公开(公告)号：US20240360153A1

公开(公告)日：2024-10-31

申请号：US18628171

申请日：2024-04-05

Applicant: Regeneron Pharmaceuticals, Inc.

Inventor： David Glass ,  Joseph P. Vacca ,  Tea Shavlakadze ,  Chester A. Metcalf, III ,  William Olson

IPC: C07D498/18 ,  A61K31/439 ,  A61K31/444 ,  A61K31/4709 ,  A61K31/497 ,  A61K39/395

CPC classification number: C07D498/18 ,  A61K31/439 ,  A61K31/444 ,  A61K31/4709 ,  A61K31/497 ,  A61K39/3955

Abstract: Compounds and pharmaceutical compositions for use in the treatment of a disorder or disease mediated by the mTOR pathway through more selective binding to FKBP12 of the group of FK506 binding proteins (FKBPs) are disclosed. With an unusual pharmacokinetic profile and an enhanced pharmacodynamic selectivity among target FKBPs, those compounds are useful for the treatment of age- or aging-related diseases, diabetes, cancers, as well as inflammation-associated disorders.

公开(公告)号：US12128036B2

公开(公告)日：2024-10-29

申请号：US18239087

申请日：2023-08-28

Applicant: Lenz Therapeutics Operations, Inc.

Inventor： Gerald Horn

IPC: A61K31/439 ,  A61K47/02 ,  A61K47/12 ,  A61K47/14 ,  A61K47/18 ,  A61K47/20 ,  A61K47/22 ,  A61K47/26 ,  A61K47/38 ,  A61J1/16

CPC classification number: A61K31/439 ,  A61K47/02 ,  A61K47/12 ,  A61K47/14 ,  A61K47/183 ,  A61K47/186 ,  A61K47/20 ,  A61K47/22 ,  A61K47/26 ,  A61K47/38 ,  A61J1/165

Abstract: The present invention is related to methods of stabilizing an ophthalmic drug by adding a surfactant and a viscosity enhancer to the ophthalmic drug to create a composition wherein the composition has a viscosity of about 25 centipoise or less at a shear rate of 1/1000 per second at 25 degrees Celsius and a viscosity of about 70 centipoise or more at shear rate of 1 per second at 25 degrees Celsius, filling the composition into a container; and storing the container at a temperature from about 2 degrees Celsius to about 25 degrees Celsius. The present invention is further directed to a container prepared by the methods of the present invention.

公开(公告)号：US20240343692A1

公开(公告)日：2024-10-17

申请号：US18682031

申请日：2022-06-21

Applicant: University of Pittsburgh - Of the Commonwealth System of Higher Education

Inventor： Peter Wipf ,  Nikhil R. Tasker ,  Ethan J. Pazur

IPC: C07D221/22 ,  A61K31/4045 ,  A61K31/437 ,  A61K31/439 ,  C07D221/18

CPC classification number: C07D221/22 ,  A61K31/4045 ,  A61K31/437 ,  A61K31/439 ,  C07D221/18

Abstract: A compound, or a pharmaceutically acceptable salt thereof, of formula I:






wherein R1, R8, R9, R31, R32, R33, and R34 are each independently H, C1-C6 alkyl, or substituted C1-C6 alkyl; and
R2, R4, R5, R6, and R7 are each independently H, F, Cl, Br, —OR1—NR8R9, C1-C6 alkyl, or substituted C1-C6 alkyl.

公开(公告)号：US20240307511A1

公开(公告)日：2024-09-19

申请号：US18538866

申请日：2023-12-13

Applicant: The Trustees of the University of Pennsylvania

Inventor： Christian Hinderer ,  James M. Wilson

IPC: A61K38/47 ,  A61K9/00 ,  A61K31/343 ,  A61K31/439 ,  A61K35/76 ,  A61K45/06 ,  A61K48/00 ,  A61P3/00 ,  C12N7/00 ,  C12N9/24 ,  C12N15/86

CPC classification number: A61K38/47 ,  A61K9/0019 ,  A61K31/343 ,  A61K31/439 ,  A61K35/76 ,  A61K45/06 ,  A61P3/00 ,  C12N7/00 ,  C12N9/2402 ,  C12N15/86 ,  C12Y302/01076 ,  A61K48/00 ,  A61K48/0083 ,  C12N2750/14121 ,  C12N2750/14132 ,  C12N2750/14143

Abstract: A co-therapeutic regimen comprising AAV9-mediated intrathecal/intracisternal and/or systemic delivery of an expression cassette containing a hIDUA gene and two or more immunosuppressants is provided herein. Also provided are methods useful for treating hIDUA deficiency (MPSI) and the symptoms associated with Hurler, Hurler-Scheie and Scheie syndromes.

公开(公告)号：US20240307334A1

公开(公告)日：2024-09-19

申请号：US18407998

申请日：2024-01-09

Applicant: Lieber Institute, Inc. ,  Oregon Health & Science University

Inventor： Brady Maher ,  Joseph Bohlen ,  Thomas Scanlan

IPC: A61K31/192 ,  A61K31/137 ,  A61K31/165 ,  A61K31/216 ,  A61K31/222 ,  A61K31/40 ,  A61K31/4025 ,  A61K31/439 ,  A61K31/445 ,  A61K31/4453 ,  A61K31/46 ,  A61K31/5386 ,  A61P25/28

CPC classification number: A61K31/192 ,  A61K31/137 ,  A61K31/165 ,  A61K31/216 ,  A61K31/222 ,  A61K31/40 ,  A61K31/4025 ,  A61K31/439 ,  A61K31/445 ,  A61K31/4453 ,  A61K31/46 ,  A61K31/5386 ,  A61P25/28

Abstract: The use of sobetirome or a prodrug or derivative thereof, including Sob-AM2, for treating a disease, disorder, or condition associated with or suspected of being associated with dysmyelination, such as Pitt-Hopkins Syndrome, is disclosed.

公开(公告)号：US20240299492A1

公开(公告)日：2024-09-12

申请号：US18279451

申请日：2022-03-04

Applicant: Shattuck Labs, Inc.

Inventor： Taylor SCHREIBER ,  Suresh DE SILVA ,  George FROMM

IPC: A61K38/17 ,  A61K31/282 ,  A61K31/337 ,  A61K31/439 ,  A61K31/454 ,  A61K31/4745 ,  A61K31/513 ,  A61K31/519 ,  A61K31/635 ,  A61K31/69 ,  A61K31/704 ,  A61K31/706 ,  A61K31/7068 ,  A61K33/243 ,  A61K38/19 ,  A61K39/395 ,  A61K45/06 ,  A61P35/00

CPC classification number: A61K38/1774 ,  A61K31/282 ,  A61K31/337 ,  A61K31/439 ,  A61K31/454 ,  A61K31/4745 ,  A61K31/513 ,  A61K31/519 ,  A61K31/635 ,  A61K31/69 ,  A61K31/704 ,  A61K31/706 ,  A61K31/7068 ,  A61K33/243 ,  A61K38/191 ,  A61K39/3955 ,  A61K45/06 ,  A61P35/00

Abstract: The present disclosure relates to, inter alia, combinations of compositions which include chimeric proteins that find use in methods for treating disease, such as immunotherapies for cancer.

公开(公告)号：US12076405B2

公开(公告)日：2024-09-03

申请号：US17164446

申请日：2021-02-01

Applicant: C4 Therapeutics, Inc.

Inventor： Andrew J. Phillips ,  Christopher G. Nasveschuk ,  James A. Henderson ,  Yanke Liang ,  Mark E. Fitzgerald ,  Minsheng He ,  Ryan E. Michael

IPC: A61K47/54 ,  A61K31/4035 ,  A61K31/438 ,  A61K31/439 ,  A61K31/454 ,  A61K31/547 ,  A61K31/55 ,  C07D401/04 ,  C07D403/04

CPC classification number: A61K47/545 ,  A61K31/4035 ,  A61K31/438 ,  A61K31/439 ,  A61K31/454 ,  A61K31/547 ,  A61K31/55 ,  A61K47/554 ,  C07D403/04

Abstract: This invention provides heterocyclic compounds that bind to E3 Ubiquitin Ligase (typically through cereblon) (“Degrons”), which can be used as is or linked to a Targeting Ligand for a selected Target Protein for therapeutic purposes and methods of use and compositions thereof as well as methods for their preparation.

公开(公告)号：US20240287108A1

公开(公告)日：2024-08-29

申请号：US18566922

申请日：2022-06-07

Applicant: Rana RAIS ,  The Johns Hopkins University

Inventor： Rana RAIS ,  Barbara SLUSHER ,  Arindom S. PAL

IPC: C07F9/6558 ,  A61K31/4178 ,  A61K31/439 ,  A61K31/4439 ,  A61K31/454 ,  A61K31/4545 ,  A61K31/675 ,  C07D409/04 ,  C07D409/14 ,  C07D471/08

CPC classification number: C07F9/65586 ,  A61K31/4178 ,  A61K31/439 ,  A61K31/4439 ,  A61K31/454 ,  A61K31/4545 ,  A61K31/675 ,  C07D409/04 ,  C07D409/14 ,  C07D471/08

Abstract: Prodrugs of 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, are provided.

公开(公告)号：US12071421B2

公开(公告)日：2024-08-27

申请号：US17699522

申请日：2022-03-21

Applicant: HELSINN HEALTHCARE SA

Inventor： Luca Fadini ,  Peter Manini ,  Claudio Pietra ,  Claudio Giuliano ,  Emanuela Lovati ,  Roberta Cannella ,  Alessio Venturini ,  Valentino J. Stella

IPC: C07D401/04 ,  A61K9/00 ,  A61K31/4178 ,  A61K31/439 ,  A61K31/44 ,  A61K31/473 ,  A61K31/496 ,  A61K31/56 ,  A61K31/573 ,  A61K31/675 ,  A61K45/06 ,  A61P1/08 ,  A61P13/10 ,  A61P25/22 ,  A61P25/24 ,  C07D213/74 ,  C07D213/76 ,  C07D213/89 ,  C07F9/02 ,  C07F9/6509

CPC classification number: C07D401/04 ,  A61K9/0019 ,  A61K31/4178 ,  A61K31/439 ,  A61K31/44 ,  A61K31/473 ,  A61K31/496 ,  A61K31/56 ,  A61K31/573 ,  A61K31/675 ,  A61K45/06 ,  A61P1/08 ,  A61P13/10 ,  A61P25/22 ,  A61P25/24 ,  C07D213/74 ,  C07D213/76 ,  C07D213/89 ,  C07F9/025 ,  C07F9/650952

Abstract: Disclosed are compounds including phosphate and N-oxide prodrugs of the NK1 antagonist netupitant, useful in the prevention and/or treatment of diseases including emesis induced by chemotherapy. Also disclosed are methods of making a di-tert-butyl (chloromethyl) phosphate useful in the manufacture of such prodrugs by reacting a dialkylphosphate salt with an acid to obtain the corresponding ester of phosphoric acid, reacting the ester with a quaternary ammonium hydroxide base to form a monobasic salt, and reacting the monobasic salt with chloroiodomethane to form the corresponding chloromethyl dialkyl phosphate.

公开(公告)号：US12070455B2

公开(公告)日：2024-08-27

申请号：US17930538

申请日：2022-09-08

Applicant: Gilead Sciences, Inc.

Inventor： Stephen E. Ammann ,  Eda Y. Canales ,  Weng K. Chang ,  Henok H. Kinfe ,  Scott E. Lazerwith ,  Michael L. Mitchell ,  Yasamin Moazami ,  Scott D. Schroeder ,  Daniel G. Shore

IPC: C07D519/00 ,  A61K31/437 ,  A61K31/439 ,  A61K31/4545 ,  A61K31/4706 ,  A61K31/496 ,  A61K31/5377 ,  A61K45/06

CPC classification number: A61K31/4545 ,  A61K31/437 ,  A61K31/439 ,  A61K31/4706 ,  A61K31/496 ,  A61K31/5377 ,  A61K45/06 ,  C07D519/00

Abstract: The present disclosure relates generally to certain compounds, pharmaceutical compositions comprising said compounds, and methods of making and using said compounds and pharmaceutical compositions. The compounds and compositions provided herein may be used for the treatment or prevention of an autoimmune disease and/or inflammatory condition, including systemic lupus erythematosus and cutaneous lupus erythematosus.