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：US20190000932A1

公开(公告)日：2019-01-03

申请号：US16110788

申请日：2018-08-23

Applicant: ModernaTX, Inc.

Inventor： Paolo MARTINI ,  Stephen G. HOGE ,  Kerry BENENATO ,  Vladimir PRESNYAK ,  Iain MCFADYEN ,  Ellalahewage Sathyajith KUMARASINGHE ,  Xuling ZHU ,  Lin Tung GUEY ,  Staci SABNIS

IPC: A61K38/47 ,  A61K9/51 ,  A61P3/00

Abstract: The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the α-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.

公开(公告)号：US20170202979A1

公开(公告)日：2017-07-20

申请号：US15326266

申请日：2015-07-17

Applicant: ModernaTX, Inc.

Inventor： Tirtha CHAKRABORTY ,  Stephen G. HOGE

IPC: A61K48/00 ,  A61K38/18 ,  C12N15/85 ,  C07K14/535 ,  C07K14/505 ,  C12N15/113 ,  A61K38/19 ,  C12N15/67

CPC classification number: A61K48/0066 ,  A61K38/1816 ,  A61K38/193 ,  C07H21/04 ,  C07K14/505 ,  C07K14/535 ,  C12N15/113 ,  C12N15/1136 ,  C12N15/67 ,  C12N15/85 ,  C12N2310/141 ,  C12N2310/3519 ,  C12N2840/102 ,  C12N2840/105

Abstract: The present invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides comprising at least one terminal modification.