公开(公告)号：WO2022036126A2

公开(公告)日：2022-02-17

申请号：PCT/US2021/045784

申请日：2021-08-12

申请人： AMGEN INC.

发明人： MURRAY, Justin K. ,  ZHANG, Jun ,  HOMANN, Oliver ,  LONG, Jason C. ,  MEADE, Bryan

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61K31/7125 ,  A61P1/16 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/332 ,  C12N2310/343 ,  C12N2310/346 ,  C12N2320/11 ,  C12N2320/30 ,  C12Q1/6869

摘要： The present invention relates to RNAi constructs for reducing expression of the MARC1 gene. Methods of using such RNAi constructs to treat or prevent liver fibrosis and fatty liver diseases, such as nonalcoholic fatty liver disease and nonalcoholic steatohepatitis, are also described.

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：WO2022214522A2

公开(公告)日：2022-10-13

申请号：PCT/EP2022/059070

申请日：2022-04-06

申请人： ASTRAZENECA AB

发明人： LI, Songyuan ,  MARESCA, Marcello ,  SVIKOVIC, Sasa

IPC分类号： C12N15/11 ,  C12N9/22 ,  C12N9/12 ,  C12N9/00 ,  C12N15/90 ,  C12N2310/20 ,  C12N2310/315 ,  C12N2310/332 ,  C12N2310/3519 ,  C12N9/93 ,  C12Y207/07049 ,  C12Y605/01

摘要： The present disclosure provides a polynucleotide comprising an RNA guide sequence, a Cas-binding region, and a DNA template sequence. The disclosure also provides compositions comprising a Cas nuclease or a Cas nickase and one or more polynucleotides comprising a guide sequence, a Cas binding region, and a DNA template sequence. The disclosure further provides a fusion protein comprising a Cas nuclease or a Cas nickase and a DNA polymerase recruitment moiety. Also provided are methods for providing a targeted insertion in a target DNA of a cell.

公开(公告)号：WO2021231698A1

公开(公告)日：2021-11-18

申请号：PCT/US2021/032195

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： C12N15/11 ,  C12N9/78 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/323 ,  C12N2310/332 ,  C12N2310/346 ,  C12N2320/34 ,  C12N9/88 ,  C12Y305/04004 ,  C12Y403/02001

摘要： The present invention relates to methods and compositions for editing an ASL polynucleotide, e.g., an ASL polynucleotide comprising a SNP associated with argininosuccinate lyase deficiency. The invention also relates to methods and compositions for treating or preventing argininosuccinate lyase deficiency in a subject.

公开(公告)号：WO2021231691A1

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 RS 1 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：WO2021231685A1

公开(公告)日：2021-11-18

申请号：PCT/US2021/032171

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： C12N15/11 ,  C12N9/78 ,  C07K14/705 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/323 ,  C12N2310/332 ,  C12N2310/346 ,  C12N2320/34 ,  C12Y305/04004

摘要： The present invention relates to methods and compositions for editing a TMC1 polynucleotide, e.g., a TMC1 polynucleotide comprising a SNP associated with autosomal recessive non-syndromic hearing loss. The invention also relates to methods and compositions for treating or preventing autosomal recessive non-syndromic hearing loss in a subject.