公开(公告)号：US20240376445A1

公开(公告)日：2024-11-14

申请号：US18572490

申请日：2022-06-22

Applicant: ModernaTX, Inc.

Inventor： David Reid

IPC: C12N9/10 ,  A61K9/00 ,  A61K9/51 ,  A61K38/00 ,  A61P3/00

Abstract: This disclosure relates to mRNA therapy for the treatment of Crigler-Najjar Syndrome Type 1 (CN1). mRNAs for use in the invention, when administered in vivo, encode uridine diphosphate glycosyltransferase 1 family, polypeptide A1 (UGT1A1). mRNA therapies of the disclosure increase and/or restore deficient levels of UGT1A1 expression and/or activity in subjects. mRNA therapies of the disclosure further decrease abnormal accumulation of bilirubin associated with deficient UGT1A1 activity in subjects.

公开(公告)号：US20240229109A1

公开(公告)日：2024-07-11

申请号：US18284938

申请日：2022-03-31

Applicant: ModernaTX, Inc.

Inventor： Amy E. Rabideau ,  David Reid ,  Huijuan Li ,  Kristian Link ,  Tao Jiang ,  Eva-Maria Schneeberger

IPC: C12Q1/6825 ,  C12Q1/6806 ,  C12Q1/6876

CPC classification number: C12Q1/6825 ,  C12Q1/6876 ,  C12Q1/6806 ,  C12Q2600/16

Abstract: Aspects of the disclosure relate to methods for analyzing compositions comprising RNA species with unique nucleotide sequences for identification and/or ratio determination of RNAs. The disclosure is based, in part, on methods of cleaving identifying sequences from RNAs in a composition, and detecting the abundance of each identifying sequence to quantify the abundance of corresponding RNA species. Other aspects relate to methods for producing compositions comprising more than two RNA species, such as multivalent RNA compositions. The disclosure is based, in part, on methods of determining the proper amount of input DNA for in vitro transcription (IVT) reactions that will result in RNA being transcribed in a predetermined ratio. In some aspects, the disclosure relates to pharmaceutical compositions comprising multivalent RNA compositions produced by methods described, by the disclosure.

公开(公告)号：US20250017867A1

公开(公告)日：2025-01-16

申请号：US18695076

申请日：2022-09-30

Applicant: ModernaTX, Inc.

Inventor： Michael Albert Zimmer ,  Xinhua Yan ,  Mihir Metkar ,  David Reid

IPC: A61K9/51 ,  A61K9/127 ,  A61K31/7105 ,  A61K38/22

Abstract: This disclosure relates to mRNA therapy for the treatment of relaxin-related diseases, disorders or conditions such as, e.g., fibrosis or cardiovascular disease (e.g., acute heart failure or acute coronary syndrome). mRNAs for use in the invention, when administered in vivo, encode relaxin. mRNA therapies of the disclosure increase and/or restore deficient levels of relaxin expression and/or activity in subjects.

公开(公告)号：US20240189449A1

公开(公告)日：2024-06-13

申请号：US18282647

申请日：2022-03-24

Applicant: ModernaTX, Inc.

Inventor： Kerry Benenato ,  Alicia Anne Bicknell ,  Mark Cornebise ,  Athanasios Dousis ,  Andrew Jacob Giessel ,  Edward Hennessy ,  Ruchi Jain ,  Caroline Köhrer ,  Stuart Spencer Licht ,  Kanchana Ravichandran ,  David Reid

IPC: A61K48/00 ,  A61K9/127 ,  A61K9/51 ,  A61K31/7105 ,  A61P7/00 ,  C12N9/10 ,  C12N15/67

CPC classification number: A61K48/005 ,  A61K9/1271 ,  A61K9/5123 ,  A61K31/7105 ,  A61K48/0041 ,  A61P7/00 ,  C12N9/1018 ,  C12N15/67 ,  C12Y201/03003

Abstract: This disclosure relates to mRNA therapy for the treatment of ornithine transcarbamylase deficiency (OTCD). mRNAs for use in the invention, when administered in vivo, encode human ornithine transcarbamylase (OTC). mRNA therapies of the disclosure increase and/or restore deficient levels of OTC expression and/or activity in subjects. mRNA therapies of the disclosure further decrease levels of toxic ammonia associated with deficient OTC activity in subjects.

公开(公告)号：US20240100151A1

公开(公告)日：2024-03-28

申请号：US18272512

申请日：2022-01-14

Applicant: ModernaTX, Inc.

Inventor： Andrea Carfi ,  Guillaume Stewart-Jones ,  Hamilton Bennett ,  Kai Wu ,  Darin Edwards ,  Gwo-Yu Chuang ,  David Reid

IPC: A61K39/215 ,  A61K9/127 ,  A61P31/14

CPC classification number: A61K39/215 ,  A61K9/1272 ,  A61P31/14 ,  A61K2039/53 ,  C12N2770/20034

Abstract: The disclosure provides coronavirus mRNA vaccines, including vaccines directed against one or more variant strains of SARS-CoV-2, as well as methods of using the vaccines.

公开(公告)号：US20230242908A1

公开(公告)日：2023-08-03

申请号：US18012094

申请日：2021-06-23

Applicant: ModernaTX, Inc.

Inventor： David Reid ,  Ruchi Jain ,  Alicia Bicknell ,  Caroline Kohrer

IPC: C12N15/11 ,  A61K9/16 ,  C12N15/88 ,  C12N15/67

CPC classification number: C12N15/11 ,  A61K9/1617 ,  C12N15/88 ,  C12N15/67

Abstract: The disclosure features a polynucleotide encoding a polypeptide, which polynucleotide comprises: a 5′UTR described herein; a coding region comprising a payload and a stop element described herein; and a 3′UTR described herein, and LNP compositions comprising the same. The polynucleotides and/or LNP compositions of the present disclosure can: increase the level and/or activity of the payload by increasing the half-life and/or duration of expression of the polynucleotide encoding the payload or of the payload polypeptide. Also disclosed herein are methods of treating a disease or disorder in a subject using the LNP compositions of the present disclosure.

公开(公告)号：US20240139309A1

公开(公告)日：2024-05-02

申请号：US18272496

申请日：2022-01-14

Applicant: ModernaTX, Inc.

Inventor： Andrea Carfi ,  Guillaume Stewart-Jones ,  Hamiltion Bennett ,  Kai Wu ,  Darin Edwards ,  Gwo-Yu Chuang ,  David Reid

IPC: A61K39/215 ,  A61P31/14 ,  C07K14/005

CPC classification number: A61K39/215 ,  A61P31/14 ,  C07K14/005 ,  A61K2039/53 ,  C12N2770/20034

Abstract: The disclosure provides coronavirus mRNA vaccines, including vaccines directed against spike proteins of one or more variant strains of SARS-CoV-2, as well as methods of using the vaccines.

公开(公告)号：US20230406895A1

公开(公告)日：2023-12-21

申请号：US18036560

申请日：2021-11-12

Applicant: ModernaTX, Inc.

Inventor： Jean C. Sung ,  David Reid ,  Alicia Anne Bicknell ,  Ana Cadete Pires ,  Jeffrey Hrkach

IPC: C07K14/47 ,  A61K47/69 ,  A61K47/10 ,  A61P11/00

CPC classification number: C07K14/4712 ,  A61K47/6909 ,  A61K47/10 ,  A61P11/00

Abstract: This disclosure relates to delivery vehicles comprising payload molecules, e.g., mRNA or gene editing therapeutics for the treatment of cystic fibrosis (CF). Nucleic acid therapeutics (e.g., mRNAs) for use in the invention, when administered in vivo, encode cystic fibrosis transmembrane conductance regulator (CFTR). Nucleic acid therapeutics (e.g., mRNAs) of the disclosure increase and/or restore deficient levels of CFTR expression and/or activity in subjects. Nucleic acid therapeutics (e.g., mRNAs) of the disclosure further decrease abnormal accumulation of ammonia associated with deficient CFTR activity in subjects.