公开(公告)号：WO2021226597A2

公开(公告)日：2021-11-11

申请号：PCT/US2021/031629

申请日：2021-05-10

Applicant: ORNA THERAPEUTICS, INC.

Inventor： GOODMAN, Brian ,  WESSELHOEFT, Alexander ,  HORHOTA, Allen T. ,  YANG, Junghoon ,  OTT, Kristen ,  BARNES, Thomas

IPC: A61K48/00 ,  A61K31/7088 ,  C12N15/115 ,  C12N15/64 ,  A61K31/7105 ,  C07K14/725 ,  C12N15/85 ,  C12N15/11 ,  A61K9/00 ,  A61K9/127 ,  A61K31/713 ,  A61K39/00 ,  A61K39/39 ,  C12N15/113 ,  C12N15/88 ,  A61K2039/5156 ,  A61K2039/572 ,  A61K39/0011 ,  A61K39/001112 ,  A61K39/001117 ,  A61K39/001124 ,  A61K48/0008 ,  A61K48/0041 ,  A61K48/005 ,  A61K9/0019 ,  A61K9/1271 ,  A61K9/1272 ,  A61P35/00

Abstract: Disclosed herein are circular RNAs and transfer vehicles, along with related compositions and methods of treatment. The circular RNAs can comprise group I intron fragments, spacers, an IRES, duplex forming regions, and/or an expression sequence, thereby having the features of improved expression, functional stability, low immunogenicity, ease of manufacturing, and/or extended half-life compared to linear RNA. Pharmaceutical compositions comprising such circular RNAs and transfer vehicles are particularly suitable for efficient protein expression in immune cells in vivo. Also disclosed are precursor RNAs and materials useful in producing the precursor or circular RNAs, which have improved circularization efficiency and/or are compatible with effective circular RNA purification methods.

公开(公告)号：WO2023081526A1

公开(公告)日：2023-05-11

申请号：PCT/US2022/049313

申请日：2022-11-08

Applicant: ORNA THERAPEUTICS, INC.

Inventor： HORHOTA, Allen T. ,  YANG, Junghoon ,  KAUFFMAN, Kevin ,  BARNES, Thomas ,  WESSELHOEFT, Robert Alexander ,  BECKER, Amy M. ,  MOTZ, Gregory

IPC: C07C229/12 ,  A61K9/127 ,  A61K9/51 ,  A61K47/18

Abstract: Disclosed herein are novel lipids that can be used in combination with other lipid components, such as helper lipids, structural lipids, and cholesterols, to form lipid nanoparticles for delivery of therapeutic agents, such as nucleic acids (e.g., circular polynucleotides), both in vitro and in vivo.

公开(公告)号：WO2021113777A2

公开(公告)日：2021-06-10

申请号：PCT/US2020/063494

申请日：2020-12-04

Applicant: ORNA THERAPEUTICS, INC.

Inventor： GOODMAN, Brian ,  WESSELHOEFT, Alexander ,  HORHOTA, Allen T. ,  YANG, Junghoon ,  OTT, Kristen

IPC: A61K48/00 ,  A61K31/7088 ,  C12N15/115 ,  C12N15/64 ,  A61K31/7105 ,  C07K14/725 ,  C12N15/85 ,  C12N15/11 ,  A61K2039/5156 ,  A61K31/713 ,  A61K39/0011 ,  A61K39/001112 ,  A61K39/39 ,  A61K48/0008 ,  A61K48/0041 ,  A61K48/005 ,  A61K9/00 ,  C07K14/7051 ,  C12N15/1137 ,  C12N15/88 ,  C12N2310/14 ,  C12N2310/20 ,  C12N2310/532 ,  C12Y304/21021 ,  A50

Abstract: Disclosed herein are circular RNAs and transfer vehicles, along with related compositions and methods of treatment. The circular RNAs can comprise group I intron fragments, spacers, an IRES, duplex forming regions, and/or an expression sequence, thereby having the features of improved expression, functional stability, low immunogenicity, ease of manufacturing, and/or extended half-life compared to linear RNA. Pharmaceutical compositions comprising such circular RNAs and transfer vehicles are particularly suitable for efficient protein expression in immune cells in vivo. Also disclosed are precursor RNAs and materials useful in producing the precursor or circular RNAs, which have improved circularization efficiency and/or are compatible with effective circular RNA purification methods.