A1

公开(公告)日：2024-01-04

申请号：US17945865

申请日：2022-09-15

Applicant: Terran Biosciences Inc.

Inventor： Sam CLARK

IPC: A61K31/5513 ,  A61K31/519 ,  A61K31/554 ,  A61K45/06 ,  A61P25/14

CPC classification number: A61K31/5513 ,  A61K31/519 ,  A61K31/554 ,  A61K45/06 ,  A61P25/14

Abstract: Provided herein are compositions relating to psychedelics and serotonin receptor modulators. Further provided herein are methods of suppressing or halting hallucinogenic effects of a psychedelic and methods of treating a disease or disorder (e.g., depression or diseases or disorders related to depression) comprising administering psychedelics and serotonin receptor modulators.