SG10201907396XA - VARIANT RNAi - Google Patents

VARIANT RNAi

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Publication number
SG10201907396XA
SG10201907396XA SG10201907396XA SG10201907396XA SG10201907396XA SG 10201907396X A SG10201907396X A SG 10201907396XA SG 10201907396X A SG10201907396X A SG 10201907396XA SG 10201907396X A SG10201907396X A SG 10201907396XA SG 10201907396X A SG10201907396X A SG 10201907396XA
Authority
SG
Singapore
Prior art keywords
rnai
guide sequence
recombinant
disease
further provided
Prior art date
Application number
SG10201907396XA
Inventor
Lisa Stanek
Adam Palermo
Brenda Richards
Sergio Sardi
Catherine O'riordan
Antonius Song
Original Assignee
Genzyme Corp
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Filing date
Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of SG10201907396XA publication Critical patent/SG10201907396XA/en

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/14—Type of nucleic acid interfering N.A.
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
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    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10022—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
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    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector

Abstract

VARIANT RNAi Provided herein are RNAi molecules including a first strand containing a guide sequence and a second strand comprising a non-guide sequence where the non-guide sequence contains a bulge opposite the seed 5 region of the guide sequences; e.g., opposite the cleavage sequence. In some aspects, the invention provides RNAi for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington' s disease. 10 [Figure 1A]
SG10201907396XA 2015-02-10 2016-02-09 VARIANT RNAi SG10201907396XA (en)

Applications Claiming Priority (1)

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US201562114578P 2015-02-10 2015-02-10

Publications (1)

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SG10201907396XA true SG10201907396XA (en) 2019-09-27

Family

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SG11201706444TA SG11201706444TA (en) 2015-02-10 2016-02-09 VARIANT RNAi
SG10201907396XA SG10201907396XA (en) 2015-02-10 2016-02-09 VARIANT RNAi
SG10201912942YA SG10201912942YA (en) 2015-02-10 2016-02-10 VARIANT RNAi

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SG11201706444TA SG11201706444TA (en) 2015-02-10 2016-02-09 VARIANT RNAi

Family Applications After (1)

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SG10201912942YA SG10201912942YA (en) 2015-02-10 2016-02-10 VARIANT RNAi

Country Status (26)

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US (3) US10450563B2 (en)
EP (1) EP3256588A2 (en)
JP (2) JP2018506304A (en)
KR (1) KR20170110149A (en)
CN (1) CN107438671B (en)
AR (1) AR103646A1 (en)
AU (2) AU2016219396B2 (en)
BR (1) BR112017017028A2 (en)
CA (1) CA2976075A1 (en)
CL (1) CL2017002027A1 (en)
CO (1) CO2017009083A2 (en)
CR (1) CR20170406A (en)
EA (1) EA201791805A1 (en)
EC (1) ECSP17059343A (en)
HK (1) HK1247641A1 (en)
IL (1) IL253893B (en)
MA (1) MA40819B1 (en)
MX (2) MX2017010369A (en)
MY (2) MY194175A (en)
PE (1) PE20171382A1 (en)
PH (1) PH12017501432A1 (en)
SG (3) SG11201706444TA (en)
TN (1) TN2017000354A1 (en)
TW (1) TWI781079B (en)
UY (1) UY36554A (en)
WO (1) WO2016130589A2 (en)

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WO2016130589A3 (en) 2016-09-15
AU2016219396B2 (en) 2022-03-17
EP3256588A2 (en) 2017-12-20
WO2016130589A2 (en) 2016-08-18
TWI781079B (en) 2022-10-21
BR112017017028A2 (en) 2018-04-10
SG10201912942YA (en) 2020-02-27
US11781137B2 (en) 2023-10-10
AU2022204274A1 (en) 2022-07-07
US10760079B2 (en) 2020-09-01
IL253893B (en) 2021-08-31
SG11201706444TA (en) 2017-09-28
MY194175A (en) 2022-11-17
JP2018506304A (en) 2018-03-08
KR20170110149A (en) 2017-10-10
AU2016219396A1 (en) 2017-09-28
UY36554A (en) 2016-09-30
MX2017010369A (en) 2017-12-14
TW201704470A (en) 2017-02-01
IL253893A0 (en) 2017-10-31
US20200109401A1 (en) 2020-04-09
AR103646A1 (en) 2017-05-24
CL2017002027A1 (en) 2018-04-20
HK1247641A1 (en) 2018-09-28
PE20171382A1 (en) 2017-09-15
US20180023082A1 (en) 2018-01-25
CN107438671B (en) 2021-05-25
MA40819A1 (en) 2018-07-31
US20210047641A1 (en) 2021-02-18
ECSP17059343A (en) 2019-03-29
MY181458A (en) 2020-12-22
CN107438671A (en) 2017-12-05
TN2017000354A1 (en) 2019-01-16
PH12017501432A1 (en) 2018-01-15
CO2017009083A2 (en) 2018-01-31
JP2021118740A (en) 2021-08-12
MX2022013005A (en) 2022-11-09
CA2976075A1 (en) 2016-08-18
US10450563B2 (en) 2019-10-22
MA40819B1 (en) 2020-04-30
CR20170406A (en) 2017-11-14
EA201791805A1 (en) 2018-05-31

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