公开(公告)号：US20210198305A1

公开(公告)日：2021-07-01

申请号：US16618003

申请日：2018-06-01

Applicant: WAVE LIFE SCIENCES LTD. ,  Pfizer Inc.

Inventor： Chandra Vargeese ,  Naoki Iwamoto ,  David Charles Donnell Butler ,  Subramanian Marappan ,  Genliang Lu ,  Jason Jingxin Zhang ,  Vinod Vathipadiekal ,  Maria David Frank-Kamenetsky ,  Luciano Henrique Apponi ,  Hanna Maria Wisniewska ,  Xiayun Cheng ,  Young Jin Cho

IPC: C07H17/04 ,  C07H19/04 ,  C12N15/113

Abstract: Among other things, the present disclosure provides designed APOC3 oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide improved single-stranded RNA interference and/or RNase H-mediated knockdown. Among other things, the present disclosure encompasses the recognition that structural elements of oligonucleotides, such as base sequence, chemical modifications (e.g., modifications of sugar, base, and/or internucleotidic linkages) or patterns thereof, conjugation with additional chemical moieties, and/or stereochemistry [e.g., stereochemistry of backbone chiral centers (chiral internucleotidic linkages)], and/or patterns thereof, can have significant impact on oligonucleotide properties and activities, e.g., RNA interference (RNAi) activity, stability, delivery, etc. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions, for example, in RNA interference and/or RNase H-mediated knockdown.

公开(公告)号：US12281127B2

公开(公告)日：2025-04-22

申请号：US18903345

申请日：2024-10-01

Applicant: PFIZER INC.

Inventor： Benjamin Joseph Burke ,  Jacob Cole Deforest ,  Asako Nagata ,  Simon Paul Planken ,  Jillian Elyse Spangler ,  Scott Channing Sutton ,  Hanna Maria Wisniewska ,  Shouliang Yang

IPC: C07D519/00 ,  A61K31/553 ,  A61P35/00

Abstract: The invention relates to compounds of Formula (I)-(III) and pharmaceutically acceptable salts thereof to their use in medicine; to compositions containing them; to processes for their preparation; and to intermediates used in such processes. The compounds the present invention may be useful in the treatment, prevention, suppression and amelioration diseases, disorders and conditions such as cancers.

公开(公告)号：US20220184041A1

公开(公告)日：2022-06-16

申请号：US17533592

申请日：2021-11-23

Applicant: Pfizer Inc.

Inventor： Shawn Cabral ,  Daniel Paul Canterbury ,  Robert Lee Dow ,  Andrew Fensome ,  Magdalena Korczynska ,  Sophie Yvette Lavergne ,  Allyn Timothy Londregan ,  Vincent Mascitti ,  David Walter Piotrowski ,  Andre Shavnya ,  Meihua Mike Tu ,  Tao Wang ,  Hanna Maria Wisniewska

IPC: A61K31/428 ,  C07D417/04 ,  C07D513/04 ,  A61K31/437 ,  A61K31/353 ,  A61K31/423 ,  A61K31/415 ,  A61K31/192 ,  A61K31/12 ,  A61K31/7105

Abstract: This application includes a compound of Formula I or a pharmaceutically acceptable salt thereof; wherein the variables R1a, R1b, R2, R3, X, Y and Z are as defined herein, pharmaceutical compositions comprising the compounds of Formula I and methods of treatment comprising administering to a patient in need thereof a compound of Formula I for the treatment of transthyretin amyloidosis and diseases related thereto.