SG11201804713VA - TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs) - Google Patents

TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)

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Publication number
SG11201804713VA
SG11201804713VA SG11201804713VA SG11201804713VA SG11201804713VA SG 11201804713V A SG11201804713V A SG 11201804713VA SG 11201804713V A SG11201804713V A SG 11201804713VA SG 11201804713V A SG11201804713V A SG 11201804713VA SG 11201804713V A SG11201804713V A SG 11201804713VA
Authority
SG
Singapore
Prior art keywords
california
international
iii
aavs
pasadena
Prior art date
Application number
SG11201804713VA
Inventor
Benjamin Deverman
Viviana Gradinaru
Ken Chan
Original Assignee
California Inst Of Techn
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 California Inst Of Techn filed Critical California Inst Of Techn
Publication of SG11201804713VA publication Critical patent/SG11201804713VA/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
    • C12N15/63—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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    • A61K38/08—Peptides having 5 to 11 amino acids
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    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18—Growth factors; Growth regulators
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    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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    • 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
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    • 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
    • C—CHEMISTRY; METALLURGY
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • C07K14/01—DNA viruses
    • C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04—Linear peptides containing only normal peptide links
    • C07K7/06—Linear peptides containing only normal peptide links having 5 to 11 amino acids
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    • 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
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    • C12Y305/01015—Aspartoacylase (3.5.1.15)

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Abstract

Title: TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs) AAV-PHRS 1 x 10' vg AAV-PHPeB 1 x 10 1 vg AAV-PHRB 1 x 10 1 vg AAV9 1x 10' 2 vg FIG. 23A (57) : Disclosed herein are peptide sequences capable of directing adeno-associated viruses (AAV) to target specific envir- onments, for example the nervous system and the heart, in a subject. Also disclosed are AAVs having non-naturally occurring capsid proteins comprising the disclosed peptide sequences, and methods of using the AAVs to treat diseases. 999900 ASH (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau 111111111111110 III OE III OE III II III 1111111111111111111111111 RIII 11111111 (10) International Publication Number WO 2017/100671 Al WIPO I PCT (43) International Publication Date 15 June 2017 (15.06.2017) (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, 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, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, 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). Declarations under Rule 4.17: (51) International Patent Classification: C12N 15/86 (2006.01) C07K 14/005 (2006.01) CO7K 7/06 (2006.01) (21) International Application Number: PCT/US2016/065969 (22) International Filing Date: 9 December 2016 (09.12.2016) English English (25) Filing Language: (26) Publication Language: (30) Priority Data: 62/266,184 11 December 2015 (11.12.2015) 62/421,891 14 November 2016 (14.11.2016) US US (71) Applicant: CALIFORNIA INSTITUTE OF TECHNO- LOGY [US/US]; Office Of Technology Transfer, 1200 E. California Blvd., Mail Code 6-32, Pasadena, CA 91125 (US). (72) Inventors: DEVERMAN, Benjamin, E.; c/o California Institute of Technology, 1200 E. California Blvd., Mail Code 6-32, Pasadena, CA 91125 (US). GRADINARU, Viviana; c/o California Institute of Technology, 1200 E. California Blvd., Mail Code 6-32, Pasadena, CA 91125 (US). CHAN, Ken, Y.; c/o California Institute of Techno- logy, 1200 E. California Blvd., Mail Code 6-32, Pasadena, CA 91125 (US). (74) Agent: ALTMAN, Daniel E.; Knobbe, Martens, Olson & Bear, LLP, 2040 Main Street, 14th Floor, Irvine, CA 92614 (US). as to applicant's entitlement to apply for and be granted a patent (Rule 4.170) as to the applicant's entitlement to claim the priority of the earlier application (Rule 4.17(iii)) Published: with international search report (Art 21(3)) with sequence listing part of description (Rule 5.2(a)) W O 20 17 / 10067 1 Al
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