EP2755986A4 - MODIFIED NUCLEIC ACIDS AND METHODS OF USE - Google Patents
MODIFIED NUCLEIC ACIDS AND METHODS OF USEInfo
- 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- Withdrawn
Links
- 150000007523 nucleic acids Chemical class 0.000 title 1
- 102000039446 nucleic acids Human genes 0.000 title 1
- 108020004707 nucleic acids Proteins 0.000 title 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal 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
- A61K48/0066—Manipulation 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0091—Purification or manufacturing processes for gene therapy compositions
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/02—Compounds 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4723—Cationic 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)
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)
| 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)
| 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)
| 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 |
-
2012
- 2012-09-11 EP EP12831509.0A patent/EP2755986A4/en not_active Withdrawn
- 2012-09-11 WO PCT/US2012/054561 patent/WO2013039857A1/en not_active Ceased
Patent Citations (1)
| 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)
| 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 |