WO2021229255A1 - Outils et procédé de prévention d'une neutralisation des virus vaa par des anticorps - Google Patents

Outils et procédé de prévention d'une neutralisation des virus vaa par des anticorps Download PDF

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Publication number
WO2021229255A1
WO2021229255A1 PCT/IB2020/000534 IB2020000534W WO2021229255A1 WO 2021229255 A1 WO2021229255 A1 WO 2021229255A1 IB 2020000534 W IB2020000534 W IB 2020000534W WO 2021229255 A1 WO2021229255 A1 WO 2021229255A1
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WO
WIPO (PCT)
Prior art keywords
aav
seq
capsid
peptide
identity
Prior art date
Application number
PCT/IB2020/000534
Other languages
English (en)
Inventor
Bérangère BERTIN
Federico Mingozzi
Elena PILETSKA
Sergey Piletsky
Philippe Veron
Original Assignee
Genethon
Institut National De La Sante Et De La Recherche Medicale (I.N.S.E.R.M.)
Universite D'evry Val D'essonne (Ueve)
University Of Leicester
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 Genethon, Institut National De La Sante Et De La Recherche Medicale (I.N.S.E.R.M.), Universite D'evry Val D'essonne (Ueve), University Of Leicester filed Critical Genethon
Priority to PCT/IB2020/000534 priority Critical patent/WO2021229255A1/fr
Priority to PCT/IB2021/054131 priority patent/WO2021229513A1/fr
Publication of WO2021229255A1 publication Critical patent/WO2021229255A1/fr

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    • 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
    • C07K7/08—Linear peptides containing only normal peptide links having 12 to 20 amino acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0014—Skin, i.e. galenical aspects of topical compositions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A61K9/0024—Solid, semi-solid or solidifying implants, which are implanted or injected in body tissue
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0043—Nose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0087—Galenical forms not covered by A61K9/02 - A61K9/7023
    • A61K9/0095—Drinks; Beverages; Syrups; Compositions for reconstitution thereof, e.g. powders or tablets to be dispersed in a glass of water; Veterinary drenches
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/08—Solutions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/20—Pills, tablets, discs, rods
    • A61K9/2004—Excipients; Inactive ingredients
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/4841—Filling excipients; Inactive ingredients
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B01—PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01J—CHEMICAL OR PHYSICAL PROCESSES, e.g. CATALYSIS OR COLLOID CHEMISTRY; THEIR RELEVANT APPARATUS
    • B01J20/00—Solid sorbent compositions or filter aid compositions; Sorbents for chromatography; Processes for preparing, regenerating or reactivating thereof
    • B01J20/22—Solid sorbent compositions or filter aid compositions; Sorbents for chromatography; Processes for preparing, regenerating or reactivating thereof comprising organic material
    • B01J20/26—Synthetic macromolecular compounds
    • B01J20/268—Polymers created by use of a template, e.g. molecularly imprinted polymers
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B01—PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01J—CHEMICAL OR PHYSICAL PROCESSES, e.g. CATALYSIS OR COLLOID CHEMISTRY; THEIR RELEVANT APPARATUS
    • B01J20/00—Solid sorbent compositions or filter aid compositions; Sorbents for chromatography; Processes for preparing, regenerating or reactivating thereof
    • B01J20/30—Processes for preparing, regenerating, or reactivating
    • B01J20/32—Impregnating or coating ; Solid sorbent compositions obtained from processes involving impregnating or coating
    • B01J20/3202—Impregnating or coating ; Solid sorbent compositions obtained from processes involving impregnating or coating characterised by the carrier, support or substrate used for impregnation or coating
    • B01J20/3206—Organic carriers, supports or substrates
    • B01J20/3208—Polymeric carriers, supports or substrates
    • B01J20/3212—Polymeric carriers, supports or substrates consisting of a polymer obtained by reactions otherwise than involving only carbon to carbon unsaturated bonds
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B01—PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01J—CHEMICAL OR PHYSICAL PROCESSES, e.g. CATALYSIS OR COLLOID CHEMISTRY; THEIR RELEVANT APPARATUS
    • B01J20/00—Solid sorbent compositions or filter aid compositions; Sorbents for chromatography; Processes for preparing, regenerating or reactivating thereof
    • B01J20/30—Processes for preparing, regenerating, or reactivating
    • B01J20/32—Impregnating or coating ; Solid sorbent compositions obtained from processes involving impregnating or coating
    • B01J20/3214—Impregnating or coating ; Solid sorbent compositions obtained from processes involving impregnating or coating characterised by the method for obtaining this coating or impregnating
    • B01J20/3217—Resulting in a chemical bond between the coating or impregnating layer and the carrier, support or substrate, e.g. a covalent bond
    • B01J20/3219—Resulting in a chemical bond between the coating or impregnating layer and the carrier, support or substrate, e.g. a covalent bond involving a particular spacer or linking group, e.g. for attaching an active group
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B01—PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01J—CHEMICAL OR PHYSICAL PROCESSES, e.g. CATALYSIS OR COLLOID CHEMISTRY; THEIR RELEVANT APPARATUS
    • B01J20/00—Solid sorbent compositions or filter aid compositions; Sorbents for chromatography; Processes for preparing, regenerating or reactivating thereof
    • B01J20/30—Processes for preparing, regenerating, or reactivating
    • B01J20/32—Impregnating or coating ; Solid sorbent compositions obtained from processes involving impregnating or coating
    • B01J20/3231—Impregnating or coating ; Solid sorbent compositions obtained from processes involving impregnating or coating characterised by the coating or impregnating layer
    • B01J20/3242—Layers with a functional group, e.g. an affinity material, a ligand, a reactant or a complexing group
    • B01J20/3268—Macromolecular compounds
    • B01J20/327—Polymers obtained by reactions involving only carbon to carbon unsaturated bonds
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B01—PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01J—CHEMICAL OR PHYSICAL PROCESSES, e.g. CATALYSIS OR COLLOID CHEMISTRY; THEIR RELEVANT APPARATUS
    • B01J20/00—Solid sorbent compositions or filter aid compositions; Sorbents for chromatography; Processes for preparing, regenerating or reactivating thereof
    • B01J20/30—Processes for preparing, regenerating, or reactivating
    • B01J20/32—Impregnating or coating ; Solid sorbent compositions obtained from processes involving impregnating or coating
    • B01J20/3291—Characterised by the shape of the carrier, the coating or the obtained coated product
    • B01J20/3295—Coatings made of particles, nanoparticles, fibers, nanofibers
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/001—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof by chemical synthesis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/08—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from viruses
    • C07K16/081—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from viruses from DNA viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K17/00—Carrier-bound or immobilised peptides; Preparation thereof
    • C07K17/02—Peptides being immobilised on, or in, an organic carrier
    • C07K17/08—Peptides being immobilised on, or in, an organic carrier the carrier being a synthetic polymer
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B01—PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01D—SEPARATION
    • B01D15/00—Separating processes involving the treatment of liquids with solid sorbents; Apparatus therefor
    • B01D15/08—Selective adsorption, e.g. chromatography
    • B01D15/26—Selective adsorption, e.g. chromatography characterised by the separation mechanism
    • B01D15/38—Selective adsorption, e.g. chromatography characterised by the separation mechanism involving specific interaction not covered by one or more of groups B01D15/265 - B01D15/36
    • B01D15/3852—Selective adsorption, e.g. chromatography characterised by the separation mechanism involving specific interaction not covered by one or more of groups B01D15/265 - B01D15/36 using imprinted phases or molecular recognition; using imprinted phases

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  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Analytical Chemistry (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Biophysics (AREA)
  • Dermatology (AREA)
  • Engineering & Computer Science (AREA)
  • Otolaryngology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nanotechnology (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Dispersion Chemistry (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Peptides Or Proteins (AREA)

Abstract

L'invention concerne un peptide qui se lie spécifiquement à une capside du VAA, ledit peptide comprenant une séquence d'acides aminés ayant au moins 80 % d'identité avec une séquence d'acides aminés choisie parmi SEQ ID NO : 1 et SEQ ID No: 2 et SEQ ID No: 3 et SEQ ID No: 4 et SEQ ID No: 5 et/ou SEQ ID NO: 6, L'invention concerne en outre une composition comprenant un ou plusieurs peptides de l'invention, un kit de pièces comprenant (i) un peptide de l'invention ou une composition de l'invention et (ii) un vecteur du VAA. L'invention concerne également une particule de polymère à empreinte moléculaire (MIP) qui se lie spécifiquement à un peptide de l'invention.
PCT/IB2020/000534 2020-05-14 2020-05-14 Outils et procédé de prévention d'une neutralisation des virus vaa par des anticorps WO2021229255A1 (fr)

Priority Applications (2)

Application Number Priority Date Filing Date Title
PCT/IB2020/000534 WO2021229255A1 (fr) 2020-05-14 2020-05-14 Outils et procédé de prévention d'une neutralisation des virus vaa par des anticorps
PCT/IB2021/054131 WO2021229513A1 (fr) 2020-05-14 2021-05-14 Outils et procédé pour empêcher la neutralisation d'aav par des anticorps

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
PCT/IB2020/000534 WO2021229255A1 (fr) 2020-05-14 2020-05-14 Outils et procédé de prévention d'une neutralisation des virus vaa par des anticorps

Publications (1)

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WO2021229255A1 true WO2021229255A1 (fr) 2021-11-18

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PCT/IB2020/000534 WO2021229255A1 (fr) 2020-05-14 2020-05-14 Outils et procédé de prévention d'une neutralisation des virus vaa par des anticorps
PCT/IB2021/054131 WO2021229513A1 (fr) 2020-05-14 2021-05-14 Outils et procédé pour empêcher la neutralisation d'aav par des anticorps

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Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015013313A2 (fr) 2013-07-22 2015-01-29 The Children's Hospital Of Philadelphia Compositions et variants de virus adéno-associés, et méthodes et utilisations pour un transfert de gènes dans des cellules, des organes et des tissus
US9861661B2 (en) 2008-02-14 2018-01-09 Life Sciences Research Partners Vzw Elimination of immune responses to viral vectors
WO2018158397A1 (fr) 2017-03-02 2018-09-07 Genethon Procédé d'élimination d'anticorps anti-aav présents dans une composition dérivée du sang
WO2018178629A1 (fr) 2017-03-27 2018-10-04 University Of Leicester Procédés et trousses de détermination de sites de liaison
WO2019193119A1 (fr) 2018-04-05 2019-10-10 Genethon Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduit

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9861661B2 (en) 2008-02-14 2018-01-09 Life Sciences Research Partners Vzw Elimination of immune responses to viral vectors
WO2015013313A2 (fr) 2013-07-22 2015-01-29 The Children's Hospital Of Philadelphia Compositions et variants de virus adéno-associés, et méthodes et utilisations pour un transfert de gènes dans des cellules, des organes et des tissus
WO2018158397A1 (fr) 2017-03-02 2018-09-07 Genethon Procédé d'élimination d'anticorps anti-aav présents dans une composition dérivée du sang
WO2018178629A1 (fr) 2017-03-27 2018-10-04 University Of Leicester Procédés et trousses de détermination de sites de liaison
WO2019193119A1 (fr) 2018-04-05 2019-10-10 Genethon Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduit

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Title
BERTIN B ET AL: "Capsid-specific removal of circulating antibodies to adeno-associated virus vectors", SCIENTIFIC REPORTS, vol. 10, no. 1, 21 January 2020 (2020-01-21), UK, pages 864, XP055767370, ISSN: 2045-2322, DOI: 10.1038/s41598-020-57893-z *
BOUTIN ET AL., HUMAN GENE THERAPY, vol. 21, 2010, pages 704 - 712
CANFAROTTA F ET AL., SOLID-PHASE SYNTHESIS OF MOLECULARLY IMPRINTED NANOPARTICLES, 2016
DATABASE Geneseq [online] 11 January 2018 (2018-01-11), "Human PTPRO protein, SEQ ID 5281.", XP002801776, retrieved from EBI accession no. GSP:BEP85036 Database accession no. BEP85036 *
DATABASE Geneseq [online] 15 April 2004 (2004-04-15), "Human NOVX protein homologue SeqID 655.", XP002801778, retrieved from EBI accession no. GSP:ADI17119 Database accession no. ADI17119 *
DATABASE Geneseq [online] 15 April 2004 (2004-04-15), "Human NOVX protein to treat human pathological conditions SeqID246.", XP002801777, retrieved from EBI accession no. GSP:ADI16710 Database accession no. ADI16710 *
DATABASE Geneseq [online] 4 December 2003 (2003-12-04), "Human secreted protein #SEQ ID 780.", XP002801775, retrieved from EBI accession no. GSP:ADB91834 Database accession no. ADB91834 *
DATABASE Geneseq [online] 6 May 2004 (2004-05-06), "Novel human polypeptide SeqID7853.", XP002801774, retrieved from EBI accession no. GSP:ADK35771 Database accession no. ADK35771 *
KOTTERMAN M. A.SCHAFFER D. V.: "Engineering adeno-associated viruses for clinical gene therapy", NAT REV GENET., vol. 15, 2014, pages 445 - 451, XP055317129, DOI: 10.1038/nrg3742
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MINGOZZI ET AL., CELL-MEDIATED IMMUNITY TO AAV VECTORS, EVOLVING CONCEPTS AND POTENTIAL SOLUTIONS, 2014
MINGOZZI, CD8+ T-CELL RESPONSES TO ADENO-ASSOCIATED VIRUS CAPSID IN HUMANS, 2007
MOCZKO E ET AL., EPITOPE APPROACH IN MOLECULAR IMPRINTING OF ANTIBODIES, 2019
MOCZKO E ET AL: "Epitope approach in molecular imprinting of antibodies", JOURNAL OF CHROMATOGRAPHY B, ELSEVIER, AMSTERDAM, NL, vol. 1124, 25 May 2019 (2019-05-25), pages 1 - 6, XP085738095, ISSN: 1570-0232, [retrieved on 20190525], DOI: 10.1016/J.JCHROMB.2019.05.024 *
MOSKALENKO ET AL., EPITOPE MAPPING OF HUMAN ANTI-ADENO-ASSOCIATED VIRUS TYPE 2 NEUTRALIZING ANTIBODIES: IMPLICATIONS FOR GENE THERAPY AND VIRUS STRUCTURE, 2000
MURPHY ET AL., DIVERSE IGG SUBCLASS RESPONSES TO ADENO-ASSOCIATED VIRUS INFECTION AND VECTOR ADMINISTRATION, 2009
PILETSKA E V ET AL: "Combinatorial screening of polymer nanoparticles for their ability to recognize epitopes of AAV-neutralizing antibodies", JOURNAL OF MOLECULAR RECOGNITION., vol. 33, no. 4, 19 November 2019 (2019-11-19), GB, pages e2824, XP055766341, ISSN: 0952-3499, DOI: 10.1002/jmr.2824 *
PILETSKY ET AL., MOLECULARLY IMPRINTED POLYMERS FOR CELL RECOGNITION, 2019
PULICHERLA N ET AL: "Peptide affinity reagents for AAV capsid recognition and purification", GENE THERAPY, vol. 18, no. 10, 14 April 2011 (2011-04-14), GB, pages 1020 - 1024, XP055766382, ISSN: 0969-7128, DOI: 10.1038/gt.2011.46 *
RAPTI ET AL., NEUTRALIZING ANTIBODIES AGAINST AAV SEROTYPES 1, 2, 6, AND 9 IN SERA OF COMMONLY USED ANIMAL MODELS, 2012
ROGERS ET AL., INNATE IMMUNE RESPONSES TO AAV VECTORS, 2011
ROSARIO ET AL., MOL THER METHODS CLIN DEV., vol. 3, 2016, pages 16026
STEINBACH S ET AL.: "Assembly of adeno-associated virus type 2 capsids in vitro", J GEN VIROL., vol. 78, June 1997 (1997-06-01), pages 1453 - 62, XP002346582
VERCAUTEREN ET AL., MOL. THER., vol. 24, no. 6, 2016, pages 1042

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