公开(公告)号：US20250163411A1

公开(公告)日：2025-05-22

申请号：US19025012

申请日：2025-01-16

Applicant: Vertex Pharmaceuticals Incorporated

Inventor： D’anna Mae Nelson ,  Gregoriy Aleksandrovich Dokshin

IPC: C12N15/11 ,  C12N9/22 ,  C12N15/86 ,  C12N15/90

Abstract: Compositions comprising tandem gRNAs (tgRNAs) are encompassed as well as their use in treating a disease or disorder that would benefit from an excision of an exon, intron, or exon-intron junction.

公开(公告)号：US20250163021A1

公开(公告)日：2025-05-22

申请号：US18836463

申请日：2023-02-08

Applicant: Vertex Pharmaceuticals Incorporated

Inventor： Timothy J. SENTER ,  Leslie A. DAKIN ,  Elena DOLGIKH ,  Jessica H. OLSEN ,  Akira J. SHIMIZU ,  Steven D. STONE

IPC: C07D401/06 ,  A61K31/454 ,  C07D409/14

Abstract: The disclosure provides at least one compound, tautomer, deuterated derivative, or pharmaceutically acceptable salt chosen from compounds of Formula I, tautomers thereof, deuterated derivatives of those compounds or tautomers, and pharmaceutically acceptable salts of any of the foregoing, compositions comprising the same, and methods of using the same, including uses in treating APOL1-mediated diseases, including pancreatic cancer, focal segmental glomerulosclerosis (FSGS), and/or non-diabetic kidney disease (NDKD).

公开(公告)号：US20250146001A1

公开(公告)日：2025-05-08

申请号：US18919091

申请日：2024-10-17

Applicant: Vertex Pharmaceuticals Incorporated

Inventor： Gregoriy Aleksandrovich Dokshin ,  Jishnu Saha

IPC: C12N15/113 ,  C07K16/28 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86 ,  C12N15/88

Abstract: Compositions and methods for enhancing AAV therapy by increasing the percentage of AAV delivered to a non-liver target in a subject by blocking binding of binding of AAVs to AAV receptors in the liver and then administering an AAV therapy targeting a non-liver tissue.

公开(公告)号：US20250136612A1

公开(公告)日：2025-05-01

申请号：US18836407

申请日：2023-02-08

Applicant: VERTEX PHARMACEUTICALS INCORPORATED

Inventor： Timothy J. SENTER ,  Samantha ANGLE ,  Michael A. BRODNEY ,  Jingrong CAO ,  Jon COME ,  Leslie A. DAKIN ,  Elena DOLGIKH ,  Zachary GALE-DAY ,  Elaine B. KRUEGER ,  Suganthini NANTHAKUMAR ,  Jessica H. OLSEN ,  Akira J. SHIMIZU ,  Steven D. STONE ,  Haoxuan WANG

IPC: C07D495/20 ,  A61K31/438 ,  A61K31/444 ,  A61K31/497 ,  A61K31/501 ,  A61K31/506

Abstract: The disclosure provides at least one compound, tautomer, deuterated derivative, or pharmaceutically acceptable salt chosen from compounds of Formula I, tautomers thereof, deuterated derivatives of those compounds or tautomers, and pharmaceutically acceptable salts of any of the foregoing, compositions comprising the same, and methods of using the same, including uses in treating APOL1-mediated diseases, including pancreatic cancer, focal segmental glomerulosclerosis (FSGS), and/or non-diabetic kidney disease (NDKD).

公开(公告)号：US20250122218A1

公开(公告)日：2025-04-17

申请号：US18836499

申请日：2023-02-07

Applicant: Vertex Pharmaceuticals Incorporated

Inventor： Jeremy J. CLEMENS ,  Alexander R. ABELA ,  Brett C. BOOKSER ,  Thomas CLEVELAND ,  Timothy R. COON ,  Michel GALLANT ,  Sara Sabina HADIDA RUAH ,  Peter GROOTENHUIS ,  Yoshihiro ISHIHARA ,  Julie LATERREUR ,  Jason MCCARTNEY ,  Mark Thomas MILLER ,  Prasuna PARASELLI ,  Yeeman K. RAMTOHUL ,  Thumkunta Jagadeeswar REDDY ,  William SCHULZ BECHARA ,  Claudio STURINO ,  Joe A. TRAN ,  Lino VALDEZ ,  Jinglan ZHOU ,  Ramkrishna DE

IPC: C07D498/22 ,  A61K31/404 ,  A61K31/4155 ,  A61K31/439 ,  A61K31/4433 ,  A61K31/4439 ,  A61K31/47 ,  A61K31/529 ,  C07D498/18

Abstract: This disclosure provides modulators of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), pharmaceutical compositions containing at least one such modulator, methods of treatment of cystic fibrosis using such modulators and pharmaceutical compositions, and processes for making such modulators.

公开(公告)号：US20250099425A1

公开(公告)日：2025-03-27

申请号：US18643207

申请日：2024-04-23

Applicant: Vertex Pharmaceuticals Incorporated

Inventor： Cathy CHU ,  Varsha DHAMANKAR ,  Eleni DOKOU ,  Eric L. HASELTINE ,  Samuel MOSKOWITZ ,  Sarah ROBERTSON ,  David WALTZ ,  Weichao George CHEN

IPC: A61K31/4045 ,  A61K31/404 ,  A61K31/443 ,  A61K31/4439 ,  A61K31/47 ,  A61P11/00 ,  A61P25/28

Abstract: A pharmaceutical composition comprising Compound I: Methods of treating cystic fibrosis comprising administering one or more of such pharmaceutical compositions to a patient.

公开(公告)号：US20250090686A1

公开(公告)日：2025-03-20

申请号：US18826808

申请日：2024-09-06

Applicant: Vertex Pharmaceuticals Incorporated

Inventor： Tudor Fulga ,  Yurong Xin ,  Yi-Li Min ,  Foram Ashar ,  Su Wang ,  Jianming Liu ,  Yang Guo ,  D'anna Nelson

IPC: A61K48/00 ,  A61P21/00 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86

Abstract: Compositions and methods for treating Duchenne Muscular Dystrophy (DMD) and excising small portions of exons 44, 50, and 53 of the DMD gene are encompassed.

公开(公告)号：US12215355B2

公开(公告)日：2025-02-04

申请号：US17171497

申请日：2021-02-09

Applicant: Vertex Pharmaceuticals Incorporated

Inventor： Felicia J. Pagliuca ,  George Harb ,  Lillian Ye

IPC: C12N5/071 ,  A61K35/12 ,  A61K35/39 ,  A61P5/50

Abstract: Provided herein are methods of producing β cells and precursors thereof utilizing a Wnt signaling inhibitor or PKC activator, or both. Also provided herein are in vitro cultures comprising said cells, methods of treating a subject with a disease characterized by high blood sugar levels over a prolonged period of time by administering said cells, and devices for encapsulating said cells.

公开(公告)号：US12195453B2

公开(公告)日：2025-01-14

申请号：US17930352

申请日：2022-09-07

Applicant: Merck Patent GmbH ,  Vertex Pharmaceuticals Incorporated

Inventor： Henry Yu ,  Michael Clark ,  Guy Bemis ,  Michael Boyd ,  Kishan Chandupatla ,  Philip Collier ,  Hongbo Deng ,  Huijun Dong ,  Warren Dorsch ,  Russell R. Hoover ,  Mac Arthur Johnson, Jr. ,  Shashank Kulkarni ,  Marina Penney ,  Steven Ronkin ,  Darin Takemoto ,  Qing Tang ,  Nathan D. Waal ,  Tiansheng Wang ,  David J. Lauffer ,  Pan Li

IPC: C07D413/04 ,  A61K31/506 ,  A61K31/553 ,  A61P35/00 ,  C07D401/14 ,  C07D403/04 ,  C07D413/14 ,  C07D417/14 ,  C07D491/107 ,  C07D491/113

Abstract: The present invention provides compounds of Formula I′, or pharmaceutically acceptable salts thereof, pharmaceutical compositions thereof, and methods of use thereof for treating cellular proliferative disorders (e.g., cancer).

公开(公告)号：US20240400544A1

公开(公告)日：2024-12-05

申请号：US18566282

申请日：2022-06-03

Applicant: VERTEX PHARMACEUTICALS INCORPORATED

Inventor： Elizabeth Mary BECK ,  Robert PULLIN ,  Gorka ETXEBARRIA JARDI ,  Dean STAMOS ,  Yvonne SCHMIDT ,  Joseph PONTILLO ,  Stephen Andrew THOMSON ,  David Matthew SHAW ,  Nadia M. AHMAD ,  Lidio Marx CARVALHO MEIRELES ,  Sarah SKERRATT ,  Sara S. HADIDA RUAH ,  Timothy Donald NEUBERT ,  Dennis James HURLEY ,  Alexander KINTZER ,  Steven John DURRANT ,  Christopher WRAY ,  Anisa Nizarali VIRANI ,  Kiri NORTH ,  Reece JACQUES ,  Mark Thomas Miller ,  Jinglan ZHOU ,  Ronald Marcellus KNEGTEL ,  Ewa Iwona CHUDYK ,  Joanne Louise PINDER ,  Bruno Artur SOUSA ,  James DODD ,  Michael Edward O'DONNELL ,  Bhairavi GALAN ,  Rebecca E. STEVENS

IPC: C07D413/14 ,  A61K31/4355 ,  A61K31/443 ,  A61K31/506 ,  A61K31/5377 ,  C07D405/12 ,  C07D405/14 ,  C07D491/04

Abstract: Compounds of formula (I) and pharmaceutically acceptable salts thereof, useful as inhibitors of sodium channels are provided. Also provided are pharmaceutical compositions comprising the compounds or pharmaceutically acceptable salts and methods of using the compounds, pharmaceutically acceptable salts, and pharmaceutical compositions in the treatment of various disorders, including pain.