MA55899A - Polynucléotides codant pour la méthylmalonyl-coa mutase pour le traitement de l'acidémie méthylmalonique - Google Patents
Polynucléotides codant pour la méthylmalonyl-coa mutase pour le traitement de l'acidémie méthylmaloniqueInfo
- Publication number
- MA55899A MA55899A MA055899A MA55899A MA55899A MA 55899 A MA55899 A MA 55899A MA 055899 A MA055899 A MA 055899A MA 55899 A MA55899 A MA 55899A MA 55899 A MA55899 A MA 55899A
- Authority
- MA
- Morocco
- Prior art keywords
- methylmalonyl
- treatment
- coa mutase
- methylmalonic acidemia
- polynucleotides encoded
- Prior art date
Links
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/10—Dispersions; Emulsions
- A61K9/127—Liposomes
- A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
- A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0041—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/51—Nanocapsules; Nanoparticles
- A61K9/5107—Excipients; Inactive ingredients
- A61K9/5123—Organic compounds, e.g. fats, sugars
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/90—Isomerases (5.)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y504/00—Intramolecular transferases (5.4)
- C12Y504/99—Intramolecular transferases (5.4) transferring other groups (5.4.99)
- C12Y504/99002—Methylmalonyl-CoA mutase (5.4.99.2)
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201962845250P | 2019-05-08 | 2019-05-08 |
Publications (1)
Publication Number | Publication Date |
---|---|
MA55899A true MA55899A (fr) | 2022-03-16 |
Family
ID=71069931
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MA055899A MA55899A (fr) | 2019-05-08 | 2020-05-08 | Polynucléotides codant pour la méthylmalonyl-coa mutase pour le traitement de l'acidémie méthylmalonique |
Country Status (4)
Country | Link |
---|---|
US (1) | US20220370354A1 (fr) |
EP (1) | EP3965806A1 (fr) |
MA (1) | MA55899A (fr) |
WO (1) | WO2020227615A1 (fr) |
Families Citing this family (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2022246020A1 (fr) * | 2021-05-19 | 2022-11-24 | Modernatx, Inc. | Polynucléotides codant pour la méthylmalonyl-coa mutase pour le traitement de l'acidémie méthylmalonique |
Family Cites Families (21)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20050222064A1 (en) | 2002-02-20 | 2005-10-06 | Sirna Therapeutics, Inc. | Polycationic compositions for cellular delivery of polynucleotides |
EP1742663A2 (fr) | 2004-04-15 | 2007-01-17 | Chiasma, Ltd. | Compositions capables de faciliter la penetration a travers une barriere biologique |
US7404969B2 (en) | 2005-02-14 | 2008-07-29 | Sirna Therapeutics, Inc. | Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules |
WO2006110776A2 (fr) | 2005-04-12 | 2006-10-19 | Nektar Therapeutics Al, Corporation | Compositions a base de polymere et conjugues d'agents antimicrobiens |
CN101420984B (zh) | 2006-02-21 | 2013-01-02 | 尼克塔治疗公司 | 嵌段可降解聚合物及由其制备的轭合物 |
PL215513B1 (pl) | 2008-06-06 | 2013-12-31 | Univ Warszawski | Nowe boranofosforanowe analogi dinukleotydów, ich zastosowanie, czasteczka RNA, sposób otrzymywania RNA oraz sposób otrzymywania peptydów lub bialka |
TR201811076T4 (tr) | 2009-06-10 | 2018-08-27 | Arbutus Biopharma Corp | Geliştirilmiş lipit formulasyonu. |
WO2011163483A2 (fr) | 2010-06-25 | 2011-12-29 | Massachusetts Institute Of Technology | Polymères pour des biomatériaux et des agents thérapeutiques |
US10307372B2 (en) | 2010-09-10 | 2019-06-04 | The Johns Hopkins University | Rapid diffusion of large polymeric nanoparticles in the mammalian brain |
WO2012099755A1 (fr) | 2011-01-11 | 2012-07-26 | Alnylam Pharmaceuticals, Inc. | Lipides pégylés et leur utilisation pour une administration de médicament |
WO2012109121A1 (fr) | 2011-02-07 | 2012-08-16 | Purdue Research Foundation | Nanoparticules glucidiques pour une efficacité prolongée d'un peptide antimicrobien |
ES2795110T3 (es) | 2011-06-08 | 2020-11-20 | Translate Bio Inc | Lípidos escindibles |
CA2845845A1 (fr) | 2011-08-31 | 2013-03-07 | Mallinckrodt Llc | Modification de nanoparticules de peg avec des h-phosphonates |
DK3988537T1 (da) | 2011-12-07 | 2022-05-23 | Alnylam Pharmaceuticals Inc | Bionedbrydelige lipider til afgivelse af aktive midler |
WO2013105101A1 (fr) | 2012-01-13 | 2013-07-18 | Department Of Biotechnology | Nanoparticules lipidiques solides encapsulant un médicament hydrophile/amphiphile et leur procédé de préparation |
US9415020B2 (en) | 2012-01-19 | 2016-08-16 | The Johns Hopkins University | Nanoparticle formulations with enhanced mucosal penetration |
WO2013116126A1 (fr) | 2012-02-01 | 2013-08-08 | Merck Sharp & Dohme Corp. | Nouveaux lipides cationiques biodégradables de faible masse moléculaire pour la délivrance d'oligonucléotides |
EP2964234A4 (fr) | 2013-03-09 | 2016-12-07 | Moderna Therapeutics Inc | Régions non traduites hétérologues pour arnm |
EP3110401A4 (fr) | 2014-02-25 | 2017-10-25 | Merck Sharp & Dohme Corp. | Adjuvants de vaccins sous forme de nanoparticules lipidiques et systèmes d'administration d'antigènes |
US20200131498A1 (en) * | 2017-06-14 | 2020-04-30 | Modernatx, Inc. | Polynucleotides encoding methylmalonyl-coa mutase |
EP3849617A1 (fr) * | 2018-09-14 | 2021-07-21 | Translate Bio, Inc. | Compositions et méthodes de traitement de l'acidémie méthylmalonique |
-
2020
- 2020-05-08 WO PCT/US2020/032055 patent/WO2020227615A1/fr unknown
- 2020-05-08 EP EP20731624.1A patent/EP3965806A1/fr active Pending
- 2020-05-08 MA MA055899A patent/MA55899A/fr unknown
- 2020-05-08 US US17/608,691 patent/US20220370354A1/en active Pending
Also Published As
Publication number | Publication date |
---|---|
WO2020227615A1 (fr) | 2020-11-12 |
US20220370354A1 (en) | 2022-11-24 |
EP3965806A1 (fr) | 2022-03-16 |
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