公开(公告)号：WO2023092102A1

公开(公告)日：2023-05-25

申请号：PCT/US2022/080188

申请日：2022-11-18

申请人： SANEGENE BIO USA INC.

发明人： ZHANG, Chunyang ,  WANG, Shiyu ,  CAI, Xiaochuan ,  WANG, Weimin ,  YANG, Zhongfa

IPC分类号： C12N15/113 ,  A61K31/713

摘要： The disclosure relates to isolated oligonucleotides comprising duplex regions targeting angiopoietin-like 3 (ANGPTL-3), and delivery systems, kits and compositions comprising same, and methods of using same for inhibiting or downregulating ANGPTL-3.

公开(公告)号：WO2023075766A1

公开(公告)日：2023-05-04

申请号：PCT/US2021/056900

申请日：2021-10-27

申请人： WAVE LIFE SCIENCES LTD. ,  BYRNE, Michael John ,  VATHIPADIEKAL, Vinod ,  IWAMOTO, Naoki ,  VARGEESE, Chandra ,  GUO, Lankai ,  HOSS, Andrew Guzior

发明人： BYRNE, Michael John ,  VATHIPADIEKAL, Vinod ,  IWAMOTO, Naoki ,  VARGEESE, Chandra ,  GUO, Lankai ,  HOSS, Andrew Guzior

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61K31/7125 ,  A61K48/00

摘要： Among other things, the present disclosure provides RHO oligonucleotides, compositions, and methods. In some embodiments, provided oligonucleotides comprise nucleobase modifications, sugar modifications, intemucleotidic linkage modifications and/or patterns thereof, and have improved properties, activities and/or selectivities. In some embodiments, the present disclosure provides RHO oligonucleotides, compositions and methods for preventing and/or treating RHO-related conditions, disorders or diseases, such as retinopathy (e.g., retinal degeneration, retinal degenerative disease, retinal degenerative disorder, inherited retinal degenerative disorder, retinitis pigmentosa, autosomal dominant retinitis pigmentosa, etc.).

公开(公告)号：WO2023075122A1

公开(公告)日：2023-05-04

申请号：PCT/KR2022/013191

申请日：2022-09-02

申请人： 사회복지법인 삼성생명공익재단

发明人： 조용범 ,  김현호 ,  김태원

IPC分类号： A61K48/00 ,  A61K31/713 ,  A61K45/06 ,  A61P35/00 ,  A61P35/04 ,  C12Q1/6886

摘要： 본 발명은 대장암(colorectal cancer)의 치료 및 전이 억제 표적으로서 AFAP1-AS1(AFAP1 Antisense RNA1) 및 MLK7-AS1(MLK7 Antisense RNA1)의 새로운 용도에 관한 것으로서, 보다 구체적으로 상기 AFAP1-AS1 및 MLK7-AS1의 발현 수준을 측정하는 것을 포함하는 대장암의 진단용 키트 및 대장암의 진단방법에 관한 것이다. 또한 본 발명은 상기 AFAP1-AS1 억제제 및 MLK7-AS1 억제제를 포함하는 대장암의 치료 또는 전이 억제용 약학적 조성물에 관한 것이다.

公开(公告)号：WO2023069754A2

公开(公告)日：2023-04-27

申请号：PCT/US2022/047491

申请日：2022-10-21

申请人： AMGEN INC.

发明人： RULIFSON, Ingrid ,  MEADE, Bryan ,  LONG, Jason C. ,  MURRAY, Justin K.

IPC分类号： C12N15/113 ,  C12N9/10 ,  A61K31/713 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  A61P1/16 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/343 ,  C12N2310/351 ,  C12N2320/11 ,  C12N2320/32

摘要： The disclosure relates to RNAi constructs, such as siRNA, for reducing expression of the GPAM gene. Methods of using such RNAi constructs to treat or prevent liver disease, such as nonalcoholic fatty liver disease (NAFLD), are also described.

公开(公告)号：WO2023067127A1

公开(公告)日：2023-04-27

申请号：PCT/EP2022/079348

申请日：2022-10-21

申请人： UNIVERSITE DE STRASBOURG ,  INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE (INSERM) ,  CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE ,  ASSOCIATION FRANÇAISE CONTRE LES MYOPATHIES (AFM)

发明人： LAPORTE, Jocelyn ,  BOHM, Johann ,  SILVA ROJAS, Roberto

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61P21/00

摘要： The present invention relates to the use of shRNA inhibiting ORAI1 expression for the treatment of a disease selected from the group consisting of tubular aggregate myopathy (TAM) and Stormorken syndrome (STRMK).

公开(公告)号：WO2023044458A1

公开(公告)日：2023-03-23

申请号：PCT/US2022/076613

申请日：2022-09-16

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： ZUBER, Jeffrey

IPC分类号： C12N15/11 ,  A61K31/713 ,  A61P3/00

摘要： The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the angiopoietin-like 4 (ANGPTL4) gene, as well as methods of inhibiting expression of ANGPTL4, and methods of treating subjects that would benefit from reduction in expression of ANGPTL4, such as subjects having an ANGPTL4-associated disease, disorder, or condition, using such dsRNA compositions.

公开(公告)号：WO2023034056A1

公开(公告)日：2023-03-09

申请号：PCT/US2022/041038

申请日：2022-08-22

申请人： BIONAUT LABS LTD. ,  BROWN, Tobin ,  CHO, Suehyun ,  STANTON, Marty ,  KISELYOV, Alex

发明人： BROWN, Tobin ,  CHO, Suehyun ,  STANTON, Marty ,  KISELYOV, Alex

IPC分类号： A61K31/713 ,  A61K31/7088 ,  A61K47/10 ,  C12N15/11

摘要： The present invention relates to compositions of a dried rapid-release high concentration oligonucleotide-loaded PEG hydrogel-based matrix, methods for formulating hydrogel matrix-encapsulated oligonucleotides in an amount that exceeds the oligonucleotides intrinsic solubility in water or aqueous media, the hydrogel matrix-encapsulated oligonucleotides produced by the described methods, and therapeutic methods for using the dried rapid-release high concentration hydrogel matrix-encapsulated oligonucleotides for systemic and local micro-delivery.

公开(公告)号：WO2023031359A1

公开(公告)日：2023-03-09

申请号：PCT/EP2022/074386

申请日：2022-09-01

申请人： SILENCE THERAPEUTICS GMBH

发明人： DAMES, Sibylle ,  MORRISON, Eliot ,  AUMILLER, Verena ,  JOHANSSEN, Timo ,  RATHJEN, Stefan ,  SCHUBERT, Steffen

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61K31/7125 ,  A61K31/712

摘要： The invention relates to nucleic acid products that interfere with complement factor B (CFB) gene expression or inhibit its expression. The nucleic acids are preferably for use in the prophylaxis or treatment of complement associated diseases, disorders or syndromes, particularly C3 glomerulopathy (C3G), paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), lupus nephritis, IgA nephropathy (IgA N), myasthenia gravis (MG), and primary membranous nephropathy.

公开(公告)号：WO2023031277A1

公开(公告)日：2023-03-09

申请号：PCT/EP2022/074208

申请日：2022-08-31

申请人： INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) ,  ASSISTANCE PUBLIQUE-HÔPITAUX DE PARIS (APHP) ,  SORBONNE UNIVERSITÉ ,  UNIVERSITÉ PARIS CITÉ

发明人： BEHAR-COHEN, Francine ,  ZHAO, Min

IPC分类号： A61K31/58 ,  A61K31/585 ,  A61P27/02 ,  A61K31/4375 ,  A61K31/713 ,  A61K9/00

摘要： Ocular Rosacea (OR) is a chronic inflammatory and neurovascular diseases of the ocular surface and eyelids, associated with abnormal tear film lipids that can lead to corneal neovascularization, loss of transparency and ulceration. Here, the inventors show that the combination of mineralocorticoid receptor blockade in association with local ocular glucocorticoids that have high GR binding affinity, have superior effects as compared to MR blockade alone, without the side effects of glucocorticoids on corneal wound healing. The combination of MR antagonist and low dose of a GR activator further reduces corneal edema, corneal neovascularization and improves corneal wound healing. The combination of MR antagonist and triamcinolone that has a strong GR binding affinity reinforces the beneficial effects of MR antagonists. Finally, the inventors show that MR is overexpressed in ocular surface tissues and Meibomian glands of patients with ocular rosacea, and that transgenic rats that over express the human MR have molecular markers in their meibomian glands, similar to those of patients with OR. Thus, MR antagonists and GR agonist with strong GR affinity is a suitable combination for the treatment of OR.

公开(公告)号：WO2023022229A1

公开(公告)日：2023-02-23

申请号：PCT/JP2022/031385

申请日：2022-08-19

申请人： 国立大学法人東京医科歯科大学

发明人： 横田　隆徳 ,  永田　哲也

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61K48/00 ,  A61P21/00

摘要： 優れた活性を有する新たな二本鎖核酸複合体を提供することを課題とする。　第1核酸鎖と第2核酸鎖とを含む二本鎖核酸複合体であって、前記第1核酸鎖は、人工核酸を含み、標的遺伝子又はその転写産物の少なくとも一部にハイブリダイズすることができ、かつ前記標的遺伝子又はその転写産物に対してエクソンスキッピング、エクソンインクルージョン、及び／又はステリックブロッキングを誘導することができ、前記第2核酸鎖は、前記第1核酸鎖の少なくとも一部に相補的な塩基配列を含み、かつ体液中に存在するヌクレアーゼに対して耐性を有する少なくとも1個のブロック領域、及び細胞内に存在するヌクレアーゼで分解される少なくとも1個の切断領域を含み、前記切断領域は、連続する2以上の糖非修飾ヌクレオシドを含む、前記二本鎖核酸複合体を提供する。