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公开(公告)号：US11066686B2
公开(公告)日：2021-07-20
申请号：US16657122
申请日：2019-10-18
Applicant: ModernaTX, Inc.
Inventor： Amy E. Rabideau , Athanasios Dousis , Kanchana Ravichandran , Elissa Hobert
Abstract: The present disclosure provides, in some aspects, variant RNA polymerases, the use of which increases transcription efficiency while reducing the number of double-stranded RNA contaminates and run-on transcripts produced during an in vitro transcription reaction.
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公开(公告)号：US20210040456A1
公开(公告)日：2021-02-11
申请号：US16765656
申请日：2018-11-21
Applicant: ModernaTX, Inc.
Inventor： Raj Rajendran , Patrick Finn , Paolo G.V. Martini , Ding An , Athanasios Dousis , Kanchana Ravichandran
Abstract: This disclosure relates to mRNA therapy for the treatment of hyperphenylalaninemias such as phenylketonuria (PKU). mRNAs for use in the invention, when administered in vivo, encode human phenylalanine hydroxylase (PAH), functional fragments thereof (e.g., those comprising the catalytic domain or the catalytic domain and the tetramerization domains), and fusion proteins comprising PAH. 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 PAH expression and/or activity in subjects. mRNA therapies of the invention further decrease abnormal accumulation of phenylalanine associated with deficient PAH activity in subjects.
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公开(公告)号：US10526629B2
公开(公告)日：2020-01-07
申请号：US16432541
申请日：2019-06-05
Applicant: ModernaTX, Inc.
Inventor： Amy E. Rabideau , Athanasios Dousis , Kanchana Ravichandran , Elissa Hobert
Abstract: The present disclosure provides, in some aspects, variant RNA polymerases, the use of which increases transcription efficiency while reducing the number of double-stranded RNA contaminates and run-on transcripts produced during an in vitro transcription reaction.
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公开(公告)号：US20190309337A1
公开(公告)日：2019-10-10
申请号：US16432541
申请日：2019-06-05
Applicant: ModernaTX, Inc.
Inventor： Amy E. Rabideau , Athanasios Dousis , Kanchana Ravichandran , Elissa Hobert
IPC: C12P19/34
Abstract: The present disclosure provides, in some aspects, variant RNA polymerases, the use of which increases transcription efficiency while reducing the number of double-stranded RNA contaminates and run-on transcripts produced during an in vitro transcription reaction.
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公开(公告)号：US20240102066A1
公开(公告)日：2024-03-28
申请号：US18448856
申请日：2023-08-11
Applicant: ModernaTX, Inc.
Inventor： Amy E. Rabideau , Athanasios Dousis , Kanchana Ravichandran , Elissa Hobert
CPC classification number: C12P19/34 , C12N9/1247 , C12Y207/11023
Abstract: The present disclosure provides, in some aspects, variant RNA polymerases, the use of which increases transcription efficiency while reducing the number of double-stranded RNA contaminates and run-on transcripts produced during an in vitro transcription reaction.
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Patent Agency Ranking
公开(公告)号：US11939601B2
公开(公告)日：2024-03-26
申请号：US16765656
申请日：2018-11-21
Applicant: ModernaTX, Inc.
Inventor： Raj Rajendran , Patrick Finn , Paolo G. V. Martini , Ding An , Athanasios Dousis , Kanchana Ravichandran
CPC classification number: C12N9/0071 , A61K9/0019 , A61K9/5123 , A61K48/0075 , A61P3/00 , C12Y114/16001
Abstract: This disclosure relates to mRNA therapy for the treatment of hyperphenylalaninemias such as phenylketonuria (PKU). mRNAs for use in the invention, when administered in vivo, encode human phenylalanine hydroxylase (PAH), functional fragments thereof (e.g., those comprising the catalytic domain or the catalytic domain and the tetramerization domains), and fusion proteins comprising PAH. 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 PAH expression and/or activity in subjects. mRNA therapies of the invention further decrease abnormal accumulation of phenylalanine associated with deficient PAH activity in subjects.
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公开(公告)号：US11485960B2
公开(公告)日：2022-11-01
申请号：US17325883
申请日：2021-05-20
Applicant: ModernaTX, Inc.
Inventor： Athanasios Dousis , Kanchana Ravichandran , Amy E. Rabideau , Margaret Franklin , Kevin Smith , Michelle Lynn Hall
Abstract: The present disclosure provides RNA polymerase variants for high efficiency transcription.
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公开(公告)号：US20210395792A1
公开(公告)日：2021-12-23
申请号：US17350662
申请日：2021-06-17
Applicant: ModernaTX, Inc.
Inventor： Amy E. Rabideau , Athanasios Dousis , Kanchana Ravichandran , Elissa Hobert
Abstract: The present disclosure provides, in some aspects, variant RNA polymerases, the use of which increases transcription efficiency while reducing the number of double-stranded RNA contaminates and run-on transcripts produced during an in vitro transcription reaction.
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公开(公告)号：US20210309976A1
公开(公告)日：2021-10-07
申请号：US17325883
申请日：2021-05-20
Applicant: ModernaTX, Inc.
Inventor： Athanasios Dousis , Kanchana Ravichandran , Amy E. Rabideau , Margaret Franklin , Kevin Smith
Abstract: The present disclosure provides RNA polymerase variants for high efficiency transcription.
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公开(公告)号：US20210217484A1
公开(公告)日：2021-07-15
申请号：US17145164
申请日：2021-01-08
Applicant: ModernaTX, Inc.
Inventor： Andrew Giessel , Athanasios Dousis , Iain McFadyen
Abstract: Techniques for manufacturing a variant of a target protein. The techniques may include accessing a latent variable statistical model (LVSM) configured to generate output indicating one or more biological sequences corresponding to one or more variants of the target protein and using the LVSM to generate a first output indicating a first biological sequence associated with a first variant of the target protein. The techniques further include manufacturing, using the first biological sequence, a first biological molecule to produce the first variant of the target protein.
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