公开(公告)号：US20190092828A1

公开(公告)日：2019-03-28

申请号：US16049212

申请日：2018-07-30

Applicant: ModernaTX, Inc.

Inventor： Stephen G. HOGE ,  William Joseph ISSA ,  Edward John MIRACCO ,  Jennifer NELSON ,  John REYNDERS ,  Matthew STANTON

IPC: C07K14/535 ,  C07K14/505 ,  C12P19/34 ,  A61K48/00 ,  C12N9/02 ,  C12P21/02

Abstract: The present disclosure provides alternative nucleosides, nucleotides, and nucleic acids, and methods of using them. In some aspects, the disclosure provides mRNA wherein the uracil content has been modified and which may be particularly effective for use in therapeutic compositions, because they may benefit from both high expression levels and limited induction of the innate immune response. In some aspects, the disclosure provides methods for the production of pharmaceutical compositions including mRNA without reverse phase chromatography.

公开(公告)号：US20220298516A1

公开(公告)日：2022-09-22

申请号：US17276983

申请日：2019-09-20

Applicant: ModernaTX, Inc.

Inventor： Aaron LARSEN ,  Jennifer NELSON ,  Melissa MOORE

IPC: C12N15/67 ,  C12N15/88 ,  A61K47/00 ,  A61K31/7088 ,  C12N15/11

Abstract: The present disclosure relates to methods and compositions for modulating protein expression. In particular, the invention features methods and compositions for increasing protein expression in a cell by delivering to the cell a composition including an mRNA encoding a polypeptide and one or more oligonucleotides, wherein each of the one or more oligonucleotides includes a region of linked nucleotides complimentary to a portion of the sequence of the mRNA. The methods and compositions described herein may be used to modulate gene expression (e.g., increase gene expression), to increase the stability of the mRNA, to decrease the immunogenicity of the mRNA, to enable selective expression (e.g., in a target cell or tissue) of the mRNA, and/or to enable the delivery of two or more mRNAs in a stoichiometric ratio.