公开(公告)号：US11590157B2

公开(公告)日：2023-02-28

申请号：US17036374

申请日：2020-09-29

Applicant: ModernaTX, Inc.

Inventor： Stephen Hoge ,  Tirtha Chakraborty ,  Gilles Besin ,  Ruchi Jain

IPC: C07H21/02 ,  A61K31/7115 ,  C12N15/67 ,  A61K9/00 ,  A61K9/127 ,  C12N15/113

Abstract: The disclosure features methods of reducing or inhibiting an anti-drug antibody response in a subject, as well as methods of reducing or inhibiting unwanted immune cell activation in a subject to be treated with a messenger RNA (mRNA), comprising administering to the subject a mRNA, e.g., a chemically modified messenger RNA (mmRNA), encoding a polypeptide of interest, wherein the mRNA comprises at least one microRNA (miR) binding site for a miR expressed in immune cells, such as miR-126 binding site and/or miR-142 binding site, such that an anti-drug antibody response to the polypeptide or interest, or unwanted immune cell activation (e.g., B cell activation, cytokine secretion), is reduced or inhibited in the subject. The disclosure further provides therapeutic treatment regimens designed to reduce or inhibit ADA or unwanted immune cell activation (e.g., B cell activation, cytokine secretion) in a subject being treated with mRNA-based therapeutics.

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：US10849920B2

公开(公告)日：2020-12-01

申请号：US15761220

申请日：2016-10-05

Applicant: ModernaTX, Inc.

Inventor： Stephen Hoge ,  Tirtha Chakraborty ,  Gilles Besin ,  Ruchi Jain

IPC: A61K48/00 ,  C07H21/04 ,  A61K31/7115 ,  C12N15/67 ,  A61K9/00 ,  A61K9/127 ,  C12N15/113

Abstract: The disclosure features methods of reducing or inhibiting an anti-drug antibody response in a subject, as well as methods of reducing or inhibiting unwanted immune cell activation in a subject to be treated with a messenger RNA (mRNA), comprising administering to the subject a mRNA, e.g., a chemically modified messenger RNA (mmRNA), encoding a polypeptide of interest, wherein the mRNA comprises at least one microRNA (miR) binding site for a miR expressed in immune cells, such as miR-126 binding site and/or miR-142 binding site, such that an anti-drug antibody response to the polypeptide or interest, or unwanted immune cell activation (e.g., B cell activation, cytokine secretion), is reduced or inhibited in the subject. The disclosure further provides therapeutic treatment regimens designed to reduce or inhibit AD A or unwanted immune cell activation (e.g., B cell activation, cytokine secretion) in a subject being treated with mRNA-based therapeutics.

公开(公告)号：US12241063B2

公开(公告)日：2025-03-04

申请号：US17933500

申请日：2022-09-20

Applicant: ModernaTX, Inc.

Inventor： Melissa J. Moore ,  Caroline Köhrer ,  Ruchi Jain ,  Vladimir Presnyak

IPC: C07H21/02 ,  A61K47/69 ,  C12N15/11 ,  C12N15/85 ,  C12N15/88

Abstract: The present disclosure provides messenger RNAs (mRNAs) having chemical and/or structural modifications, including RNA elements and/or modified nucleotides, which provide a desired translational regulatory activity to the mRNA.

公开(公告)号：US20230173104A1

公开(公告)日：2023-06-08

申请号：US17924456

申请日：2021-05-14

Applicant: ModernaTX, Inc.

Inventor： Ruchi Jain ,  Alicia Anne Bicknell

IPC: A61K48/00 ,  A61K9/51 ,  A61K38/17 ,  A61K38/16

CPC classification number: A61K48/0058 ,  A61K9/5123 ,  A61K48/0033 ,  A61K38/1709 ,  A61K9/5146 ,  A61K38/162

Abstract: The disclosure features LNP compositions and systems comprising a therapeutic payload or prophylactic payload, a binding element, a tether molecule and/or an effector molecule and uses thereof. The LNP compositions or systems of the present disclosure comprise: (a) a first polynucleotide (e.g., mRNA) comprising: (1) a sequence encoding a therapeutic payload or prophylactic payload, and (2) a binding element; and (b) a second polynucleotide (e.g., mRNA) comprising a sequence encoding: (1) an effector molecule, and/or (2) a polypeptide that recognizes the binding element (a tether molecule). Such compositions or systems can: increase the level and/or activity of the therapeutic payload or prophylactic payload, e.g., increase the level, stability and/or activity of the mRNA encoding the therapeutic payload or prophylactic payload. Also disclosed herein are methods of treating a disorder, or for modulating an immune response in a subject using the disclosed LNP compositions or systems.

公开(公告)号：US20240384277A1

公开(公告)日：2024-11-21

申请号：US18569888

申请日：2022-06-14

Applicant: ModernaTX, Inc.

Inventor： Ruchi Jain ,  Brian Robert Fritz ,  Mihir Metkar

IPC: C12N15/63 ,  C12N9/22 ,  C12N15/88

Abstract: The disclosure features compositions or systems and uses thereof, comprising a first polynucleotide encoding a target molecule, optionally a second polynucleotide encoding an effector, repressor, or endonuclease molecule; optionally a recognition or cleavage site in the first or second polynucleotide, and optionally a repressor/effector binding site in the first polynucleotide. The compositions or systems of the present disclosure can increase the level and/or activity of the target molecule in desired cells and/or microenvironments while suppressing the level and/or activity of the target molecule in off-target cells and/or microenvironments.

公开(公告)号：US20240269248A1

公开(公告)日：2024-08-15

申请号：US18560498

申请日：2022-05-19

Applicant: ModernaTX, Inc.

Inventor： Husain Attarwala ,  Kimberly Ann Coughlan ,  Ruchi Jain ,  Matthew Lumley ,  Vladimir Presnyak

IPC: A61K38/52 ,  A61K9/00 ,  A61K9/51 ,  A61K31/167 ,  A61K48/00 ,  A61P3/00

CPC classification number: A61K38/52 ,  A61K9/0019 ,  A61K9/5123 ,  A61K31/167 ,  A61K48/0033 ,  A61P3/00

Abstract: This disclosure relates to mRNA therapy for the treatment of methylmalonic acidemia (MMA). mRNAs for use in the invention, when administered in vivo, encode methylmalonyl-CoA mutase (MUT). mRNA therapies of the disclosure increase and/or restore deficient levels of MUT expression and/or activity in subjects.

公开(公告)号：US11485972B2

公开(公告)日：2022-11-01

申请号：US16614245

申请日：2018-05-18

Applicant: ModernaTX, Inc.

Inventor： Melissa J. Moore ,  Caroline Köhrer ,  Ruchi Jain ,  Vladimir Presnyak

IPC: C07H21/02 ,  C07H21/04 ,  C12N15/11 ,  A61K47/69 ,  C12N15/85 ,  C12N15/88

Abstract: The present disclosure provides messenger RNAs (mRNAs) having chemical and/or structural modifications, including RNA elements and/or modified nucleotides, which provide a desired translational regulatory activity to the mRNA.

公开(公告)号：US12246030B2

公开(公告)日：2025-03-11

申请号：US18167984

申请日：2023-02-13

Applicant: ModernaTX, Inc.

Inventor： Stephen Hoge ,  Tirtha Chakraborty ,  Gilles Besin ,  Ruchi Jain

IPC: C07H21/04 ,  A61K9/00 ,  A61K9/127 ,  A61K31/7115 ,  C12N15/113 ,  C12N15/67

Abstract: The disclosure features methods of reducing or inhibiting an anti-drug antibody response in a subject, as well as methods of reducing or inhibiting unwanted immune cell activation in a subject to be treated with a messenger RNA (mRNA), comprising administering to the subject a mRNA, e.g., a chemically modified messenger RNA (mmRNA), encoding a polypeptide of interest, wherein the mRNA comprises at least one microRNA (miR) binding site for a miR expressed in immune cells, such as miR-126 binding site and/or miR-142 binding site, such that an anti-drug antibody response to the polypeptide or interest, or unwanted immune cell activation (e.g., B cell activation, cytokine secretion), is reduced or inhibited in the subject. The disclosure further provides therapeutic treatment regimens designed to reduce or inhibit ADA or unwanted immune cell activation (e.g., B cell activation, cytokine secretion) in a subject being treated with mRNA-based therapeutics.

公开(公告)号：US20240226025A1

公开(公告)日：2024-07-11

申请号：US18282676

申请日：2022-03-24

Applicant: ModernaTX, Inc.

Inventor： Kerry Benenato ,  Mark Cornebise ,  Edward Hennessy ,  Ruchi Jain ,  Vladimir Presnyak

IPC: A61K9/51 ,  A61K38/52 ,  A61P3/00 ,  C12N9/90

CPC classification number: A61K9/5123 ,  A61K38/52 ,  A61P3/00 ,  C12N9/90 ,  C12Y504/99002

Abstract: This disclosure relates to messenger RNA (mRNA) therapy for the treatment of methylmalonic acidemia. mRNAs for use in the invention, when administered in vivo, encode methylmalonyl-CoA mutase. mRNA therapies of the disclosure increase and/or restore deficient levels of MUT expression and/or activity in subjects.