公开(公告)号：WO2022271955A1

公开(公告)日：2022-12-29

申请号：PCT/US2022/034736

申请日：2022-06-23

申请人： MUSC FOUNDATION FOR RESEARCH DEVELOPMENT

发明人： MISRA, Suniti ,  GHATAK, Shibnath

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61K9/51 ,  A61P35/00 ,  A61K9/0019 ,  A61K9/1272 ,  A61K9/5138 ,  A61K9/5146 ,  C12N15/1138 ,  C12N2320/31 ,  C12N2320/32 ,  C12N2320/34

摘要： The present invention generally relates to compositions comprising one or more modulator of one or more chemoresistance promoting molecule, one or more chemosensitivity promoting molecule, or combination thereof, and methods of use thereof. The present invention also relates to CD44v6 inhibitor compositions and methods of use for treating or preventing one or more disease or disorder, such as cancer.

公开(公告)号：WO2022256642A2

公开(公告)日：2022-12-08

申请号：PCT/US2022/032144

申请日：2022-06-03

申请人： ARBOR BIOTECHNOLOGIES, INC.

发明人： WESSELLS, Quinton Norman ,  HASWELL, Jeffrey Raymond ,  DITOMMASO, Tia Marie ,  JAKIMO, Noah Michael ,  SENGUPTA, Sejuti

IPC分类号： C12N15/113 ,  A61K48/00 ,  C12N9/22 ,  A61K38/46 ,  C12N15/1137 ,  C12N2310/20 ,  C12N2320/34 ,  C12Y101/03015

摘要： Provided herein are gene editing systems and/or compositions comprising RNA guides targeting HAO1 for use in genetic editing of the HAO1 gene. Also provide herein are methods of using the gene editing system for introducing edits to the HAO1 gene and/or for treatment of primary hyperoxaluria (PH), and processes for characterizing the gene editing system.

公开(公告)号：WO2022204476A1

公开(公告)日：2022-09-29

申请号：PCT/US2022/021879

申请日：2022-03-25

申请人： THE BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM

发明人： OLSON, Eric, N. ,  CHEMELLO, Francesco ,  BASSEL-DUBY, Rhonda

IPC分类号： C12N15/113 ,  C12N9/12 ,  C12N9/78 ,  C07K14/4707 ,  C12N2310/20 ,  C12N2320/33 ,  C12N2320/34

摘要： Duchenne muscular dystrophy (DMD) is a fatal muscle disease caused by the lack of dystrophin, which maintains muscle membrane integrity. Provided herein are methods of using adenine base editor (ABE) to modify splice sites of the dystrophin gene, causing skipping or reframing of common DMD exon deletion mutations, restoring dystrophin expression. Also provided herein are methods of using prime editing to reframe the dystrophin open reading frame and restore dystrophin expression.

公开(公告)号：WO2022147249A1

公开(公告)日：2022-07-07

申请号：PCT/US2021/065682

申请日：2021-12-30

申请人： MAYO FOUNDATION FOR MEDICAL EDUCATION AND RESEARCH

发明人： ACKERMAN, Michael J. ,  DOTZLER, Steven M. ,  GENDRON, William ,  BAINS, Sahej ,  KIM, Chang Sung ,  TESTER, David J.

IPC分类号： A61K31/7088 ,  A61K31/713 ,  A61K38/17 ,  A61K48/00 ,  A61P9/00 ,  C07H21/02 ,  A01K2217/054 ,  A01K2227/107 ,  A01K2267/0375 ,  A61K31/7105 ,  A61K38/00 ,  A61K48/005 ,  C07K14/705 ,  C12N15/1138 ,  C12N15/86 ,  C12N2310/14 ,  C12N2320/31 ,  C12N2320/34 ,  C12N2740/16043 ,  C12N2750/14143

摘要： Methods and materials for treating a mammal having a congenital disease (e.g., a congenital heart disease such as congenital long QT syndrome) are provided herein. For example, this document provides methods and materials for generating and using nucleic acids to treat a mammal having a congenital disease, where the nucleic acids can suppress expression of mutant disease-related alleles in the mammal while providing a replacement cDNA that does not contain the disease-related mutation(s).

公开(公告)号：WO2021243301A2

公开(公告)日：2021-12-02

申请号：PCT/US2021/035015

申请日：2021-05-28

申请人： FLAGSHIP PIONEERING INNOVATIONS VI, LLC.

发明人： ANASTASSIADIS, Theonie ,  BERRY, David, Arthur ,  HAJDIN, Christine, Elizabeth ,  AFEYAN, Noubar, Boghos ,  BUTLER, David, Charles, Donnell ,  LI, Qingyi

IPC分类号： C12N15/67 ,  C12N15/11 ,  C12N2310/10 ,  C12N2320/34

摘要： The disclosure relates generally to uses of tRNA-based effector molecules having a non-naturally occurring modification.

公开(公告)号：WO2021231691A8

公开(公告)日：2021-11-18

申请号：PCT/US2021/032184

申请日：2021-05-13

申请人： KORRO BIO, INC.

发明人： JARPE, Matthew ,  PUTTA, Mallikarjuna Reddy ,  FRALEY, Andrew

IPC分类号： C12N15/113 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  A61K48/00 ,  A61K38/50 ,  C07H21/00 ,  A61P27/02 ,  C12N2310/20 ,  C12N2310/315 ,  C12N2310/318 ,  C12N2310/319 ,  C12N2310/3231 ,  C12N2310/3233 ,  C12N2310/3235 ,  C12N2310/332 ,  C12N2320/34 ,  C12Y305/04004

摘要： The present invention relates to methods and compositions for editing an RS1 polynucleotide, e.g., an RS1 polynucleotide comprising a SNP associated with X-linked retinoschisis (XLRS). The invention also relates to methods and compositions for treating or preventing XLRS in a subject.

公开(公告)号：WO2021202621A2

公开(公告)日：2021-10-07

申请号：PCT/US2021/025019

申请日：2021-03-30

申请人： NEUBASE THERAPEUTICS, INC.

发明人： LY, Danith H. ,  THADKE, Shivaji A. ,  BATWAL, Ramesh U. ,  DI CARO, Valentina ,  STEPHAN, Dietrich A. ,  SOOTER, Letha J. ,  BACKENROTH, Samuel I.

IPC分类号： C12Q1/6848 ,  C12Q1/6853 ,  A61K38/00 ,  A61K47/02 ,  A61K47/12 ,  A61K47/542 ,  A61K47/645 ,  A61K51/0491 ,  A61K9/0019 ,  A61K9/0053 ,  A61K9/0085 ,  A61K9/0095 ,  A61K9/06 ,  A61K9/08 ,  A61K9/10 ,  A61K9/107 ,  A61K9/127 ,  A61K9/2004 ,  A61K9/4841 ,  A61P25/28 ,  C07K14/003 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/3181 ,  C12N2320/34 ,  G01N30/7233 ,  G01N33/6896

摘要： The present disclosure relates to compounds useful for the detection or modulation of target nucleic acids, including DNA and RNA. The present disclosure further relates to methods for treatment of trinucleotide repeat disorders, which can include administration of oligonucleotide analogues that can bind pathogenic nucleotide repeats in DNA or RNA.

公开(公告)号：WO2020243368A1

公开(公告)日：2020-12-03

申请号：PCT/US2020/035001

申请日：2020-05-28

申请人： MONSANTO TECHNOLOGY LLC

发明人： CARGILL, Edward James ,  EUDY, Douglas Michael ,  KOURANOV, Andrei Y. ,  LAWRENCE, Richard Joseph ,  SLEWINSKI, Thomas L. ,  SHULTZ, Randy ,  TO, PokChun Jennnifer ,  YANG, Samuel Sukhwan ,  ZHANG, Yuanji

IPC分类号： C12N15/113 ,  A61K48/00 ,  C12N5/10 ,  C07K14/415 ,  C12N15/102 ,  C12N15/1137 ,  C12N15/8213 ,  C12N15/8216 ,  C12N15/8218 ,  C12N15/8223 ,  C12N15/8226 ,  C12N15/8261 ,  C12N15/8262 ,  C12N15/8286 ,  C12N15/8297 ,  C12N15/902 ,  C12N2310/11 ,  C12N2310/127 ,  C12N2310/141 ,  C12N2310/20 ,  C12N2320/34 ,  C12N9/0071 ,  C12N9/22 ,  A40

摘要： The present disclosure provides methods and compositions for generating dominant alleles using targeted editing techniques. Also provided are modified chromosomes, cells, tissues, and plants comprising modified dominant allele.

公开(公告)号：WO2023288240A1

公开(公告)日：2023-01-19

申请号：PCT/US2022/073668

申请日：2022-07-13

申请人： VANDA PHARMACEUTICALS INC.

发明人： PRZYCHODZEN, Bartlomiej ,  SMIESZEK, Sandra

IPC分类号： A61K31/7088 ,  C12N15/113 ,  A61P19/00 ,  A61P21/00 ,  C12N15/1137 ,  C12N2310/11 ,  C12N2320/30 ,  C12N2320/34

摘要： Embodiments of the invention related generally to antisense oligonucleotides (ASOs) and, more particularly, to compositions and methods for regulating protein synthesis using ASOs. In one embodiment, the invention provides a method of treating a patient diagnosed with myelodysplastic syndrome (MDS) comprising: administering to the patient an amount of an antisense oligonucleotide (ASO) targeted to a nucleic acid molecule encoding JAK2 (ASO-T-JAK2) compound effective to treat such disease.

公开(公告)号：WO2022256655A2

公开(公告)日：2022-12-08

申请号：PCT/US2022/032162

申请日：2022-06-03

申请人： ARBOR BIOTECHNOLOGIES, INC.

发明人： WESSELLS, Quinton Norman ,  HASWELL, Jeffrey Raymond ,  DITOMMASO, Tia Marie ,  JAKIMO, Noah Michael ,  SENGUPTA, Sejuti

IPC分类号： C12N15/113 ,  C12N15/90 ,  C12N9/22 ,  A61K48/00 ,  A61K38/46 ,  C12N15/1137 ,  C12N2310/20 ,  C12N2320/34 ,  C12Y101/01027

摘要： Provided herein are gene editing systems and/or compositions comprising RNA guides targeting LDHA for use in genetic editing of the LDHA gene. Also provide herein are methods of using the gene editing system for introducing edits to the LDHA gene and/or for treatment of primary hyperoxaluria (PH), and processes for characterizing the gene editing system.