公开(公告)号：US20240066150A1

公开(公告)日：2024-02-29

申请号：US18466575

申请日：2023-09-13

Applicant: Vertex Phamaceuticals Incorporated

Inventor： Ante Sven Lundberg ,  Samarth Kulkarni ,  Lawrence Klein ,  Hari Kumar Padmanabhan

IPC: A61K48/00 ,  A61K9/00 ,  A61K9/127 ,  A61K9/51 ,  A61K35/30 ,  A61P43/00 ,  C07K14/705 ,  C12N5/0775 ,  C12N5/0793 ,  C12N9/22 ,  C12N15/113 ,  C12N15/90

CPC classification number: A61K48/0066 ,  A61K9/0019 ,  A61K9/127 ,  A61K9/51 ,  A61K35/30 ,  A61P43/00 ,  C07K14/705 ,  C12N5/0619 ,  C12N5/0663 ,  C12N9/22 ,  C12N15/1138 ,  C12N15/907 ,  C12N2310/20

Abstract: The present application provides materials and methods for treating a patient with one or more conditions associated with SCN10A whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of SCN10A gene in a cell by genome editing.

公开(公告)号：US20130131356A1

公开(公告)日：2013-05-23

申请号：US13735757

申请日：2013-01-07

Applicant: VERTEX PHAMACEUTICALS INCORPORATED

Inventor： WILLIAM A. NUGENT ,  Adam R. Looker ,  Raymond E. Forslund ,  Theodore A. Martinot ,  Cristian L. Harrison ,  Sheree Ibrahim

IPC: C07D339/06

CPC classification number: C07D339/06

Abstract: The invention provides β-sulfonamide α-keto esters and amides in which the α-keto is protected as a 1,3-dithiolane derivative. Also provided are methods for preparing such esters and amides and for incorporating them into peptides.

Abstract translation: 本发明提供β-砜酰胺α-酮酯和酰胺，其中α-酮被保护为1,3-二硫戊环衍生物。 还提供了制备这些酯和酰胺并将其并入肽中的方法。

公开(公告)号：WO2012158515A1

公开(公告)日：2012-11-22

申请号：PCT/US2012/037515

申请日：2012-05-11

Applicant: VERTEX PHAMACEUTICALS INCORPORATED ,  TANOURY, Gerald, J.

Inventor： TANOURY, Gerald, J.

IPC: C07B53/00 ,  C07D209/52

CPC classification number: C07B53/00 ,  C07D209/52

Abstract: A process for preparing enantioselectively a compound of formula (la) or (Ib) over a compound of formulas I-2—1h.

Abstract translation: 在式I-2-1h的化合物上对映选择性地制备式（Ia）或（Ib）的化合物的方法。