EP2755986A4 - Engineered nucleic acids and methods of use thereof - Google Patents

Engineered nucleic acids and methods of use thereof

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
engineered nucleic
engineered
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Withdrawn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Withdrawn legal-status Critical Current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 Engineered nucleic acids and methods of use thereof 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 Engineered nucleic acids and methods of use thereof

Country Status (2)

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

Families Citing this family (159)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT3338765T (en) 2009-12-01 2019-03-18 Translate Bio Inc Steroid derivative for the delivery of mrna 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
HRP20220796T1 (en) 2010-10-01 2022-10-14 ModernaTX, Inc. Ribonucleic acids containing n1-methyl-pseudouracils and uses thereof
US8853377B2 (en) 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
CA2831613A1 (en) 2011-03-31 2012-10-04 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
AU2012267531B2 (en) 2011-06-08 2017-06-22 Translate Bio, Inc. Lipid nanoparticle compositions and methods for mRNA delivery
CA2853829C (en) 2011-07-22 2023-09-26 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
EP3492109B1 (en) 2011-10-03 2020-03-04 ModernaTX, Inc. Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
RS63244B1 (en) * 2011-12-16 2022-06-30 Modernatx Inc Modified mrna compositions
US10501513B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
US9303079B2 (en) 2012-04-02 2016-04-05 Moderna Therapeutics, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
WO2013151664A1 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins
CA2868391A1 (en) * 2012-04-02 2013-10-10 Stephane Bancel Polynucleotides comprising n1-methyl-pseudouridine and methods for preparing the same
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
EP3536787A1 (en) 2012-06-08 2019-09-11 Translate Bio, Inc. Nuclease resistant polynucleotides and uses thereof
EA201492055A1 (en) 2012-06-08 2015-11-30 Шир Хьюман Дженетик Терапис, Инк. INHALATIVE DELIVERY OF mRNA IN TIGHTNESS CELL TARGETS
WO2014028429A2 (en) 2012-08-14 2014-02-20 Moderna Therapeutics, Inc. Enzymes and polymerases for the synthesis of rna
PL2922554T3 (en) 2012-11-26 2022-06-20 Modernatx, Inc. Terminally modified 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
JP6586075B2 (en) 2013-03-14 2019-10-02 トランスレイト バイオ, インコーポレイテッド Method for purifying messenger RNA
AU2014239184B2 (en) 2013-03-14 2018-11-08 Translate Bio, Inc. Methods and compositions for delivering mRNA coded antibodies
WO2014152211A1 (en) 2013-03-14 2014-09-25 Moderna Therapeutics, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
HUE042640T2 (en) 2013-03-14 2019-07-29 Translate Bio Inc Cftr mrna compositions and related methods and uses
WO2014152030A1 (en) 2013-03-15 2014-09-25 Moderna Therapeutics, Inc. Removal of dna fragments in mrna production process
EP2972360B1 (en) 2013-03-15 2018-03-07 Translate Bio, Inc. Synergistic enhancement of the delivery of nucleic acids via blended formulations
US10590161B2 (en) 2013-03-15 2020-03-17 Modernatx, Inc. Ion exchange purification of mRNA
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
US11377470B2 (en) 2013-03-15 2022-07-05 Modernatx, Inc. Ribonucleic acid purification
WO2014152027A1 (en) 2013-03-15 2014-09-25 Moderna Therapeutics, Inc. Manufacturing methods for production of rna transcripts
EP3019619B1 (en) 2013-07-11 2021-08-25 ModernaTX, Inc. Compositions comprising synthetic polynucleotides encoding crispr related proteins and synthetic sgrnas and methods of use
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
EP3052106A4 (en) 2013-09-30 2017-07-19 ModernaTX, Inc. Polynucleotides encoding immune modulating polypeptides
WO2015051169A2 (en) 2013-10-02 2015-04-09 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
SG11201602503TA (en) 2013-10-03 2016-04-28 Moderna Therapeutics Inc Polynucleotides encoding low density lipoprotein receptor
CN112656954A (en) 2013-10-22 2021-04-16 夏尔人类遗传性治疗公司 Lipid formulations for delivery of messenger RNA
ES2707966T3 (en) 2013-10-22 2019-04-08 Translate Bio Inc MRNA therapy for the deficiency in argininosuccinate synthesis
EA201690588A1 (en) 2013-10-22 2016-09-30 Шир Хьюман Дженетик Терапис, Инк. DELIVERY OF MRNA IN THE CNS AND ITS APPLICATION
EP3060258A1 (en) 2013-10-22 2016-08-31 Shire Human Genetic Therapies, Inc. Mrna therapy for phenylketonuria
US10981953B2 (en) * 2013-12-26 2021-04-20 Toagosei Co, Ltd. Method for promoting expression of calreticulin, and synthetic peptide for use in method for promoting expression of calreticulin
WO2015112980A2 (en) 2014-01-24 2015-07-30 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
KR20220158867A (en) 2014-04-25 2022-12-01 샤이어 휴먼 지네틱 테라피즈 인크. Methods for purification of messenger rna
AU2015266764B2 (en) 2014-05-30 2019-11-07 Translate Bio, Inc. Biodegradable lipids for delivery of nucleic acids
CN111588695A (en) 2014-06-24 2020-08-28 川斯勒佰尔公司 Stereochemically enriched compositions for delivery of nucleic acids
AU2015283954B2 (en) 2014-07-02 2020-11-12 Translate Bio, Inc. Encapsulation of messenger RNA
JP2017522028A (en) 2014-07-16 2017-08-10 モデルナティエックス インコーポレイテッドModernaTX,Inc. Circular polynucleotide
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
KR20170065583A (en) 2014-10-01 2017-06-13 플랜트 헬스 케어, 인코포레이티드 Hypersensitive response elicitor peptides and use thereof
MX2017004278A (en) 2014-10-01 2018-03-28 Plant Health Care Inc Elicitor peptides having disrupted hypersensitive response box and use thereof.
WO2016056665A1 (en) * 2014-10-08 2016-04-14 学校法人慶應義塾 White blood cell extracellular trap formation inhibitor
TWI640540B (en) 2014-10-27 2018-11-11 中央研究院 Plant defense signaling peptides and applications thereof
EP3218508A4 (en) 2014-11-10 2018-04-18 Modernatx, Inc. Multiparametric nucleic acid optimization
US9943595B2 (en) 2014-12-05 2018-04-17 Translate Bio, Inc. Messenger RNA therapy for treatment of articular disease
EP3256591A4 (en) 2015-02-13 2018-08-08 Translate Bio Ma, Inc. Hybrid oligonucleotides and uses thereof
US10172924B2 (en) 2015-03-19 2019-01-08 Translate Bio, Inc. MRNA therapy for pompe disease
RU2739392C2 (en) * 2015-06-15 2020-12-23 Ордженезис Инк. Pharmaceutical preparations for treating viral infections of eye
MX2017016251A (en) * 2015-07-02 2018-04-20 Dana Farber Cancer Inst 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
CA3001003A1 (en) 2015-10-05 2017-04-13 Modernatx, Inc. Methods for therapeutic administration of messenger ribonucleic acid drugs
AU2016338559B2 (en) 2015-10-14 2022-11-24 Translate Bio, Inc. Modification of RNA-related enzymes for enhanced production
IL294014B2 (en) 2015-10-23 2024-07-01 Harvard College Nucleobase editors and uses thereof
WO2017079505A1 (en) * 2015-11-04 2017-05-11 Research Institute At Nationwide Children's Hospital Rnase7 for treatment of bacterial infection
RS63135B1 (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
CN109069578A (en) 2016-02-29 2018-12-21 达纳-法伯癌症研究所股份有限公司 For treating the intracellular targeted antimicrobial peptides of the stitching of infection
AU2017248189B2 (en) 2016-04-08 2021-04-29 Translate Bio, Inc. Multimeric coding nucleic acid and uses thereof
MA45052A (en) 2016-05-18 2019-03-27 Modernatx Inc POLYNUCLEOTIDES CODING FOR JAGGED1 FOR THE TREATMENT OF ALAGILLUS SYNDROME
KR102482867B1 (en) 2016-05-18 2023-01-02 모더나티엑스, 인크. Combinations of mRNAs encoding immune modulatory polypeptides and uses thereof
MA45036A (en) 2016-05-18 2019-03-27 Modernatx Inc POLYNUCLEOTIDES CODING CITRINE FOR THE TREATMENT OF CITRULLINEMIA TYPE 2
PT3458083T (en) 2016-05-18 2023-03-06 Modernatx Inc Polynucleotides encoding interleukin-12 (il12) and uses thereof
EP3896164A1 (en) 2016-05-18 2021-10-20 ModernaTX, Inc. Polynucleotides encoding alpha-galactosidase a for the treatment of fabry disease
EP3458104A1 (en) 2016-05-18 2019-03-27 Modernatx, Inc. Polynucleotides encoding porphobilinogen deaminase for the treatment of acute intermittent porphyria
ES2973443T3 (en) 2016-05-18 2024-06-20 Modernatx Inc Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1
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
EP3469074B1 (en) 2016-06-13 2020-12-09 Translate Bio, Inc. Messenger rna therapy for the treatment of ornithine transcarbamylase deficiency
EP3471751A1 (en) * 2016-06-15 2019-04-24 Rajiv Gandhi Centre for Biotechnology, an Autonomous Institute under the Department of Bio-Technology, Government of India Therapeutic compositions from the brevinin-1 family of peptides and uses thereof
AU2017293931A1 (en) 2016-07-07 2019-01-17 Rubius Therapeutics, Inc. Compositions and methods related to therapeutic cell systems expressing exogenous RNA
IL308426A (en) 2016-08-03 2024-01-01 Harvard College Adenosine nucleobase editors and uses thereof
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
SG11201903089RA (en) 2016-10-14 2019-05-30 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 antimicrobial and anti-cancer therapy
CA3046927A1 (en) * 2016-12-13 2018-06-21 Emory University Polypeptides for managing viral infections
WO2018119359A1 (en) 2016-12-23 2018-06-28 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
EP3585417B1 (en) 2017-02-27 2023-02-22 Translate Bio, Inc. Method of making a codon-optimized cftr mrna
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
EP3592777A1 (en) 2017-03-10 2020-01-15 President and Fellows of Harvard College Cytosine to guanine base editor
JP7191388B2 (en) 2017-03-23 2022-12-19 プレジデント アンド フェローズ オブ ハーバード カレッジ Nucleobase editors comprising nucleic acid programmable DNA binding proteins
US11560566B2 (en) 2017-05-12 2023-01-24 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
JP7285220B2 (en) 2017-05-18 2023-06-01 モデルナティエックス インコーポレイテッド Lipid nanoparticles comprising linked interleukin-12 (IL12) polypeptide-encoding polynucleotides
US20200131498A1 (en) 2017-06-14 2020-04-30 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase
US20200268666A1 (en) 2017-06-14 2020-08-27 Modernatx, Inc. Polynucleotides encoding coagulation factor viii
US11325955B2 (en) 2017-07-19 2022-05-10 Dana-Farber Cancer Institute, Inc. Stabilized anti-microbial peptides for the treatment of antibiotic-resistant bacterial infections
CN111801345A (en) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 Methods and compositions using an evolved base editor for 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
CN111757937A (en) 2017-10-16 2020-10-09 布罗德研究所股份有限公司 Use of adenosine base editor
MA50803A (en) 2017-11-22 2020-09-30 Modernatx Inc POLYNUCLEOTIDES CODING ORNITHINE TRANSCARBAMYLASE FOR THE TREATMENT OF UREA CYCLE DISORDERS
JP7423521B2 (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 phenylalanine hydroxylase for the treatment of phenylketonuria
JP7424976B2 (en) 2017-11-22 2024-01-30 モダーナティエックス・インコーポレイテッド Polynucleotide encoding propionyl-CoA carboxylase alpha and beta subunits for the treatment of propionic acidemia
AU2018392716A1 (en) 2017-12-20 2020-06-18 Translate Bio, Inc. Improved 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
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
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
CA3108544A1 (en) 2018-08-24 2020-02-27 Translate Bio, Inc. Methods for purification of 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
EP3849594A2 (en) 2018-09-13 2021-07-21 Modernatx, Inc. Polynucleotides encoding branched-chain alpha-ketoacid dehydrogenase complex e1-alpha, e1-beta, and e2 subunits for the treatment of maple syrup urine disease
EP3849595A2 (en) 2018-09-13 2021-07-21 Modernatx, Inc. Polynucleotides encoding glucose-6-phosphatase for the treatment of glycogen storage disease
MA53615A (en) 2018-09-14 2021-07-21 Modernatx Inc POLYNUCLEOTIDES ENCODED FOR POLYPEPTIDE A1, OF THE URIDINE DIPHOSPHATE GLYCOSYLTRANSFERASE 1 FAMILY, FOR THE TREATMENT OF CRIGLER-NAJJAR SYNDROME
WO2020069169A1 (en) 2018-09-27 2020-04-02 Modernatx, Inc. Polynucleotides encoding arginase 1 for the treatment of arginase deficiency
EP3897128A4 (en) * 2018-12-12 2022-12-28 Provincial Health Services Authority Antimicrobial peptides
WO2020191243A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
US20220226438A1 (en) 2019-05-08 2022-07-21 Astrazeneca Ab Compositions for skin and wounds and methods of use thereof
WO2021094608A1 (en) * 2019-11-15 2021-05-20 Ospedale San Raffaele S.R.L. Chromogranin a-derived peptides and uses thereof
WO2021151001A1 (en) 2020-01-22 2021-07-29 Outpace Bio, Inc. Chimeric polypeptides
WO2021160346A1 (en) 2020-02-13 2021-08-19 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
EP4158005A1 (en) 2020-06-01 2023-04-05 ModernaTX, Inc. Phenylalanine hydroxylase variants and uses thereof
US20230406895A1 (en) 2020-11-13 2023-12-21 Modernatx, Inc. Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis
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
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
US20240207374A1 (en) 2021-03-24 2024-06-27 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding glucose-6-phosphatase and uses thereof
EP4330278A1 (en) 2021-04-26 2024-03-06 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 SNP molecular marker for improving disease resistance of procambarus clarkii and application
EP4408871A1 (en) 2021-10-01 2024-08-07 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 Tick defensin OPTX-1 and application thereof
EP4426340A1 (en) 2021-11-01 2024-09-11 ModernaTX, Inc. Polynucleotides encoding integrin beta-6 and methods of use thereof
CA3237300A1 (en) 2021-11-01 2023-05-04 Tome Biosciences, Inc. Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo
IL313765A (en) 2021-12-22 2024-08-01 Tome Biosciences Inc Co-delivery of a gene editor construct and a donor template
WO2023159197A1 (en) 2022-02-18 2023-08-24 Modernatx, Inc. 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
WO2023209055A1 (en) * 2022-04-28 2023-11-02 Bühlmann Laboratories Ag Calprotectin binding peptides
WO2023215831A1 (en) 2022-05-04 2023-11-09 Tome Biosciences, Inc. Guide rna compositions for programmable gene insertion
WO2023225665A1 (en) 2022-05-19 2023-11-23 Lyell Immunopharma, Inc. 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
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

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 (4)

* Cited by examiner, † Cited by third party
Title
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
EP2755986A1 (en) 2014-07-23
WO2013039857A1 (en) 2013-03-21

Similar Documents

Publication Publication Date Title
EP2755986A4 (en) Engineered nucleic acids and methods of use thereof
EP2755693A4 (en) Engineered nucleic acids and methods of use thereof
ZA201403666B (en) Engineered nucleic acids and methods of use thereof
EP2600901A4 (en) Engineered nucleic acids and methods of use thereof
HK1255608A1 (en) Anti-htra1 antibodies and methods of use
EP2710136A4 (en) Engineered nucleic acids and methods of use thereof for non-human vertebrates
HK1199892A1 (en) Modified mini-hepcidin peptides and methods of using thereof
ZA201405858B (en) Recombinant microorganisms and methods of use thereof
EP2689253A4 (en) Microfluidic devices and methods of manufacture and use
ZA201403760B (en) Anti-cd98 antibodies and methods of use thereof
ZA201401653B (en) Pyrazol-4yl-heterocyclyl-carboxamide compounds and methods of use
HK1201463A1 (en) Cd40l-specific tn3-derived scaffolds and methods of use thereof cd40l- tn3-
HK1201843A1 (en) Anti-polyubiquitin antibodies and methods of use
EP2766501A4 (en) Nucleic acid amplification and use thereof
EP2709999A4 (en) Quinazoline-7-ether compounds and methods of use
EP2753359A4 (en) Serpinf2-binding molecules and methods of use
EP2702999A4 (en) Brain-targeting functional nucleic acid and use thereof
GB201016452D0 (en) Rapid extrection of nucleic acids

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