公开(公告)号：US11753638B2

公开(公告)日：2023-09-12

申请号：US16322212

申请日：2017-08-11

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Anastasia Khvorova ,  Mehran Nikan ,  Matthew Hassler ,  Maire Osborn ,  Reka Haraszti ,  Andrew Coles ,  Anton Turanov ,  Neil Aronin ,  Annabelle Biscans

IPC: A61K31/7088 ,  C12N15/113 ,  A61K47/54 ,  C07H21/02 ,  C07H21/04 ,  C07H21/00 ,  C12N15/11 ,  A61K47/55 ,  A61P1/16 ,  A61P9/00 ,  A61P13/12 ,  A61P1/00 ,  A61P5/00 ,  A61P11/00 ,  A61K9/00

CPC classification number: C12N15/113 ,  A61K9/0019 ,  A61K31/7088 ,  A61K47/54 ,  A61K47/543 ,  A61K47/549 ,  A61K47/551 ,  A61K47/554 ,  A61P1/00 ,  A61P1/16 ,  A61P5/00 ,  A61P9/00 ,  A61P11/00 ,  A61P13/12 ,  C07H21/00 ,  C07H21/02 ,  C07H21/04 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/312 ,  C12N2310/315 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2310/3515 ,  C12N2320/32 ,  C12N2310/321 ,  C12N2310/3521 ,  C12N2310/322 ,  C12N2310/3533

Abstract: Provided herein are conjugated oligonucleotides that are characterized by efficient and specific tissue distribution.

公开(公告)号：US20250059533A1

公开(公告)日：2025-02-20

申请号：US18769546

申请日：2024-07-11

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Anastasia Khvorova ,  Mehran Nikan ,  Matthew Hassler ,  Maire Osborn ,  Reka Haraszti ,  Andrew Coles ,  Anton Turanov ,  Neil Aronin

IPC: C12N15/11 ,  A61K47/54 ,  A61K47/61 ,  C07H21/00

Abstract: Provided herein are self-delivering oligonucleotides that are characterized by efficient RISC entry, minimum immune response and off-target effects, efficient cellular uptake without formulation, and efficient and specific tissue distribution.

公开(公告)号：US12077755B2

公开(公告)日：2024-09-03

申请号：US16812714

申请日：2020-03-09

Applicant: University of Massachusetts

Inventor： Anastasia Khvorova ,  Mehran Nikan ,  Matthew Hassler ,  Maire Osborn ,  Reka Haraszti ,  Andrew Coles ,  Anton Turanov ,  Neil Aronin

IPC: C12N15/11 ,  A61K47/54 ,  A61K47/61 ,  C07H21/00

CPC classification number: C12N15/111 ,  A61K47/542 ,  A61K47/554 ,  A61K47/61 ,  C07H21/00 ,  C12N2310/14 ,  C12N2310/3515 ,  C12N2320/32

Abstract: Provided herein are self-delivering oligonucleotides that are characterized by efficient RISC entry, minimum immune response and off-target effects, efficient cellular uptake without formulation, and efficient and specific tissue distribution.

公开(公告)号：US20200339983A1

公开(公告)日：2020-10-29

申请号：US16812714

申请日：2020-03-09

Applicant: University of Massachusetts

Inventor： Anastasia Khvorova ,  Mehran Nikan ,  Matthew Hassler ,  Maire Osborn ,  Reka Haraszti ,  Andrew Coles ,  Anton Turanov ,  Neil Aronin

IPC: C12N15/11 ,  A61K47/54 ,  A61K47/61 ,  C07H21/00

Abstract: Provided herein are self-delivering oligonucleotides that are characterized by efficient RISC entry, minimum immune response and off-target effects, efficient cellular uptake without formulation, and efficient and specific tissue distribution.

公开(公告)号：US20170043024A1

公开(公告)日：2017-02-16

申请号：US15236051

申请日：2016-08-12

Applicant: University of Massachusetts

Inventor： Anastasia Khvorova ,  Mehran Nikan ,  Matthew Hassler ,  Maire Osborn ,  Reka Haraszti ,  Andrew Coles ,  Anton Turanov ,  Neil Aronin

IPC: A61K47/48 ,  A61K31/713 ,  C12N15/113

CPC classification number: C12N15/111 ,  A61K47/542 ,  A61K47/554 ,  A61K47/61 ,  C07H21/00 ,  C12N2310/14 ,  C12N2310/3515 ,  C12N2320/32

Abstract: Provided herein are self-delivering oligonucleotides that are characterized by efficient RISC entry, minimum immune response and off-target effects, efficient cellular uptake without formulation, and efficient and specific tissue distribution.

Abstract translation: 本文提供的是自身递送的寡核苷酸，其特征在于有效的RISC进入，最小的免疫应答和脱靶效应，有效的细胞摄取而不需要配制，以及有效和特定的组织分布。