MA49634A - Arnm modifié codant pour une propionyl-coa-carboxylase et utilisations associées - Google Patents

Arnm modifié codant pour une propionyl-coa-carboxylase et utilisations associées

Info

Publication number
MA49634A
MA49634A MA049634A MA49634A MA49634A MA 49634 A MA49634 A MA 49634A MA 049634 A MA049634 A MA 049634A MA 49634 A MA49634 A MA 49634A MA 49634 A MA49634 A MA 49634A
Authority
MA
Morocco
Prior art keywords
carboxylase
propionyl
coa
modified rna
rna coding
Prior art date
Application number
MA049634A
Other languages
English (en)
Inventor
Judith L Campagnari
Zhiliang Cheng
Susan Sobolov-Jaynes
Romesh R Subramanian
Haren Vasavada
Original Assignee
Modernatx 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 Modernatx Inc filed Critical Modernatx Inc
Publication of MA49634A publication Critical patent/MA49634A/fr

Links

Classifications

    • 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
    • C12N9/93—Ligases (6)
    • 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
    • C12N9/88—Lyases (4.)
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y401/00—Carbon-carbon lyases (4.1)
    • C12Y401/01—Carboxy-lyases (4.1.1)
    • C12Y401/01041—Methylmalonyl-CoA decarboxylase (4.1.1.41)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y604/00—Ligases forming carbon-carbon bonds (6.4)
    • C12Y604/01—Ligases forming carbon-carbon bonds (6.4.1)
    • C12Y604/01003—Propionyl-CoA carboxylase (6.4.1.3)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
MA049634A 2017-07-21 2018-07-20 Arnm modifié codant pour une propionyl-coa-carboxylase et utilisations associées MA49634A (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201762535289P 2017-07-21 2017-07-21

Publications (1)

Publication Number Publication Date
MA49634A true MA49634A (fr) 2020-05-27

Family

ID=63165470

Family Applications (1)

Application Number Title Priority Date Filing Date
MA049634A MA49634A (fr) 2017-07-21 2018-07-20 Arnm modifié codant pour une propionyl-coa-carboxylase et utilisations associées

Country Status (4)

Country Link
US (1) US20200165593A1 (fr)
EP (1) EP3655040A1 (fr)
MA (1) MA49634A (fr)
WO (1) WO2019018765A1 (fr)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA50802A (fr) * 2017-11-22 2020-09-30 Modernatx Inc Polynucléotides codant pour des sous-unités alpha et bêta de propionyl-coa carboxylase pour le traitement de l'acidémie propionique
WO2023225524A1 (fr) * 2022-05-17 2023-11-23 Modernatx, Inc. Préparation d'arnm hautement concentré

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4485045A (en) 1981-07-06 1984-11-27 Research Corporation Synthetic phosphatidyl cholines useful in forming liposomes
US4544545A (en) 1983-06-20 1985-10-01 Trustees University Of Massachusetts Liposomes containing modified cholesterol for organ targeting
US5013556A (en) 1989-10-20 1991-05-07 Liposome Technology, Inc. Liposomes with enhanced circulation time
SI3611266T1 (sl) 2005-08-23 2023-02-28 The Trustees Of The University Of Pennsylvania Modificirani nukleozidi, ki vsebujejo RNA, in postopki za njihovo uporabo
CN101378782A (zh) 2005-12-21 2009-03-04 惠氏公司 粘度降低的蛋白质制剂及其用途
PT3338765T (pt) 2009-12-01 2019-03-18 Translate Bio Inc Derivado de esteróide adequado para a administração de arnm em doenças genéticas humanas
WO2012075040A2 (fr) 2010-11-30 2012-06-07 Shire Human Genetic Therapies, Inc. Arnm pour l'utilisation dans le traitement de maladies génétiques humaines
WO2012129648A1 (fr) 2011-03-25 2012-10-04 University Of Guelph Activation d'expression de protéines de vecteurs de virus adéno-associés
EP2791160B1 (fr) 2011-12-16 2022-03-02 ModernaTX, Inc. Compositions de mrna modifiés
AU2013243946A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of membrane proteins
EA201492055A1 (ru) 2012-06-08 2015-11-30 Шир Хьюман Дженетик Терапис, Инк. ИНГАЛЯЦИОННАЯ ДОСТАВКА мРНК В НЕЛЕГОЧНЫЕ КЛЕТКИ-МИШЕНИ
LT2922554T (lt) 2012-11-26 2022-06-27 Modernatx, Inc. Terminaliai modifikuota rnr
US20160032316A1 (en) * 2013-03-14 2016-02-04 The Trustees Of The University Of Pennsylvania Purification and Purity Assessment of RNA Molecules Synthesized with Modified Nucleosides
CA2974503A1 (fr) * 2015-01-21 2016-07-28 Phaserx, Inc. Procedes, compositions et systemes permettant l'administration d'agents therapeutiques et diagnostiques dans des cellules
US11389546B2 (en) * 2015-12-09 2022-07-19 Modernatx, Inc. Heterologous UTR sequences for enhanced mRNA expression

Also Published As

Publication number Publication date
WO2019018765A1 (fr) 2019-01-24
EP3655040A1 (fr) 2020-05-27
US20200165593A1 (en) 2020-05-28

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