公开(公告)号：US20180051262A1

公开(公告)日：2018-02-22

申请号：US15806096

申请日：2017-11-07

Applicant: Arcturus Therapeutics, Inc.

Inventor： Padmanabh Chivukula ,  Luigi Warren ,  Kiyoshi Tachikawa ,  Joseph E. Payne

IPC: C12N9/10 ,  C07K14/445 ,  C07K14/46 ,  C07K14/47 ,  C12N9/88 ,  C12N9/64 ,  C12N9/44 ,  C12N9/12 ,  C12N9/02 ,  C07K14/81 ,  C07K14/805 ,  C07K14/745 ,  C07K14/705 ,  C07K14/575 ,  C07K14/52 ,  C07K14/505 ,  A61K48/00 ,  A61K39/00

Abstract: This invention provides a range of translatable messenger UNA (mUNA) molecules. The mUNA molecules can be translated in vitro and in vivo to provide an active polypeptide or protein, or to provide an immunization agent or vaccine component. The mUNA molecules can be used as an active agent to express an active polypeptide or protein in cells or subjects. Among other things, the mUNA molecules are useful in methods for treating rare diseases.

公开(公告)号：US20150307881A1

公开(公告)日：2015-10-29

申请号：US14667680

申请日：2015-03-25

Applicant: Arcturus Therapeutics, Inc.

Inventor： Kiyoshi Tachikawa ,  Joseph E. Payne ,  Padmanabh Chivukula

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

CPC classification number: C12N15/113 ,  A61K31/713 ,  A61K47/14 ,  C12N2310/14 ,  C12N2310/323

Abstract: This invention provides UNA oligomers for gene silencing with reduced off-target effects. The UNA oligomers can have a first strand and a second strand, each of the strands being 19-29 monomers in length, the monomers being UNA monomers and various nucleic acid monomers. Embodiments include pharmaceutical compositions and methods for treating or preventing TTR-related amyloidosis with reduced off-target effects by administering a UNA oligomer to a subject.

Abstract translation: 本发明提供用于基因沉默的UNA寡聚体，具有降低的脱靶效应。 UNA低聚物可以具有第一链和第二链，每条链长度为19-29个单体，单体为UNA单体和各种核酸单体。 实施方案包括通过向受试者施用UNA寡聚体来治疗或预防TTR相关淀粉样变性的药物组合物和方法，其具有减少的脱靶效应。