公开(公告)号：US20240360452A1

公开(公告)日：2024-10-31

申请号：US18771603

申请日：2024-07-12

Applicant: BioMarin Technologies B.V. ,  Academisch Ziekenhuis Leiden

Inventor： Judith Christina Theodora van Deutekom

IPC: C12N15/113 ,  A61K31/56 ,  A61K31/57 ,  A61K31/573 ,  A61K31/58 ,  A61K31/7088 ,  A61K38/17 ,  A61K45/06 ,  A61K48/00

CPC classification number: C12N15/113 ,  A61K31/56 ,  A61K31/57 ,  A61K31/573 ,  A61K31/58 ,  A61K31/7088 ,  A61K38/1719 ,  A61K45/06 ,  A61K48/00 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/31 ,  C12N2310/313 ,  C12N2310/314 ,  C12N2310/315 ,  C12N2310/3181 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3233 ,  C12N2310/346 ,  C12N2320/31 ,  C12N2320/33

Abstract: The invention relates to a method wherein a molecule is used for inducing and/or promoting skipping of at least one of exon 43, exon 46, or exons 50-53 of the DMD pre-mRNA in a patient, the method comprising providing the patient with the molecule. The invention also relates to the molecule as such.

公开(公告)号：US20240360450A1

公开(公告)日：2024-10-31

申请号：US18685880

申请日：2022-08-23

Applicant: COLD SPRING HARBOR HARBOR LABORATORY

Inventor： Adrian Krainer ,  Qian Zhang

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/322 ,  C12N2310/341 ,  C12N2320/33

Abstract: Disclosed herein are compositions and methods for modulating, such as decreasing, the expression of a mutant histone 3.3 (H3.3) allele or a mutant H3.3 gene. In some embodiments, the compositions are antisense oligonucleotides (ASOs), such as gapmer ASOs and splice-modulating ASOs, that target a region of a mutant H3.3 allele or a mutant H3.3 gene, or products thereof, to reduce expression of the mutant H3.3 allele or a mutant H3.3 gene. The compositions and methods are useful in the treatment of diseases and disorders associated with a mutant H3.3 allele or a mutant H3.3 gene, such as cancer.

公开(公告)号：US20240327840A1

公开(公告)日：2024-10-03

申请号：US18579115

申请日：2022-07-15

Applicant: ACADEMISCH ZIEKENHUIS LEIDEN H.O.D.N. LUMC

Inventor： Anton Jan VAN ZONNEVELD ,  Ruben Gosewinus DE BRUIN ,  Jurriën PRINS ,  Gerardus Theodorus Marie WAGENAAR ,  Falk WACHOWIUS ,  Eric Peter VAN DER VEER

IPC: C12N15/113 ,  A61P29/00 ,  C12N15/86

CPC classification number: C12N15/113 ,  A61P29/00 ,  C12N15/86 ,  C12N2310/13 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2750/14143

Abstract: The invention relates to the field of oligonucleotides that can inhibit a RNA-binding protein (RBP) such as Quaking (QKI) by acting as a binding sequence for said RBP (“decoys”). Such oligonucleotide may be used for the treatment of any disease or condition associated with an elevated expression level of QKI, such as inflammation or fibrosis.

公开(公告)号：US12104159B2

公开(公告)日：2024-10-01

申请号：US17808247

申请日：2022-06-22

Applicant: AcuraStem Incorporated

Inventor： Wen-Hsuan Chang ,  Emily Elizabeth Lee

IPC: C07H21/02 ,  A61P25/00 ,  C12N15/113

CPC classification number: C12N15/1137 ,  A61P25/00 ,  C12N2310/11 ,  C12N2310/31 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/345 ,  C12N2310/346 ,  C12N2320/32

Abstract: The present invention relates to PIKFYVE antisense oligonucleotides (ASOs), pharmaceutical compositions containing them, and methods for treating, inhibiting, suppressing, and preventing neurological diseases with them.

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：US12098370B2

公开(公告)日：2024-09-24

申请号：US17399934

申请日：2021-08-11

Applicant: SomaLogic Operating Co., Inc.

Inventor： Dominic Zichi ,  Sheri K Wilcox ,  Chris Bock ,  Daniel J. Schneider ,  Bruce Eaton ,  Larry Gold

IPC: C12N15/115 ,  C12N9/74 ,  C12N15/10 ,  C12Q1/6811 ,  C12Q1/6816 ,  C12Q1/6832 ,  C12Q1/6834 ,  G01N33/53 ,  G01N33/58

CPC classification number: C12N15/115 ,  C12N9/6429 ,  C12N15/1048 ,  C12Q1/6811 ,  C12Q1/6816 ,  C12Q1/6832 ,  C12Q1/6834 ,  G01N33/5308 ,  G01N33/58 ,  C12N2310/16 ,  C12N2310/314 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/333 ,  C12N2310/334 ,  C12N2310/335 ,  C12N2310/336 ,  C12N2330/31 ,  C12Q1/6811 ,  C12Q2525/205 ,  C12Q1/6816 ,  C12Q2565/514 ,  C12Q2525/205 ,  C12Q2525/161 ,  C12Q1/6832 ,  C12Q2537/101 ,  C12Q2525/205 ,  C12Q2523/313 ,  C12Q1/6834 ,  C12Q2525/205 ,  C12Q2525/161

Abstract: The present disclosure describes improved SELEX methods for producing aptamers that are capable of binding to target molecules and improved photoSELEX methods for producing photoreactive aptamers that are capable of both binding and covalently crosslinking to target molecules. Specifically, the present disclosure describes methods for producing aptamers and photoaptamers having slower dissociation rate constants than are obtained using prior SELEX and photoSELEX methods. The disclosure further describes aptamers and photoaptamers having slower dissociation rate constants than those obtained using prior methods. In addition, the disclosure describes aptamer constructs that include a variety of functionalities, including a cleavable element, a detection element, and a capture or immobilization element.

公开(公告)号：US12097263B2

公开(公告)日：2024-09-24

申请号：US18416981

申请日：2024-01-19

Applicant: Dyne Therapeutics, Inc.

Inventor： Romesh R. Subramanian ,  Mohammed T. Qatanani ,  Timothy Weeden ,  Cody A. Desjardins

IPC: C07K16/28 ,  A61K47/60 ,  A61K47/68 ,  A61P21/00 ,  C12N15/113

CPC classification number: A61K47/6807 ,  A61K47/60 ,  A61K47/6849 ,  A61P21/00 ,  C07K16/2881 ,  C12N15/1137 ,  C07K2317/55 ,  C07K2317/92 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/322 ,  C12N2310/3513

Abstract: Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of a DMPK allele comprising a disease-associated-repeat. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.

公开(公告)号：US20240309386A1

公开(公告)日：2024-09-19

申请号：US18374166

申请日：2023-09-28

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： David Bumcrot ,  Brian Bettencourt ,  Ivanka Toudjarska

IPC: C12N15/113 ,  A61K31/713

CPC classification number: C12N15/1138 ,  A61K31/713 ,  C12N15/1137 ,  C12Y304/21 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/344 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2320/30

Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the TMPRSS6 gene, and methods of using such dsRNA compositions to inhibit expression of TMPRSS6.

公开(公告)号：US20240309364A1

公开(公告)日：2024-09-19

申请号：US17341368

申请日：2021-06-07

Applicant: THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA ,  Murdoch University

Inventor： Tejvir S. KHURANA ,  Steve WILTON

IPC: C12N15/113 ,  A61K31/7088 ,  A61P21/00

CPC classification number: C12N15/113 ,  A61K31/7088 ,  A61P21/00 ,  C12N2310/113 ,  C12N2310/314 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3233 ,  C12N2320/32

Abstract: This invention provides a method for enhancing utrophin protein production in a cell by inhibiting an utrophin microRNA molecule. Moreover, the invention provides that methods for enhancing utrophin protein production in a muscle cell are used for treating a muscular dystrophy and/or other myopathies.

公开(公告)号：US12091666B2

公开(公告)日：2024-09-17

申请号：US17709529

申请日：2022-03-31

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： Kevin Fitzgerald ,  Gregory Hinkle ,  Timothy Ryan Mooney

IPC: C12N15/113 ,  A61K9/00 ,  A61K31/713 ,  A61K47/14

CPC classification number: C12N15/1137 ,  A61K9/0019 ,  A61K31/713 ,  A61K47/14 ,  C12N2310/14 ,  C12N2310/3125 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/335 ,  C12N2310/351 ,  C12N2310/3515 ,  C12N2310/321 ,  C12N2310/3521 ,  C12N2310/322 ,  C12N2310/3533

Abstract: The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting a xanthine dehydrogenase (XDH) gene, and methods of using such double stranded RNAi agents to inhibit expression of an XDH gene and methods of treating subjects having an XDH-associated disease.