公开(公告)号：US20250034576A1

公开(公告)日：2025-01-30

申请号：US18743170

申请日：2024-06-14

Applicant: Hoffmann-La Roche Inc.

Inventor： Philipp Friedrich BERNINGER ,  Konrad BLEICHER ,  Erik FUNDER ,  Helle JACOBSEN ,  Dennis Jul HANSEN ,  Michael KELLER ,  Inna Appeldorff LARSEN ,  Meiling LI ,  Disa Elisabet TEHLER ,  Lotte WINTHER ,  Jesper WORM ,  Lena WYSS ,  Tiago Francisco SANTOS FERREIRA

IPC: C12N15/113

Abstract: The present invention relates to double stranded oligonucleotides that are complementary to JAK1, leading to modulation of the expression of JAK1. Modulation of JAK1 expression is beneficial for a range of medical disorders including inflammatory bowel disease, organ transplant rejection, graft-versus-host disease, multiple sclerosis, rheumatoid arthritis (RA), juvenile idiopathic arthritis, psoriasis, dermatitis, diabetic nephropathy, systemic lupus erythematosus (SLE), dry eye disease, cancer, myelofibrosis, and asthma. Also included are compositions comprising the double stranded oligonucleotide and methods of treatment using the double stranded oligonucleotide.

公开(公告)号：US20220033818A1

公开(公告)日：2022-02-03

申请号：US17383709

申请日：2021-07-23

Applicant: Hoffmann-La Roche Inc.

Inventor： Congwei WANG ,  Christian WEILE ,  Martin EBELING ,  Lars JOENSON ,  Jonas VIKESAA ,  Ravi JAGASIA ,  Meiling LI

IPC: C12N15/113

Abstract: The present invention relates to antisense oligonucleotides which are complementary to conserved TDP-43 binding sites on pre-mRNA transcripts, which are capable of restoring RNA binding protein function in the processing of multiple independent mRNAs in TDP-43 depleted cells.

公开(公告)号：US20240327837A1

公开(公告)日：2024-10-03

申请号：US18435935

申请日：2024-02-07

Applicant: Hoffmann-La Roche Inc.

Inventor： Congwei WANG ,  Christian WEILE ,  Martin EBELING ,  Lars JOENSON ,  Jonas VIKESAA ,  Ravi JAGASIA ,  Meiling LI

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N2310/11 ,  C12N2310/3231

Abstract: The present invention relates to antisense oligonucleotides which are complementary to conserved TDP-43 binding sites on pre-mRNA transcripts, which are capable of restoring RNA binding protein function in the processing of multiple independent mRNAs in TDP-43 depleted cells.

公开(公告)号：US20250145996A1

公开(公告)日：2025-05-08

申请号：US18948897

申请日：2024-11-15

Applicant: Hoffmann-La Roche Inc.

Inventor： Jessica Marine Aurore BASTIEN ,  Katarzyna CHYZYNSKA ,  Lars JOENSON ,  Meiling LI ,  Bettina NORDBO ,  Jonas VIKESAA

IPC: C12N15/113

Abstract: The present invention relates to antisense oligonucleotides which are complementary to conserved TDP-43 binding sites on pre-mRNA transcripts, which are capable of restoring RNA binding protein function in the processing of multiple independent mRNAs in TDP-43 depleted cells. The contiguous nucleotide sequence of the antisense oligonucleotides comprise 2′-O-methoxyethyl-RNA (2′-MOE) nucleosides and the antisense oligonucleotides are attached to cholesterol moieties.

公开(公告)号：US20240336920A1

公开(公告)日：2024-10-10

申请号：US18749156

申请日：2024-06-20

Applicant: Hoffmann-La Roche Inc.

Inventor： Jessica Marine Aurore BASTIEN ,  Konrad BLEICHER ,  Helle CHRISTIANSEN ,  Erich KOLLER ,  Meiling LI ,  Helle NYMARK ,  Adrian SCHAEUBLIN ,  Steffen SCHMIDT

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N2310/11 ,  C12N2310/321 ,  C12N2310/323 ,  C12N2310/341

Abstract: Described are antisense oligonucleotides comprising one or more α-L-threofuranosyl (TNA) nucleosides as well as methods to modulate the properties of antisense oligonucleotides by the introduction of TNA nucleosides. These are particularly applicable for antisense gapmer oligonucleotides.