MX2023006445A - Composiciones y usos de estas para el tratamiento del síndrome de angelman. - Google Patents

Composiciones y usos de estas para el tratamiento del síndrome de angelman.

Info

Publication number
MX2023006445A
MX2023006445A MX2023006445A MX2023006445A MX2023006445A MX 2023006445 A MX2023006445 A MX 2023006445A MX 2023006445 A MX2023006445 A MX 2023006445A MX 2023006445 A MX2023006445 A MX 2023006445A MX 2023006445 A MX2023006445 A MX 2023006445A
Authority
MX
Mexico
Prior art keywords
angelman syndrome
compositions
treatment
ube3a
raav
Prior art date
Application number
MX2023006445A
Other languages
English (en)
Inventor
James M Wilson
Ralf Schmid
Justin Percival
Original Assignee
Univ Pennsylvania
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 Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of MX2023006445A publication Critical patent/MX2023006445A/es

Links

Classifications

    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • 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
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • 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
    • C12N9/93—Ligases (6)
    • 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)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)

Abstract

Se proporciona un rAAV que tiene un genoma de vector con una secuencia que codifica UBE3A. También se proporciona un método para tratar uno o más síntomas del síndrome de Angelman (AS) en un paciente que tiene expresión de UBE3A deficiente en neuronas, en donde el método comprende administrar una rAAV que tiene una secuencia de ácidos nucleicos que codifica UBE3A.
MX2023006445A 2020-12-01 2021-12-01 Composiciones y usos de estas para el tratamiento del síndrome de angelman. MX2023006445A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063119860P 2020-12-01 2020-12-01
US202163179807P 2021-04-26 2021-04-26
PCT/US2021/061346 WO2022119890A1 (en) 2020-12-01 2021-12-01 Compositions and uses thereof for treatment of angelman syndrome

Publications (1)

Publication Number Publication Date
MX2023006445A true MX2023006445A (es) 2023-08-10

Family

ID=80123326

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2023006445A MX2023006445A (es) 2020-12-01 2021-12-01 Composiciones y usos de estas para el tratamiento del síndrome de angelman.

Country Status (9)

Country Link
US (1) US20230414785A1 (es)
EP (1) EP4255500A1 (es)
JP (1) JP2023551911A (es)
KR (1) KR20230128001A (es)
AU (1) AU2021392642A1 (es)
CA (1) CA3200192A1 (es)
IL (1) IL303239A (es)
MX (1) MX2023006445A (es)
WO (1) WO2022119890A1 (es)

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE520707T1 (de) 2001-11-13 2011-09-15 Univ Pennsylvania Verfahren für den nachweis und/oder die identifikation von sequenzen des adeno- assoziierten virus (aav) sowie isolation von dadurch identifizierten, neuen sequenzen
EP2359869B1 (en) 2001-12-17 2018-12-26 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same and uses therefor
EP2298926A1 (en) 2003-09-30 2011-03-23 The Trustees of The University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
EP3409296A1 (en) 2005-04-07 2018-12-05 The Trustees of the University of Pennsylvania Method of increasing the function of an aav vector
WO2007120542A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid library and aav capsid proteins
CN104910025B (zh) 2008-11-07 2019-07-16 麻省理工学院 氨基醇类脂质和其用途
SG10201908848RA (en) 2010-03-29 2019-10-30 Univ Pennsylvania Pharmacologically induced transgene ablation system
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
EP2643468B1 (en) 2010-11-22 2018-07-04 Amicus Therapeutics, Inc. Novel signal sequences to improve protein expressions and secretion of recombinant enzymes and other proteins
KR102128248B1 (ko) 2011-06-08 2020-07-01 샤이어 휴먼 지네틱 테라피즈 인크. Mrna 전달을 위한 지질 나노입자 조성물 및 방법
FR2977562B1 (fr) 2011-07-06 2016-12-23 Gaztransport Et Technigaz Cuve etanche et thermiquement isolante integree dans une structure porteuse
DK2858677T3 (da) 2012-06-08 2020-08-31 Ethris Gmbh Pulmonær levering af messenger rna
US9567369B2 (en) 2012-08-03 2017-02-14 A.T. Still University Method of treating metastatic cancer
ES2739288T3 (es) 2013-09-13 2020-01-30 California Inst Of Techn Recuperación selectiva
ES2745335T3 (es) 2014-02-28 2020-02-28 Univ Bologna Alma Mater Studiorum Proteínas de fusión TATk-CDKL5, composiciones, formulaciones y uso de estas
WO2017160360A2 (en) 2015-12-11 2017-09-21 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
US11015173B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV1
LT3411484T (lt) * 2016-02-05 2023-11-27 Emory University Viengrandžio arba sau pačiam komplementaraus adeno-asocijuoto viruso 9 injekcija į smegenų skystį
AU2017290047A1 (en) 2016-06-28 2019-01-17 Alma Mater Studiorum - Universitá Di Bologna TATĸ-CDKL5 fusion proteins, compositions, formulations, and use thereof
SG11201907714UA (en) 2017-02-28 2019-09-27 Univ Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor
CN110869031A (zh) 2017-06-28 2020-03-06 南佛罗里达大学 用于天使人综合征的基因治疗方法的修饰的ube3a基因
MX2020005670A (es) 2017-11-30 2020-11-24 Amicus Therapeutics Inc Variantes de expresión de cdkl5 y proteínas de fusión de cdkl5.
AU2019261982A1 (en) 2018-04-30 2020-10-15 Amicus Therapeutics, Inc. Gene therapy constructs and methods of use
CN112739353A (zh) * 2018-06-14 2021-04-30 奥维德医疗公司 Mir-92a或mir-145在治疗安格曼综合征中的用途
AU2019401314A1 (en) 2018-12-21 2021-06-24 The Trustees Of The University Of Pennsylvania Compositions for DRG-specific reduction of transgene expression
MX2021013266A (es) * 2019-04-29 2021-11-17 Univ Pennsylvania Nuevas capsides de aav y composiciones que las contienen.
CN114127296A (zh) * 2019-05-22 2022-03-01 北卡罗来纳大学查佩尔希尔分校 Ube3a基因和表达盒及其应用
IT201900008877A1 (it) 2019-06-13 2020-12-13 Univ Bologna Alma Mater Studiorum Nuovi costrutti per terapia genica
JP2022552254A (ja) 2019-10-10 2022-12-15 アミカス セラピューティックス インコーポレイテッド バリアントｉｇｆ２構築体
CN114901802A (zh) 2019-10-30 2022-08-12 阿米库斯治疗学公司 重组cdkl5蛋白、基因疗法和生产方法

Also Published As

Publication number Publication date
AU2021392642A1 (en) 2023-06-22
EP4255500A1 (en) 2023-10-11
CA3200192A1 (en) 2022-06-09
KR20230128001A (ko) 2023-09-01
JP2023551911A (ja) 2023-12-13
US20230414785A1 (en) 2023-12-28
WO2022119890A1 (en) 2022-06-09
IL303239A (en) 2023-07-01
WO2022119890A9 (en) 2023-07-27

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