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MX2024007890A - Metodos y composiciones para tratar el sindrome de barth. - Google Patents

Metodos y composiciones para tratar el sindrome de barth.

Info

Publication number
MX2024007890A
MX2024007890A MX2024007890A MX2024007890A MX2024007890A MX 2024007890 A MX2024007890 A MX 2024007890A MX 2024007890 A MX2024007890 A MX 2024007890A MX 2024007890 A MX2024007890 A MX 2024007890A MX 2024007890 A MX2024007890 A MX 2024007890A
Authority
MX
Mexico
Prior art keywords
compositions
methods
barth syndrome
treating
treating barth
Prior art date
Application number
MX2024007890A
Other languages
English (en)
Inventor
Francisco Leon
Paul Dunford
Diana Miller
Barry John Byrne
Manuela Corti
Original Assignee
Provention Bio 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 Provention Bio Inc filed Critical Provention Bio Inc
Publication of MX2024007890A publication Critical patent/MX2024007890A/es

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    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/4353—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/436—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a six-membered ring having oxygen as a ring hetero atom, e.g. rapamycin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39533—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
    • A61K39/39541—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against normal tissues, cells
    • 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
    • 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
    • A61K48/0083—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 administration regime
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
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    • 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
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    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
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    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2803—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the immunoglobulin superfamily
    • C—CHEMISTRY; METALLURGY
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    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2803—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the immunoglobulin superfamily
    • C07K16/283—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the immunoglobulin superfamily against Fc-receptors, e.g. CD16, CD32, CD64
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    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
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    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2887—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against CD20
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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    • 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
    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2896—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against molecules with a "CD"-designation, not provided for elsewhere
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    • 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
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    • C12N9/10—Transferases (2.)
    • C12N9/1025—Acyltransferases (2.3)
    • C12N9/1029—Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/545—Medicinal preparations containing antigens or antibodies characterised by the dose, timing or administration schedule
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/30—Immunoglobulins specific features characterized by aspects of specificity or valency
    • C07K2317/31—Immunoglobulins specific features characterized by aspects of specificity or valency multispecific
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    • C07K2317/56—Immunoglobulins specific features characterized by immunoglobulin fragments variable (Fv) region, i.e. VH and/or VL
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    • C07—ORGANIC CHEMISTRY
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    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/60—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
    • C07K2317/62—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
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    • C07—ORGANIC CHEMISTRY
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    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/70—Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12Y—ENZYMES
    • C12Y203/00—Acyltransferases (2.3)
    • C12Y203/01—Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
    • C12Y203/01023—1-Acylglycerophosphocholine O-acyltransferase (2.3.1.23), i.e. lysophosphatidylcholine acyltransferase or LPCAT

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  • Health & Medical Sciences (AREA)
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  • Organic Chemistry (AREA)
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  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
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Abstract

En el presente documento, en un aspecto, se desvela un método para reducir la inmunogenicidad, que comprende administrar a un paciente que recibe o ha recibido una terapia génica para el BTHS una cantidad eficaz de inhibidor de linfocitos B no depletivo. También se proporcionan composiciones relacionadas.
MX2024007890A 2021-12-23 2022-12-23 Metodos y composiciones para tratar el sindrome de barth. MX2024007890A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163293514P 2021-12-23 2021-12-23
PCT/US2022/082367 WO2023122798A2 (en) 2021-12-23 2022-12-23 Methods and compositions for treating barth syndrome

Publications (1)

Publication Number Publication Date
MX2024007890A true MX2024007890A (es) 2024-07-10

Family

ID=86903821

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2024007890A MX2024007890A (es) 2021-12-23 2022-12-23 Metodos y composiciones para tratar el sindrome de barth.

Country Status (10)

Country Link
US (1) US20250304696A1 (es)
EP (1) EP4453040A4 (es)
JP (1) JP2025500413A (es)
KR (1) KR20240125973A (es)
CN (1) CN119137158A (es)
AU (1) AU2022419656A1 (es)
CA (1) CA3242085A1 (es)
IL (1) IL313717A (es)
MX (1) MX2024007890A (es)
WO (1) WO2023122798A2 (es)

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN106349390B (zh) * 2008-04-02 2019-12-10 宏观基因有限公司 Bcr-复合体-特异性抗体和其使用方法
UA116479C2 (uk) * 2013-08-09 2018-03-26 Макродженікс, Інк. БІСПЕЦИФІЧНЕ МОНОВАЛЕНТНЕ Fc-ДІАТІЛО, ЯКЕ ОДНОЧАСНО ЗВ'ЯЗУЄ CD32B I CD79b, ТА ЙОГО ЗАСТОСУВАННЯ
SG11202111616RA (en) * 2019-04-26 2021-11-29 Sangamo Therapeutics Inc Engineering aav
US20210130464A1 (en) * 2019-07-30 2021-05-06 Provention Bio, Inc. Methods and Compositions for Reducing Immunogenicity By Non-Depletional B Cell Inhibitors
BR112022001699A2 (pt) * 2019-07-30 2022-05-24 Provention Bio Inc Métodos e composições para reduzir imunogenicidade através de inibidores não deplecionais de célula b
WO2021163499A1 (en) * 2020-02-14 2021-08-19 Children's Medical Center Corporation Taz gene or enzyme replacement therapy

Also Published As

Publication number Publication date
JP2025500413A (ja) 2025-01-09
EP4453040A4 (en) 2025-12-24
CN119137158A (zh) 2024-12-13
KR20240125973A (ko) 2024-08-20
AU2022419656A1 (en) 2024-08-08
WO2023122798A2 (en) 2023-06-29
IL313717A (en) 2024-08-01
US20250304696A1 (en) 2025-10-02
EP4453040A2 (en) 2024-10-30
CA3242085A1 (en) 2023-06-29
WO2023122798A3 (en) 2023-09-28

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