[go: up one dir, main page]

EP2755986A4 - MODIFIED NUCLEIC ACIDS AND METHODS OF USE - Google Patents

MODIFIED NUCLEIC ACIDS AND METHODS OF USE

Info

Publication number
EP2755986A4
EP2755986A4 EP12831509.0A EP12831509A EP2755986A4 EP 2755986 A4 EP2755986 A4 EP 2755986A4 EP 12831509 A EP12831509 A EP 12831509A EP 2755986 A4 EP2755986 A4 EP 2755986A4
Authority
EP
European Patent Office
Prior art keywords
methods
nucleic acids
modified nucleic
modified
acids
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Withdrawn
Application number
EP12831509.0A
Other languages
German (de)
French (fr)
Other versions
EP2755986A1 (en
Inventor
Stephane Bancel
Jason P Schrum
Alexander Aristarkhov
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Moderna Inc
Original Assignee
Moderna Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Moderna Therapeutics Inc filed Critical Moderna Therapeutics Inc
Publication of EP2755986A1 publication Critical patent/EP2755986A1/en
Publication of EP2755986A4 publication Critical patent/EP2755986A4/en
Withdrawn legal-status Critical Current

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0091Purification or manufacturing processes for gene therapy compositions
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4723Cationic antimicrobial peptides, e.g. defensins

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Zoology (AREA)
  • Toxicology (AREA)
  • Manufacturing & Machinery (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
EP12831509.0A 2011-09-12 2012-09-11 MODIFIED NUCLEIC ACIDS AND METHODS OF USE Withdrawn EP2755986A4 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201161533537P 2011-09-12 2011-09-12
PCT/US2012/054561 WO2013039857A1 (en) 2011-09-12 2012-09-11 Engineered nucleic acids and methods of use thereof

Publications (2)

Publication Number Publication Date
EP2755986A1 EP2755986A1 (en) 2014-07-23
EP2755986A4 true EP2755986A4 (en) 2015-05-20

Family

ID=47883633

Family Applications (1)

Application Number Title Priority Date Filing Date
EP12831509.0A Withdrawn EP2755986A4 (en) 2011-09-12 2012-09-11 MODIFIED NUCLEIC ACIDS AND METHODS OF USE

Country Status (2)

Country Link
EP (1) EP2755986A4 (en)
WO (1) WO2013039857A1 (en)

Families Citing this family (184)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2666559T3 (en) 2009-12-01 2018-05-07 Translate Bio, Inc. Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
EP2600901B1 (en) 2010-08-06 2019-03-27 ModernaTX, Inc. A pharmaceutical formulation comprising engineered nucleic acids and medical use thereof
CN104531812A (en) 2010-10-01 2015-04-22 现代治疗公司 Engineered nucleic acids and methods of use thereof
WO2012075040A2 (en) 2010-11-30 2012-06-07 Shire Human Genetic Therapies, Inc. mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES
US8710200B2 (en) 2011-03-31 2014-04-29 Moderna Therapeutics, Inc. Engineered nucleic acids encoding a modified erythropoietin and their expression
BR112013031553A2 (en) 2011-06-08 2020-11-10 Shire Human Genetic Therapies, Inc. compositions, mrna encoding a gland and its use, use of at least one mrna molecule and a vehicle for transfer and use of an mrna encoding for exogenous protein
US10323236B2 (en) 2011-07-22 2019-06-18 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
BR112014007852B1 (en) 2011-10-03 2021-11-03 Moderna Therapeutics, Inc MODIFIED ISOLATED POLYNUCLEOTIDE AND PHARMACEUTICAL COMPOSITION
PL2791160T3 (en) 2011-12-16 2022-06-20 Modernatx, Inc. Modified mrna compositions
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
HK1206779A1 (en) 2012-04-02 2016-01-15 Modernatx, Inc. Modified polynucleotides for the production of nuclear proteins
AU2013243949A1 (en) * 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and proteins associated with human disease
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
US10501513B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
EP2859102A4 (en) 2012-06-08 2016-05-11 Shire Human Genetic Therapies Nuclease resistant polynucleotides and uses thereof
CA2876155C (en) 2012-06-08 2022-12-13 Ethris Gmbh Pulmonary delivery of mrna to non-lung target cells
US9512456B2 (en) 2012-08-14 2016-12-06 Modernatx, Inc. Enzymes and polymerases for the synthesis of RNA
DK2922554T3 (en) 2012-11-26 2022-05-23 Modernatx Inc Terminalt modificeret rna
AU2013360738B2 (en) * 2012-12-18 2018-08-30 University College Dublin, National University Of Ireland, Dublin A vaccine for treatment or prevention of Burkholderia infection in a mammal
WO2014153052A2 (en) 2013-03-14 2014-09-25 Shire Human Genetic Therapies, Inc. Cftr mrna compositions and related methods and uses
HUE055044T2 (en) 2013-03-14 2021-10-28 Translate Bio Inc Methods and compositions for delivering mrna coded antibodies
EP4446413A3 (en) 2013-03-14 2024-12-18 Translate Bio, Inc. Methods for purification of messenger rna
EP2971010B1 (en) 2013-03-14 2020-06-10 ModernaTX, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
WO2014144767A1 (en) 2013-03-15 2014-09-18 Moderna Therapeutics, Inc. Ion exchange purification of mrna
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
US10138507B2 (en) 2013-03-15 2018-11-27 Modernatx, Inc. Manufacturing methods for production of RNA transcripts
US11377470B2 (en) 2013-03-15 2022-07-05 Modernatx, Inc. Ribonucleic acid purification
ES2795249T3 (en) 2013-03-15 2020-11-23 Translate Bio Inc Synergistic enhancement of nucleic acid delivery through mixed formulations
US10077439B2 (en) 2013-03-15 2018-09-18 Modernatx, Inc. Removal of DNA fragments in mRNA production process
PT3019619T (en) 2013-07-11 2021-11-11 Modernatx Inc COMPOSITIONS COMPRISING SYNTHETIC POLYNUCLEOTIDES ENCODING SYNTHETIC CRISPR AND SGARN-RELATED PROTEINS AND METHODS OF USE
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
EP3052511A4 (en) 2013-10-02 2017-05-31 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
WO2015051214A1 (en) 2013-10-03 2015-04-09 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor
AU2014340155B2 (en) 2013-10-22 2018-11-01 Massachusetts Institute Of Technology Lipid formulations for delivery of messenger RNA
CN105658242A (en) 2013-10-22 2016-06-08 夏尔人类遗传性治疗公司 MRNA therapy for phenylketonuria
ES2707966T3 (en) 2013-10-22 2019-04-08 Translate Bio Inc MRNA therapy for the deficiency in argininosuccinate synthesis
EP4036241A1 (en) 2013-10-22 2022-08-03 Translate Bio, Inc. Cns delivery of mrna and uses thereof
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
WO2015098963A1 (en) * 2013-12-26 2015-07-02 東亞合成株式会社 Method for promoting expression of calreticulin, and synthetic peptide for use in method for promoting expression of calreticulin
EP3096616B1 (en) * 2014-01-24 2020-04-29 The Regents of the University of Colorado, a Body Corporate Dermaseptin-type and piscidin-type antimicrobial peptides
US9919034B2 (en) 2014-03-28 2018-03-20 Tamir Biotechnology, Inc. Methods of treating and prophylactically protecting mammalian patients infected by viruses classified in Baltimore group V
CN106164248B (en) 2014-04-25 2019-10-15 川斯勒佰尔公司 Purification of messenger RNA
CN106659731A (en) 2014-05-30 2017-05-10 夏尔人类遗传性治疗公司 Topical compositions and methods for treating wounds
JP6599373B2 (en) 2014-06-24 2019-10-30 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド Stereochemically enriched compositions for delivery of nucleic acids
US9668980B2 (en) 2014-07-02 2017-06-06 Rana Therapeutics, Inc. Encapsulation of messenger RNA
EP3169335B8 (en) 2014-07-16 2019-10-09 ModernaTX, Inc. Circular polynucleotides
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
US10835598B2 (en) 2014-08-18 2020-11-17 Orgenesis Inc. Prophylactic protection against viral infections, particularly HIV
CA2962945A1 (en) 2014-10-01 2016-04-07 Plant Health Care, Inc. Elicitor peptides having disrupted hypersensitive response box and use thereof
MX2017004146A (en) 2014-10-01 2017-09-05 Plant Health Care Inc Hypersensitive response elicitor peptides and use thereof.
EP3205347A4 (en) * 2014-10-08 2018-06-06 Keio University White blood cell extracellular trap formation inhibitor
TWI686408B (en) 2014-10-27 2020-03-01 中央研究院 Plant defense signaling peptides and applications thereof
WO2016077123A1 (en) 2014-11-10 2016-05-19 Moderna Therapeutics, Inc. Multiparametric nucleic acid optimization
EP3226912B1 (en) 2014-12-05 2021-01-20 Translate Bio, Inc. Messenger rna therapy for treatment of articular disease
US10758558B2 (en) 2015-02-13 2020-09-01 Translate Bio Ma, Inc. Hybrid oligonucleotides and uses thereof
JP6895892B2 (en) 2015-03-19 2021-06-30 トランスレイト バイオ, インコーポレイテッド MRNA treatment for Pompe disease
AU2016277827B2 (en) * 2015-06-15 2021-04-29 Orgenesis Inc. Pharmaceuticals for treatment of viral infections of the eye
AU2016287754B2 (en) * 2015-07-02 2021-02-25 Dana-Farber Cancer Institute, Inc. Stabilized anti-microbial peptides
WO2017049286A1 (en) 2015-09-17 2017-03-23 Moderna Therapeutics, Inc. Polynucleotides containing a morpholino linker
AU2016324463B2 (en) 2015-09-17 2022-10-27 Modernatx, Inc. Polynucleotides containing a stabilizing tail region
ES2810701T5 (en) 2015-10-05 2024-07-11 Modernatx Inc Procedures for the therapeutic administration of messenger ribonucleic acid drugs
WO2017066573A1 (en) 2015-10-14 2017-04-20 Shire Human Genetic Therapies, Inc. Modification of rna-related enzymes for enhanced production
US10167457B2 (en) 2015-10-23 2019-01-01 President And Fellows Of Harvard College Nucleobase editors and uses thereof
EP3368089B1 (en) 2015-10-26 2025-11-05 Translate Bio Ma, Inc. Nanoparticle formulations for delivery of nucleic acid complexes
WO2017079505A1 (en) * 2015-11-04 2017-05-11 Research Institute At Nationwide Children's Hospital Rnase7 for treatment of bacterial infection
SI3394093T1 (en) 2015-12-23 2022-05-31 Modernatx, Inc. Methods of using ox40 ligand encoding polynucleotides
US20190241658A1 (en) 2016-01-10 2019-08-08 Modernatx, Inc. Therapeutic mRNAs encoding anti CTLA-4 antibodies
WO2017151617A1 (en) 2016-02-29 2017-09-08 Dana-Farber Cancer Institute, Inc. Stapled intracellular-targeting antimicrobial peptides to treat infection
CA3020343A1 (en) 2016-04-08 2017-10-12 Translate Bio, Inc. Multimeric coding nucleic acid and uses thereof
CA3024624A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding porphobilinogen deaminase for the treatment of acute intermittent porphyria
EP3458105B1 (en) 2016-05-18 2024-01-17 Modernatx, Inc. Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1
JP7114485B2 (en) 2016-05-18 2022-08-08 モデルナティエックス インコーポレイテッド Polynucleotides encoding α-galactosidase A for the treatment of Fabry disease
JP7210287B2 (en) 2016-05-18 2023-01-23 モダーナティエックス・インコーポレイテッド Polynucleotides encoding citrine for the treatment of type II citrullinemia
EP4137509A1 (en) 2016-05-18 2023-02-22 ModernaTX, Inc. Combinations of mrnas encoding immune modulating polypeptides and uses thereof
WO2017201332A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding acyl-coa dehydrogenase, very long-chain for the treatment of very long-chain acyl-coa dehydrogenase deficiency
WO2017201342A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding jagged1 for the treatment of alagille syndrome
SG11201809381XA (en) 2016-05-18 2018-12-28 Modernatx Inc Polynucleotides encoding interleukin-12 (il12) and uses thereof
US10835583B2 (en) 2016-06-13 2020-11-17 Translate Bio, Inc. Messenger RNA therapy for the treatment of ornithine transcarbamylase deficiency
US20190298796A1 (en) * 2016-06-15 2019-10-03 Rajiv Gandhi Centre for Biotechnology, an Autonom- ous Institute under the Department of Biotechnolo Therapeutic Compositions from the Brevinin-1 Family of Peptides and Uses Thereof
US12385034B2 (en) 2016-06-24 2025-08-12 Modernatx, Inc. Methods and apparatus for filtration
MX2019000205A (en) 2016-07-07 2019-09-23 Rubius Therapeutics Inc Compositions and methods related to therapeutic cell systems expressing exogenous rna.
KR20250103795A (en) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 Adenosine nucleobase editors and uses thereof
EP3497214B1 (en) 2016-08-09 2023-06-28 President and Fellows of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
EP3541405A4 (en) * 2016-11-21 2020-07-29 University of Pittsburgh - of The Commonwealth System of Higher Education Novel anti-microbial and anti-cancer therapy
AU2017377993A1 (en) * 2016-12-13 2019-07-04 Emory University Polypeptides for managing viral infections
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
US10093706B2 (en) 2017-01-30 2018-10-09 Indiana University Research And Technology Corporation Dominant positive hnRNP-E1 polypeptide compositions and methods
MX2019010155A (en) 2017-02-27 2020-12-10 Translate Bio Inc Novel codon-optimized cftr mrna.
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
BR112019019655A2 (en) 2017-03-23 2020-04-22 Harvard College nucleobase editors comprising nucleic acid programmable dna binding proteins
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
WO2018213476A1 (en) 2017-05-16 2018-11-22 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding cftr
US11421011B2 (en) 2017-05-18 2022-08-23 Modernatx, Inc. Polynucleotides encoding tethered interleukin-12 (IL12) polypeptides and uses thereof
WO2018231990A2 (en) 2017-06-14 2018-12-20 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase
US20200268666A1 (en) 2017-06-14 2020-08-27 Modernatx, Inc. Polynucleotides encoding coagulation factor viii
AU2018304230A1 (en) 2017-07-19 2020-02-06 Dana-Farber Cancer Institute, Inc. Stabilized anti-microbial peptides for the treatment of antibiotic-resistant bacterial infections
US11732274B2 (en) 2017-07-28 2023-08-22 President And Fellows Of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE)
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
AU2018352592C1 (en) 2017-10-16 2025-09-25 Beam Therapeutics, Inc. Uses of adenosine base editors
JP7423522B2 (en) 2017-11-22 2024-01-29 モダーナティエックス・インコーポレイテッド Polynucleotide encoding ornithine transcarbamylase for the treatment of urea cycle disorders
US11939601B2 (en) 2017-11-22 2024-03-26 Modernatx, Inc. Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria
EP3714045A1 (en) 2017-11-22 2020-09-30 Modernatx, Inc. Polynucleotides encoding propionyl-coa carboxylase alpha and beta subunits for the treatment of propionic acidemia
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US11167043B2 (en) 2017-12-20 2021-11-09 Translate Bio, Inc. Composition and methods for treatment of ornithine transcarbamylase deficiency
MA51523A (en) 2018-01-05 2020-11-11 Modernatx Inc POLYNUCLEOTIDES CODING ANTI-BODY ANTI-CHIKUNGUNYA VIRUS
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
CN108743918A (en) * 2018-06-21 2018-11-06 贵州医科大学 The new application and preparation method thereof of people's beta-alexin 2
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
US20220184185A1 (en) 2018-07-25 2022-06-16 Modernatx, Inc. Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders
KR20210060480A (en) 2018-08-24 2021-05-26 트랜슬레이트 바이오 인코포레이티드 Method for purifying messenger RNA
WO2020047201A1 (en) 2018-09-02 2020-03-05 Modernatx, Inc. Polynucleotides encoding very long-chain acyl-coa dehydrogenase for the treatment of very long-chain acyl-coa dehydrogenase deficiency
MA53608A (en) 2018-09-13 2021-07-21 Modernatx Inc POLYNUCLEOTIDES ENCODED FOR THE E1-ALPHA, E1-BETA AND E2 SUBUNITS OF THE BRANCHED-CHAIN ALPHA-KETOACID DEHYDROGENASE COMPLEX FOR THE TREATMENT OF LEUCINOSIS
MX2021003015A (en) 2018-09-13 2021-07-15 Modernatx Inc Polynucleotides encoding glucose-6-phosphatase for the treatment of glycogen storage disease.
EP3850102A1 (en) 2018-09-14 2021-07-21 ModernaTX, Inc. Polynucleotides encoding uridine diphosphate glycosyltransferase 1 family, polypeptide a1 for the treatment of crigler-najjar syndrome
US20220152225A1 (en) 2018-09-27 2022-05-19 Modernatx, Inc. Polynucleotides encoding arginase 1 for the treatment of arginase deficiency
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
CA3120647A1 (en) 2018-11-21 2020-05-28 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mrna encoding cftr
WO2020118427A1 (en) * 2018-12-12 2020-06-18 Provincial Health Services Authority Antimicrobial peptides
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
CN114127285B (en) 2019-03-19 2024-09-10 布罗德研究所股份有限公司 Methods and compositions for editing nucleotide sequences
EP3956349A1 (en) 2019-04-17 2022-02-23 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US20220226438A1 (en) 2019-05-08 2022-07-21 Astrazeneca Ab Compositions for skin and wounds and methods of use thereof
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
CA3158422A1 (en) * 2019-11-15 2021-05-20 Flavio Curnis Chromogranin a-derived peptides and uses thereof
WO2021151001A1 (en) 2020-01-22 2021-07-29 Outpace Bio, Inc. Chimeric polypeptides
EP4103228A1 (en) 2020-02-13 2022-12-21 Institut Pasteur Nucleic acid vaccine against the sars-cov-2 coronavirus
DE112021002672T5 (en) 2020-05-08 2023-04-13 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EDIT BOTH STRANDS SIMULTANEOUSLY OF A DOUBLE STRANDED NUCLEOTIDE TARGET SEQUENCE
ES2880043A1 (en) * 2020-05-20 2021-11-23 Fundacion Para El Fomento De La Investig Sanitaria Y Biomedica De La Comunitat Valenciana Fisabio PEPTIDE FOR USE IN THE PREVENTION AND/OR TREATMENT OF A DISEASE CAUSED BY A VIRUS THAT AFFECTS THE RESPIRATORY TRACT (Machine-translation by Google Translate, not legally binding)
JP2023527875A (en) 2020-06-01 2023-06-30 モダーナティエックス・インコーポレイテッド Phenylalanine hydroxylase variants and uses thereof
IL302625A (en) 2020-11-13 2023-07-01 Modernatx Inc Cystic fibrosis-encoding polynucleotides transmembrane conductance regulator for the treatment of cystic fibrosis
WO2022204369A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase for the treatment of methylmalonic acidemia
US20240207444A1 (en) 2021-03-24 2024-06-27 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding phenylalanine hydroxylase and uses thereof
JP2024512026A (en) 2021-03-24 2024-03-18 モデルナティエックス インコーポレイテッド Lipid nanoparticles and polynucleotide encoding ornithine transcarbamylase for the treatment of ornithine transcarbamylase deficiency
WO2022204380A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding propionyl-coa carboxylase alpha and beta subunits and uses thereof
WO2022204371A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding glucose-6-phosphatase and uses thereof
WO2022228827A1 (en) 2021-04-26 2022-11-03 Institut Pasteur Human neutralizing monoclonal antibodies against sars-cov-2 and uses thereof
WO2022251644A1 (en) 2021-05-28 2022-12-01 Lyell Immunopharma, Inc. Nr4a3-deficient immune cells and uses thereof
EP4347826A1 (en) 2021-06-02 2024-04-10 Lyell Immunopharma, Inc. Nr4a3-deficient immune cells and uses thereof
WO2022271776A1 (en) 2021-06-22 2022-12-29 Modernatx, Inc. Polynucleotides encoding uridine diphosphate glycosyltransferase 1 family, polypeptide a1 for the treatment of crigler-najjar syndrome
CN113502337B (en) * 2021-07-13 2022-03-22 华中农业大学 A SNP Molecular Marker for Improving Disease Resistance of Crayfish and Its Application
US20250017867A1 (en) 2021-10-01 2025-01-16 Modernatx, Inc. Polynucleotides encoding relaxin for the treatment of fibrosis and/or cardiovascular disease
CN114045258B (en) * 2021-10-21 2024-04-16 辽宁盛京干细胞科技有限公司 Serum-free culture medium for mesenchymal stem cell culture and application
CN113912690B (en) * 2021-10-29 2023-06-23 中国科学院昆明动物研究所 A tick defensin OPTX-1 and its application
IL312452A (en) 2021-11-01 2024-06-01 Tome Biosciences Inc Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo
JP2024540170A (en) 2021-11-01 2024-10-31 モデルナティエックス インコーポレイテッド Polynucleotides encoding integrin beta-6 and methods of use thereof
WO2023122764A1 (en) 2021-12-22 2023-06-29 Tome Biosciences, Inc. Co-delivery of a gene editor construct and a donor template
TW202345864A (en) 2022-02-18 2023-12-01 美商現代公司 Mrnas encoding checkpoint cancer vaccines and uses thereof
WO2023183909A2 (en) 2022-03-25 2023-09-28 Modernatx, Inc. Polynucleotides encoding fanconi anemia, complementation group proteins for the treatment of fanconi anemia
WO2023196399A1 (en) 2022-04-06 2023-10-12 Modernatx, Inc. Lipid nanoparticles and polynucleotides encoding argininosuccinate lyase for the treatment of argininosuccinic aciduria
WO2023205744A1 (en) 2022-04-20 2023-10-26 Tome Biosciences, Inc. Programmable gene insertion compositions
CN119053615A (en) * 2022-04-28 2024-11-29 布尔曼实验室股份公司 Calprotectin binding peptides
WO2023215831A1 (en) 2022-05-04 2023-11-09 Tome Biosciences, Inc. Guide rna compositions for programmable gene insertion
JP2025516823A (en) 2022-05-19 2025-05-30 ライエル・イミュノファーマ・インコーポレイテッド Polynucleotides targeting NR4A3 and uses thereof
WO2023225670A2 (en) 2022-05-20 2023-11-23 Tome Biosciences, Inc. Ex vivo programmable gene insertion
WO2024020587A2 (en) 2022-07-22 2024-01-25 Tome Biosciences, Inc. Pleiopluripotent stem cell programmable gene insertion
WO2024027860A1 (en) * 2022-08-05 2024-02-08 Česká zemědělská univerzita v Praze A bacteriocin composition for coding linocin-m18-like protein with antimicrobial activity and its usage
CN116102640A (en) * 2022-10-13 2023-05-12 浙江双糖生物科技有限公司 Recombinant lactoferrin derived peptides and their use in enhancing immunity
WO2024130158A1 (en) 2022-12-16 2024-06-20 Modernatx, Inc. Lipid nanoparticles and polynucleotides encoding extended serum half-life interleukin-22 for the treatment of metabolic disease
WO2024138194A1 (en) 2022-12-22 2024-06-27 Tome Biosciences, Inc. Platforms, compositions, and methods for in vivo programmable gene insertion
WO2024182301A2 (en) 2023-02-27 2024-09-06 Modernatx, Inc. Lipid nanoparticles and polynucleotides encoding galactose-1-phosphate uridylyltransferase (galt) for the treatment of galactosemia
WO2024206329A1 (en) 2023-03-27 2024-10-03 Modernatx, Inc. Nucleic acid molecules encoding bi-specific secreted engagers and uses thereof
WO2024205020A1 (en) * 2023-03-28 2024-10-03 서울대학교산학협력단 Anti-trichomonas uses of alloferon
EP4442276A1 (en) 2023-04-07 2024-10-09 Institut Pasteur Combined antibodies against sarbecoviruses and uses thereof
AU2024265267A1 (en) 2023-05-03 2025-11-20 Modernatx, Inc. Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis
WO2024234006A1 (en) 2023-05-11 2024-11-14 Tome Biosciences, Inc. Systems, compositions, and methods for targeting liver sinusodial endothelial cells (lsecs)
WO2024236192A1 (en) 2023-05-17 2024-11-21 Institut Pasteur Heterodimer of poxvirus a16 and g9 proteins as an immunogen
CN116693650A (en) * 2023-07-04 2023-09-05 云南师范大学 Antibacterial peptide SpCru5 from flatfish intestinal tract, and preparation method and application thereof
WO2025050069A1 (en) 2023-09-01 2025-03-06 Tome Biosciences, Inc. Programmable gene insertion using engineered integration enzymes
CN116874614B (en) * 2023-09-06 2023-11-17 南京华盖制药有限公司 Antibacterial polypeptide APH171 with high activity and low cracking effect, and preparation method and application thereof
EP4626453A1 (en) * 2023-10-30 2025-10-08 Ramakrishna Reddy Isanaka Peptide with anti-inflammatory and anti-microbial activity
WO2025153707A1 (en) 2024-01-19 2025-07-24 Spikimm Broadly sars-cov-2 neutralizing monoclonal antibodies and uses thereof
WO2025194138A1 (en) 2024-03-14 2025-09-18 Tessera Therapeutics, Inc. St1cas9 compositions and methods for modulating a genome
EP4677108A1 (en) 2024-04-22 2026-01-14 Basecamp Research Ltd Method and compositions for detecting off-target editing
WO2025224182A2 (en) 2024-04-23 2025-10-30 Basecamp Research Ltd Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo
WO2025255199A1 (en) 2024-06-05 2025-12-11 Modernatx, Inc. Argininosuccinate synthase 1 and argininosuccinate lyase polypeptides and polynucleotides and uses thereof
CN119954903A (en) * 2025-01-14 2025-05-09 中国农业科学院兰州兽医研究所(中国动物卫生与流行病学中心兰州分中心) A group of polypeptides with anti-polysaccharide blue ear disease virus activity and their application

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011005799A2 (en) * 2009-07-06 2011-01-13 Novartis Ag Self replicating rna molecules and uses thereof

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8202835B2 (en) * 2005-06-17 2012-06-19 Yitzchak Hillman Disease treatment via antimicrobial peptides or their inhibitors
WO2010037408A1 (en) * 2008-09-30 2010-04-08 Curevac Gmbh Composition comprising a complexed (m)rna and a naked mrna for providing or enhancing an immunostimulatory response in a mammal and uses thereof
WO2010141135A2 (en) * 2009-03-05 2010-12-09 Trustees Of Boston University Bacteriophages expressing antimicrobial peptides and uses thereof

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011005799A2 (en) * 2009-07-06 2011-01-13 Novartis Ag Self replicating rna molecules and uses thereof

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
K. O. KISICH ET AL: "Antimycobacterial Agent Based on mRNA Encoding Human -Defensin 2 Enables Primary Macrophages To Restrict Growth of Mycobacterium tuberculosis", INFECTION AND IMMUNITY, vol. 69, no. 4, 1 April 2001 (2001-04-01), US, pages 2692 - 2699, XP055282571, ISSN: 0019-9567, DOI: 10.1128/IAI.69.4.2692-2699.2001 *
KARIKO KATALIN ET AL: "Impacts of nucleoside modification on RNA-mediated activation of toll-like receptors", 1 January 2008, NUCLEIC ACIDS IN INNATE IMMUNITY, CRC PRESS-TAYLOR & FRANCIS GROUP, 6000 BROKEN SOUND PARKWAY NW, STE 300, BOCA RATON, FL 33487-2742 USA, PAGE(S) 171 - 188, ISBN: 978-1-4200-6825-2, XP008166255 *
KARIKÓ KATALIN ET AL: "Incorporation of pseudouridine into mRNA yields superior nonimmunogenic vector with increased translational capacity and biological stability", MOLECULAR THERAPY, NATURE PUBLISHING GROUP, GB, vol. 16, no. 11, 1 November 2008 (2008-11-01), pages 1833 - 1840, XP002598556, ISSN: 1525-0024, [retrieved on 20080916], DOI: 10.1038/MT.2008.200 *
KATALIN KARIKÓ ET AL: "Generating the optimal mRNA for therapy: HPLC purification eliminates immune activation and improves translation of nucleoside-modified, protein-encoding mRNA", NUCLEIC ACIDS RESEARCH, OXFORD UNIVERSITY PRESS, GB, vol. 39, no. 21, 2 September 2011 (2011-09-02), pages e142 - 1, XP002696190, ISSN: 1362-4962, [retrieved on 20110902], DOI: 10.1093/NAR/GKR695 *
See also references of WO2013039857A1 *

Also Published As

Publication number Publication date
WO2013039857A1 (en) 2013-03-21
EP2755986A1 (en) 2014-07-23

Similar Documents

Publication Publication Date Title
EP2755986A4 (en) MODIFIED NUCLEIC ACIDS AND METHODS OF USE
EP2755693A4 (en) MODIFIED NUCLEIC ACIDS AND METHODS OF USE
EP2600901A4 (en) MODIFIED NUCLEIC ACIDS AND METHODS OF USE
EP2922989A4 (en) NORMALIZED SEQUENCING METHODS OF NUCLEIC ACIDS AND USES THEREOF
EP2788370A4 (en) MODIFIED MINI-HEPCIDINE PEPTIDES AND METHODS OF USE
EP2670866A4 (en) BIOMARKERS AND METHODS OF USE
EP2766393A4 (en) ANTI-HTRA1 ANTIBODIES AND METHODS OF USE
EP2710136A4 (en) MANIPULATED NUCLEIC ACIDS AND METHODS OF USE FOR NON-HUMAN VERTEBRATES
EP2702078A4 (en) ANTI-CD40 ANTIBODIES AND METHODS OF USE
EP2748199A4 (en) ANTI-OX40 ANTIBODIES AND METHODS OF USE
EP2831098A4 (en) CHARGED NUTRIENT PROTEINS AND METHODS
EP2920319A4 (en) SITE SPECIFIC ENZYMES AND METHODS OF USE THEREOF
EP2771503A4 (en) DEEP DRAWING COMPOSITES AND METHODS OF USE
EP2834421A4 (en) STABILIZING AGENTS AND METHODS OF USE
EP2870138A4 (en) N-SUBSTITUTED BENZAMIDES AND METHODS OF USE
EP2710117A4 (en) RECOMBINANT MICROORGANISMS AND METHODS OF USE
EP2912178A4 (en) SUPER-ACTIVATORS AND METHODS OF USE THEREOF
EP2819822A4 (en) POLYMERIZABLE CATIONIC COMPOSITIONS AND METHODS OF USE
EP2673381A4 (en) BIOSONS AND METHODS OF USE THEREOF
EP2831100A4 (en) NUTRIENT PROTEINS AND METHODS
EP2726477A4 (en) SMALL MOLECULE CD38 INHIBITORS AND METHODS OF USE
EP2552959A4 (en) ANTI-MUC16 ANTIBODIES AND METHODS OF USE
EP2663565A4 (en) HETEROARYL COMPOUNDS AND METHODS OF USE
EP2769012A4 (en) TRESSING MECHANISM AND METHODS OF USE
EP2882750A4 (en) DOT1L PROTEIN METHYLTRANSFERASE INHIBITORS AND METHODS OF USE

Legal Events

Date Code Title Description
PUAI Public reference made under article 153(3) epc to a published international application that has entered the european phase

Free format text: ORIGINAL CODE: 0009012

17P Request for examination filed

Effective date: 20140328

AK Designated contracting states

Kind code of ref document: A1

Designated state(s): AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR

DAX Request for extension of the european patent (deleted)
RA4 Supplementary search report drawn up and despatched (corrected)

Effective date: 20150416

RIC1 Information provided on ipc code assigned before grant

Ipc: C07K 14/47 20060101ALI20150410BHEP

Ipc: C07H 21/02 20060101AFI20150410BHEP

Ipc: A61K 48/00 20060101ALI20150410BHEP

RAP1 Party data changed (applicant data changed or rights of an application transferred)

Owner name: MODERNA THERAPEUTICS, INC.

RIN1 Information on inventor provided before grant (corrected)

Inventor name: BANCEL, STEPHANE

Inventor name: SCHRUM, JASON, P.

Inventor name: ARISTARKHOV, ALEXANDER

17Q First examination report despatched

Effective date: 20170329

STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: THE APPLICATION IS DEEMED TO BE WITHDRAWN

18D Application deemed to be withdrawn

Effective date: 20171010