AU2020385387A1 - Materials and methods for treatment of disorders associated with the IGHMBP2 gene - Google Patents

Materials and methods for treatment of disorders associated with the IGHMBP2 gene Download PDF

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Publication number
AU2020385387A1
AU2020385387A1 AU2020385387A AU2020385387A AU2020385387A1 AU 2020385387 A1 AU2020385387 A1 AU 2020385387A1 AU 2020385387 A AU2020385387 A AU 2020385387A AU 2020385387 A AU2020385387 A AU 2020385387A AU 2020385387 A1 AU2020385387 A1 AU 2020385387A1
Authority
AU
Australia
Prior art keywords
raav
composition
ighmbp2
delivery
seq
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
AU2020385387A
Other languages
English (en)
Inventor
Stefania Paola CORTI
Kevin Foust
Brian K. Kaspar
Shibi LIKHITE
Kathrin Christine Meyer
Monica NIZZARDO
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Universita degli Studi di Milano
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Nationwide Childrens Hospital Inc
Original Assignee
Universita degli Studi di Milano
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Nationwide Childrens Hospital Inc
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 Universita degli Studi di Milano, Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico, Nationwide Childrens Hospital Inc filed Critical Universita degli Studi di Milano
Publication of AU2020385387A1 publication Critical patent/AU2020385387A1/en
Assigned to UNIVERSITÀ DEGLI STUDI DI MILANO, RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL, FONDAZIONE IRCCS CA' GRANDA OSPEDALE MAGGIORE FOLICLINICO reassignment UNIVERSITÀ DEGLI STUDI DI MILANO Amend patent request/document other than specification (104) Assignors: FONDAZIONE IRCCS CA' GRANDA OSPEDALE MAGGIORE FOLICLINICO, RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL, UNIVERSTA DEGLI STUDI DI MILANO
Pending legal-status Critical Current

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    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/04—Hydrolases acting on acid anhydrides (3.6) acting on acid anhydrides; involved in cellular and subcellular movement (3.6.4)
    • C12Y306/04012—DNA helicase (3.6.4.12)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2020385387A 2019-11-22 2020-11-23 Materials and methods for treatment of disorders associated with the IGHMBP2 gene Pending AU2020385387A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962939270P 2019-11-22 2019-11-22
US62/939,270 2019-11-22
PCT/US2020/061863 WO2021102435A1 (en) 2019-11-22 2020-11-23 Materials and methods for treatment of disorders associated with the ighmbp2 gene

Publications (1)

Publication Number Publication Date
AU2020385387A1 true AU2020385387A1 (en) 2022-06-02

Family

ID=73839115

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2020385387A Pending AU2020385387A1 (en) 2019-11-22 2020-11-23 Materials and methods for treatment of disorders associated with the IGHMBP2 gene

Country Status (6)

Country Link
US (1) US20230211018A1 (https=)
EP (1) EP4061831A1 (https=)
JP (2) JP7684296B2 (https=)
AU (1) AU2020385387A1 (https=)
IL (1) IL293210A (https=)
WO (1) WO2021102435A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2023360998A1 (en) 2022-10-11 2025-05-22 Research Institute At Nationwide Children's Hospital Adeno-associated virus delivery to treat spinal muscular atrophy with respiratory distress type 1 (smard1) and charcot-marie-tooth type 2s (cmt2s)

Family Cites Families (28)

* Cited by examiner, † Cited by third party
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US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
PT733103E (pt) 1993-11-09 2004-07-30 Targeted Genetics Corp Criacao de elevados titulos de vectores de aav recombinantes
PT728214E (pt) 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
AU707866B2 (en) 1994-12-06 1999-07-22 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
AU722196B2 (en) 1995-08-30 2000-07-27 Genzyme Corporation Chromatographic purification of adenovirus and AAV
EP1983057A3 (en) 1995-09-08 2009-01-07 Genzyme Corporation Improved AAV vectors for gene therapy
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
EP0932418B1 (en) 1996-09-06 2007-12-05 The Trustees Of The University Of Pennsylvania Method for recombinant adeno-associated virus-directed gene therapy
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
JP2001514845A (ja) 1997-09-05 2001-09-18 ターゲティッド ジェネティクス コーポレイション 組換えａａｖベクターの高力価ヘルパーなし調製物を生成するための方法
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
CA2406743A1 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
US20060110364A1 (en) * 2004-08-20 2006-05-25 Ludwig Institute For Cancer Research Vector-mediated delivery of polynucleotides encoding soluble VEGF receptors
EP2019588A4 (en) * 2006-05-04 2010-11-24 Univ Wayne State RESTORATION OF VISUAL RESPONSES BY IN VIVO ADMINISTRATION OF NUCLEIC ACIDS OF RHODOPSIN
WO2010134939A2 (en) * 2008-12-19 2010-11-25 Zirus, Inc. Mammalian genes involved in infection
US11219696B2 (en) * 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
WO2014070949A1 (en) * 2012-11-01 2014-05-08 California Institute Of Technology Reversible gene expression
JP2015092462A (ja) 2013-09-30 2015-05-14 Tdk株式会社 正極及びそれを用いたリチウムイオン二次電池
JP6202701B2 (ja) 2014-03-21 2017-09-27 株式会社日立国際電気 基板処理装置、半導体装置の製造方法及びプログラム
JP6197169B2 (ja) 2014-09-29 2017-09-20 東芝メモリ株式会社 半導体装置の製造方法
BR112019009834B1 (pt) * 2016-11-17 2022-05-17 Ohio State Innovation Foundation Vetor viral adeno-associado recombinante (raav), vírus, composição farmacêutica e uso de um vetor raav
JP2020518259A (ja) * 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ハンチントン病治療組成物および方法
TWI835747B (zh) * 2017-09-22 2024-03-21 賓州大學委員會 用於治療黏多醣病 ii 型之基因治療

Also Published As

Publication number Publication date
WO2021102435A1 (en) 2021-05-27
EP4061831A1 (en) 2022-09-28
JP2025124694A (ja) 2025-08-26
JP2023502474A (ja) 2023-01-24
JP7684296B2 (ja) 2025-05-27
IL293210A (en) 2022-07-01
US20230211018A1 (en) 2023-07-06
WO2021102435A8 (en) 2022-07-07

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Owner name: UNIVERSITA DEGLI STUDI DI MILANO

Free format text: FORMER NAME(S): RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL; FONDAZIONE IRCCS CA' GRANDA OSPEDALE MAGGIORE FOLICLINICO; UNIVERSTA DEGLI STUDI DI MILANO

Owner name: FONDAZIONE IRCCS CA' GRANDA OSPEDALE MAGGIORE FOLICLINICO

Free format text: FORMER NAME(S): RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL; FONDAZIONE IRCCS CA' GRANDA OSPEDALE MAGGIORE FOLICLINICO; UNIVERSTA DEGLI STUDI DI MILANO

Owner name: RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL

Free format text: FORMER NAME(S): RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL; FONDAZIONE IRCCS CA' GRANDA OSPEDALE MAGGIORE FOLICLINICO; UNIVERSTA DEGLI STUDI DI MILANO