SG11201811189RA - Variant adeno-associated viruses and methods of using - Google Patents

Variant adeno-associated viruses and methods of using

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Publication number
SG11201811189RA
SG11201811189RA SG11201811189RA SG11201811189RA SG11201811189RA SG 11201811189R A SG11201811189R A SG 11201811189RA SG 11201811189R A SG11201811189R A SG 11201811189RA SG 11201811189R A SG11201811189R A SG 11201811189RA SG 11201811189R A SG11201811189R A SG 11201811189RA
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SG
Singapore
Prior art keywords
international
ashburn
virginia
helix
pct
Prior art date
Application number
SG11201811189RA
Inventor
Joshua Dudman
Adam Hantman
Loren Looger
Kimberly Ritola
David Schaffer
Gowan Tervo
Sarada Viswanathan
Original Assignee
Joshua Dudman
Adam Hantman
Hwang Bum Yeol
Karpova Alla
Loren Looger
Kimberly Ritola
David Schaffer
Gowan Tervo
Sarada Viswanathan
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Publication date
Application filed by Joshua Dudman, Adam Hantman, Hwang Bum Yeol, Karpova Alla, Loren Looger, Kimberly Ritola, David Schaffer, Gowan Tervo, Sarada Viswanathan filed Critical Joshua Dudman
Publication of SG11201811189RA publication Critical patent/SG11201811189RA/en

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    • 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
    • C07K14/01—DNA viruses
    • C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
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    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • 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
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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
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
    • A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • 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
    • A61P27/00—Drugs for disorders of the senses
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    • 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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    • C40—COMBINATORIAL TECHNOLOGY
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    • C40B30/06—Methods of screening libraries by measuring effects on living organisms, tissues or cells
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    • C40B40/00—Libraries per se, e.g. arrays, mixtures
    • C40B40/04—Libraries containing only organic compounds
    • C40B40/06—Libraries containing nucleotides or polynucleotides, or derivatives thereof
    • C40B40/08—Libraries containing RNA or DNA which encodes proteins, e.g. gene libraries
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145—Special targeting system for viral vectors
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    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
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    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property 1111111011110111011111111010111110011111111111111111111111111111111111111111111111 Organization International Bureau (10) International Publication Number (43) International Publication Date .....0\"\"\" WO 2017/218842 Al 21 December 2017 (21.12.2017) WIP0 I PCT (51) International Patent Classification: Kimberly [US/US]; 19700 Helix Drive, Ashburn, Vir- C07K 14/015 (2006.01) A61K 48/00 (2006.01) ginia 20147 (US). SCHAFFER, David [US/US]; 19 Rima C12N 7/01 (2006.01) Court, Danville, California 94526 (US). TERVO, Gowan (21) International Application Number: [CA/US]; 19700 Helix Dr., Ashburn, Virginia 20147 (US). PCT/US2017/037781 VISWANATHAN, Sarada [US/US]; 19700 Helix Drive, Ashburn, Virginia 20147 (US). (22) International Filing Date: (74) Agent: PARSONS, Angela M. et al.; Fish & Richardson 15 June 2017 (15.06.2017) P.C., P.O. Box 1022, Minneapolis, Minnesota 55440-1022 (25) Filing Language: English (US). (26) Publication Language: English (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, (30) Priority Data: AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, 62/350,361 15 June 2016 (15.06.2016) US CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, 62/404,585 05 October 2016 (05.10.2016) US DZ, EC, EE, EG, ES, FL GB, GD, GE, GH, GM, GT, HN, (71) Applicants: HWANG, Bum-Yeol [KR/US]; 303 Deer- HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, field Dr., Moraga, California 94556 (US). KARPOVA, Al- KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, la [US/US]; 19700 Helix Drive, Ashburn, Virginia 20147 MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, (US). OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, (72) Inventors; and TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. _ (71) Applicants: DUDMAN, Joshua [US/US]; 207 Browns = Meadow Ct, Leesburg, Virginia 20176 (US). HANT- (84) Designated States (unless otherwise indicated, for every MAN, Adam [US/US]; 19700 Helix Drive, Ashburn, Vir- kind of regional protection available): ARIPO (BW, GH, = ginia 20147 (US). LOOGER, Loren [US/US]; 20149 GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, _ Broad Run Dr, Sterling, Virginia 20165 (US). RITOLA, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, = = (54) Title: VARIANT ADENO-ASSOCIATED - rAAVX or CAV2 = Syn-Cre = = VIRUSES AND METHODS OF USING (57) : The present disclosure provides AAV variants that exhibit a pref- erence for retrograde movement in neurons and methods of using such variants. I I = = tx : = = Rosa26-LSL-H2B-GFP = = = = = 11 ei ii- cc cc 1-1 ei IN 1-1 FIG. 2B 0 ei 4 .BPN C [Continued on next page] WO 2017/218842 Al MIDEDIMOMOIDEIRMEM00111HEIEHIMOVOIMIE TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). Published: — with international search report (Art. 21(3)) — before the expiration of the time limit for amending the claims and to be republished in the event of receipt of amendments (Rule 48.2(h)) — with sequence listing part of description (Rule 5.2(a))
SG11201811189RA 2016-06-15 2017-06-15 Variant adeno-associated viruses and methods of using SG11201811189RA (en)

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US201662350361P 2016-06-15 2016-06-15
US201662404585P 2016-10-05 2016-10-05
PCT/US2017/037781 WO2017218842A1 (en) 2016-06-15 2017-06-15 Variant adeno-associated viruses and methods of using

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EP (2) EP4268852A3 (en)
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KR (2) KR102522661B1 (en)
CN (2) CN116003532A (en)
AU (2) AU2017286652B2 (en)
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DK (1) DK3472183T3 (en)
EA (1) EA201990033A1 (en)
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ZA201900278B (en) 2022-04-28
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