公开(公告)号：US20240417723A1

公开(公告)日：2024-12-19

申请号：US18675023

申请日：2024-05-27

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Anastasia Khvorova ,  Chantal Ferguson

IPC: C12N15/113 ,  A61K47/54

Abstract: This disclosure relates to a therapeutic combination of drugs for the treatment or management of a neurodegenerative disease, the combination comprising: a first conjugate comprising an RNA silencing agent and a first targeting agent that targets the first conjugate to the central nervous system, and a second conjugate comprising an antagonist of the RNA silencing agent and a second targeting agent that targets the second conjugate to a off-target tissue.

公开(公告)号：US20230193281A1

公开(公告)日：2023-06-22

申请号：US17982993

申请日：2022-11-08

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Anastasia Khvorova ,  James W. Gilbert ,  Chantal Ferguson ,  Robert Brown ,  Alexandra Weiss

IPC: C12N15/113 ,  A61P25/28

CPC classification number: C12N15/1137 ,  A61P25/28 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2320/32

Abstract: This disclosure relates to novel SOD1 targeting sequences. Novel SOD1 targeting oligonucleotides for the treatment of SOD1-related familial amyotrophic lateral sclerosis (ALS).

公开(公告)号：US20250043284A1

公开(公告)日：2025-02-06

申请号：US18760779

申请日：2024-07-01

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Anastasia Khvorova ,  Chantal Ferguson ,  Sarah Davis ,  Kathryn Monopoli

IPC: C12N15/113 ,  A61K47/54 ,  A61K47/55 ,  A61K48/00

Abstract: This disclosure relates to novel SNCA targeting sequences. Novel SNCA targeting oligonucleotides for the treatment of neurodegenerative diseases are also provided.

公开(公告)号：US20240035027A1

公开(公告)日：2024-02-01

申请号：US18142844

申请日：2023-05-03

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Anastasia Khvorova ,  Daniel O'Reilly ,  Chantal Ferguson ,  Jillian Belgrad

IPC: C12N15/113 ,  C12N15/86 ,  A61K31/713 ,  A61K31/203 ,  A61K31/355

CPC classification number: C12N15/113 ,  C12N15/86 ,  A61K31/713 ,  A61K31/203 ,  A61K31/355 ,  A61P43/00

Abstract: This disclosure relates to novel PMS2 targeting sequences. Novel PMS2 targeting oligonucleotides for the treatment of a trinucleotide repeat disease or disorder are also provided.

公开(公告)号：US20210363524A1

公开(公告)日：2021-11-25

申请号：US17204483

申请日：2021-03-17

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Anastasia Khvorova ,  Chantal Ferguson ,  Sarah Davis ,  Kathryn Monopoli

IPC: C12N15/113 ,  A61K47/54 ,  A61K48/00 ,  A61K47/55

Abstract: This disclosure relates to novel SNCA targeting sequences. Novel SNCA targeting oligonucleotides for the treatment of neurodegenerative diseases are also provided.

公开(公告)号：US20210340533A1

公开(公告)日：2021-11-04

申请号：US17132803

申请日：2020-12-23

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Anastasia Khvorova ,  Chantal Ferguson

IPC: C12N15/113 ,  A61K47/54

Abstract: This disclosure relates to a therapeutic combination of drugs for the treatment or management of a neurodegenerative disease, the combination comprising: a first conjugate comprising an RNA silencing agent and a first targeting agent that targets the first conjugate to the central nervous system, and a second conjugate comprising an antagonist of the RNA silencing agent and a second targeting agent that targets the second conjugate to a off-target tissue.

公开(公告)号：US12037585B2

公开(公告)日：2024-07-16

申请号：US17132803

申请日：2020-12-23

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Anastasia Khvorova ,  Chantal Ferguson

IPC: C12N15/113 ,  A61K47/54

CPC classification number: C12N15/113 ,  A61K47/542 ,  A61K47/549 ,  A61K47/554 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/322 ,  C12N2310/3515 ,  C12N2320/32

Abstract: This disclosure relates to a therapeutic combination of drugs for the treatment or management of a neurodegenerative disease, the combination comprising: a first conjugate comprising an RNA silencing agent and a first targeting agent that targets the first conjugate to the central nervous system, and a second conjugate comprising an antagonist of the RNA silencing agent and a second targeting agent that targets the second conjugate to a off-target tissue.

公开(公告)号：US20240052344A1

公开(公告)日：2024-02-15

申请号：US18142852

申请日：2023-05-03

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Anastasia Khvorova ,  Daniel O'Reilly ,  Chantal Ferguson ,  Jillian Belgrad

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2310/315

Abstract: This disclosure relates to novel PMS1 targeting sequences. Novel PMS1 targeting oligonucleotides for the treatment of trinucleotide repeat disease or disorder are also provided.

公开(公告)号：US20230392146A1

公开(公告)日：2023-12-07

申请号：US18120030

申请日：2023-03-10

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Anastasia Khvorova ,  Chantal Ferguson ,  Sarah Davis

IPC: C12N15/113 ,  A61P25/28 ,  C12N15/86

CPC classification number: C12N15/113 ,  A61P25/28 ,  C12N15/86 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/32 ,  C12N2750/14143

Abstract: This disclosure relates to novel APP targeting sequences. Novel APP targeting oligonucleotides for the treatment of neurodegenerative diseases are also provided.

公开(公告)号：US20230340475A1

公开(公告)日：2023-10-26

申请号：US18082654

申请日：2022-12-16

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Anastasia Khvorova ,  Daniel O'Reilly ,  Chantal Ferguson ,  Jillian Belgrad

IPC: C12N15/113 ,  A61P25/28 ,  C12N15/86

CPC classification number: C12N15/113 ,  A61P25/28 ,  C12N15/86 ,  C12N2310/11 ,  C12N2310/32 ,  C12N2310/31 ,  C12N2750/14143

Abstract: This disclosure relates to novel MLH1 targeting sequences. Novel MLH1 targeting oligonucleotides for the treatment of neurodegenerative diseases are also provided.