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1. WO2020006423 - SYNTHETIC GUIDE MOLECULES, COMPOSITIONS AND METHODS RELATING THERETO

Publication Number WO/2020/006423
Publication Date 02.01.2020
International Application No. PCT/US2019/039848
International Filing Date 28.06.2019
IPC
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
70
Carbohydrates; Sugars; Derivatives thereof
7088
Compounds having three or more nucleosides or nucleotides
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
70
Carbohydrates; Sugars; Derivatives thereof
7088
Compounds having three or more nucleosides or nucleotides
7105
Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
9
Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
14
Hydrolases (3.)
16
acting on ester bonds (3.1)
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
9
Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
14
Hydrolases (3.)
16
acting on ester bonds (3.1)
22
Ribonucleases
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
11
DNA or RNA fragments; Modified forms thereof
113
Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A61K 31/7088 (2006.01)
A61K 31/7105 (2006.01)
C12N 9/16 (2006.01)
C12N 9/22 (2006.01)
C12N 15/113 (2010.01)
CPC
A61K 31/7088
A61K 31/7105
C12N 15/111
C12N 2310/20
C12N 2310/3181
C12N 2310/3183
Applicants
  • EDITAS MEDICINE, INC. [US/US]; 11 Hurley St. Cambridge, Massachusetts 02141, US
Inventors
  • SACCOMANO, Sam; US
  • CAPEHART, Stacy; US
  • EATON, Bruce; US
  • BERRY, Karin Zemski; US
Agents
  • D'AMATO, Erica M.; US
  • ADATO, Ronen; US
  • AUGST, Alexander D.; US
  • BUTEAU, Kristen C.; US
  • BYCHOWSKI, Meaghan E.; US
  • DAUKSS, Dana M.; US
  • HAULBROOK, William R.; US
  • HAWVER, Lisa A.; US
  • JARRELL, Brenda Herschbach; US
  • JUDA, Cristin E.; US
  • LI, Bin; US
  • LI, Xiaodong; US
  • LYON, Charles E.; US
  • MA, Longle; US
  • MEDINA, Rolando; US
  • MONG, Surin K.; US
  • MONROE, Margo R.; US
  • PACE, Nicholas J.; US
  • PYSHER, Paul A.; US
  • REARICK, John P.; US
  • REESE, Brian E.; US
  • SAHR, Robert N.; US
  • SCHMITT, Michael D.; US
  • SCHONEWALD, Stephanie L.; US
  • SHINALL, Michael A.; US
  • SHORE, David E.; US
  • SMITH, Maria C.; US
  • SUH, Su Kyung; US
  • TSE, Janet M.; US
  • VETTER, Michael L.; US
  • VRABLIK, Tracy L.; US
Priority Data
62/692,49229.06.2018US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) SYNTHETIC GUIDE MOLECULES, COMPOSITIONS AND METHODS RELATING THERETO
(FR) MOLÉCULES DE GUIDAGE SYNTHÉTIQUES, COMPOSITIONS ET PROCÉDÉS ASSOCIÉS
Abstract
(EN)
Chemical syntheses of guide molecules are disclosed, along with compositions and methods relating thereto. A cost-effective and straightforward chemical synthesis of high-purity unimolecular guide molecules with minimal n-1 and/or n+1 species, truncation species, and other contaminants by providing, among other things, methods for synthesizing unimolecular guide molecules that involve cross-linking two or more pre-annealed guide fragments.
(FR)
L'invention concerne des synthèses chimiques de molécules de guidage, ainsi que des compositions et des procédés s'y rapportant.
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