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：US20240336919A1

公开(公告)日：2024-10-10

申请号：US18618604

申请日：2024-03-27

Applicant: Hoffmann-La Roche Inc.

Inventor： Lars JOENSON ,  Tobias MERKLE ,  Anja MOELHART HOEG ,  Jonas VIKESAA

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2320/30

Abstract: The present invention relates to an antisense oligonucleotide for use in treating and/or preventing a polyglutamine (polyQ) disease, wherein said antisense oligonucleotide is capable of specifically binding to a CAG repeat region of an mRNA encoding a polyQ disease-related protein such that a double stranded RNA is formed which is capable of attracting an Adenosine Deaminase Acting on RNA (ADAR) inserting an A to I exchange into at least one CAG trinucleotide of the CAG repeat region of said mRNA. The present invention further relates to a pharmaceutical composition comprising said antisense molecule.

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：US20240318180A1

公开(公告)日：2024-09-26

申请号：US18735668

申请日：2024-06-06

Applicant: Hoffmann-La Roche Inc.

Inventor： Lars JOENSON ,  Bettina NORDBO ,  Jonas VIKESAA

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3233 ,  C12N2320/33

Abstract: The present invention relates to antisense oligonucleotide splice modulators of actin-like 6B (ACTL6B). These antisense oligonucleotide splice modulators are complementary, such as fully complementary, to the ACTL6B precursor-mRNA, and are capable of increasing or restoring expression of ACTL6B in TDP-43 depleted cells, such as for use in conditions and medical indications where TDP-43 is functionally depleted.

公开(公告)号：US20230122753A1

公开(公告)日：2023-04-20

申请号：US17822668

申请日：2022-08-26

Applicant: Hoffmann-La Roche Inc.

Inventor： Mads Aaboe JENSEN ,  Lars JOENSON ,  Gianluigi LICHINCHI ,  Jonas VIKESAA

IPC: C12N15/113 ,  A61P35/00

Abstract: The present invention relates to antisense oligonucleotides that are complementary to mammalian CD73 (NT5E) pre-mRNA, wherein the antisense oligonucleotides are capable of modulating the splicing of mammalian CD73 pre-mRNA exon 7. Splice modulation of mammalian CD73 exon 7 is beneficial for a range of medical disorders, including disorders in the field of immune-oncology.

公开(公告)号：US20210388357A1

公开(公告)日：2021-12-16

申请号：US17320139

申请日：2021-05-13

Applicant: Hoffmann-La Roche Inc.

Inventor： Ravi JAGASIA ,  Lars JOENSON ,  Søren RASMUSSEN ,  Disa TEHLER ,  Dorthe Vang LARSEN ,  Jesper WORM

IPC: C12N15/113

Abstract: The present invention relates to oligonucleotides which up-regulate or restore the expression of progranulin in cells, and their use in the treatment of neurological disorders, and disorders associated with progranulin haploinsufficiency.