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1. WO2020118246 - OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF

Publication Number WO/2020/118246
Publication Date 11.06.2020
International Application No. PCT/US2019/065058
International Filing Date 06.12.2019
IPC
C12N 15/113
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C12N 15/11 2006.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
C12N 15/09 2006.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
Applicants
  • WAVE LIFE SCIENCES LTD. [SG]/[SG]
  • ZHANG, Jason Jingxin [US]/[US] (US)
  • VARGEESE, Chandra [US]/[US] (US)
  • IWAMOTO, Naoki [JP]/[US] (US)
  • SHIVALILA, Chikdu Shakti [US]/[US] (US)
  • KOTHARI, Nayantara [US]/[US] (US)
  • DURBIN, Ann Fiegen [US]/[US] (US)
  • RAMASAMY, Selvi [IN]/[US] (US)
  • KANDASAMY, Pachamuthu [CA]/[US] (US)
  • KUMARASAMY, Jayakanthan [CA]/[US] (US)
  • BOMMINENI, Gopal Reddy [US]/[US] (US)
  • MARAPPAN, Subramanian [US]/[US] (US)
  • DIVAKARAMENON, Sethumadhavan [IN]/[US] (US)
  • BUTLER, David Charles Donnell [GB]/[US] (US)
  • LU, Genliang [US]/[US] (US)
  • YANG, Hailin [US]/[US] (US)
  • SHIMIZU, Mamoru [JP]/[US] (US)
  • MONIAN, Prashant [IN]/[US] (US)
Inventors
  • ZHANG, Jason Jingxin
  • VARGEESE, Chandra
  • IWAMOTO, Naoki
  • SHIVALILA, Chikdu Shakti
  • KOTHARI, Nayantara
  • DURBIN, Ann Fiegen
  • RAMASAMY, Selvi
  • KANDASAMY, Pachamuthu
  • KUMARASAMY, Jayakanthan
  • BOMMINENI, Gopal Reddy
  • MARAPPAN, Subramanian
  • DIVAKARAMENON, Sethumadhavan
  • BUTLER, David Charles Donnell
  • LU, Genliang
  • YANG, Hailin
  • SHIMIZU, Mamoru
  • MONIAN, Prashant
Agents
  • LI, Xiaodong
  • ADAMS, Melissa M.
  • ADATO, Ronen
  • AUGST, Alexander
  • BUTEAU, Kristen C.
  • BYCHOWSKI, Meaghan E.
  • D'AMATO, Erica M.
  • DAUKSS, Dana M.
  • FLYNN, Peter A.
  • HAULBROOK, William R.
  • JARRELL, Brenda Herschbach
  • JUDA, Cristin E.
  • LI, Bin
  • LYON, Charles E.
  • MA, Longle
  • MEDINA, Rolando
  • MONROE, Margo R.
  • PACE, Nicholas J.
  • REARICK, John P.
  • REESE, Brian E.
  • SCHMITT, Michael
  • SCHONEWALD, Stephanie L.
  • SHINALL, Michael A.
  • SHORE, David E.
  • SMITH, Maria C.
  • SUBMARANIAN, Sowmya
  • SUH, Su Kyung
  • TSE, Janet M.
  • VETTER, Michael L.
  • VRABLIK, Tracy L.
  • ZHANG, Jialan
Priority Data
62/776,43206.12.2018US
62/916,19216.10.2019US
62/916,19416.10.2019US
PCT/US2019/02710911.04.2019US
PCT/US2019/03167210.05.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
(FR) COMPOSITIONS D'OLIGONUCLÉOTIDES ET PROCÉDÉS ASSOCIÉS
Abstract
(EN)
Among other things, the present disclosure provides designed DMD oligonucleotides, compositions, and methods of use thereof. In some embodiments, the present disclosure provides technologies useful for repairing mutant DMD transcripts by skipping exon 51, so that the transcript can be translated into an internally truncated but at least partially functional Dystrophin protein variant. In some embodiments, the present disclosure provides technologies useful for modulating DMD transcript splicing. In some embodiments, provided technologies can alter splicing of a dystrophin (DMD) DMD transcript. In some embodiments, the present disclosure provides methods for treating diseases, such as muscular dystrophy, including but not limited to Duchenne muscular dystrophy, Becker's muscular dystrophy, etc.
(FR)
Entre autres, la présente invention concerne des oligonucléotides DMD sur mesure, des compositions et des procédés d'utilisation de ceux-ci. Dans certains modes de réalisation, la présente invention concerne des technologies utiles pour réparer des transcrits DMD mutants par saut de l'exon 51, de telle sorte que le transcrit peut être traduit en un variant de protéine de dystrophine tronquée intérieurement mais au moins partiellement fonctionnelle. Dans certains modes de réalisation, la présente invention concerne des technologies utiles pour moduler l'épissage de transcrit DMD. Dans certains modes de réalisation, des technologies fournies peuvent modifier l'épissage d'un transcrit DMD de dystrophine (DMD). Dans certains modes de réalisation, la présente invention concerne des méthodes de traitement de maladies, telles que la dystrophie musculaire y compris mais sans limitation la dystrophie musculaire de Duchenne, la dystrophie musculaire de Becker, etc.
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