CA2370081C - Lpl variant therapeutics - Google Patents

Lpl variant therapeutics Download PDF

Info

Publication number
CA2370081C
CA2370081C CA2370081A CA2370081A CA2370081C CA 2370081 C CA2370081 C CA 2370081C CA 2370081 A CA2370081 A CA 2370081A CA 2370081 A CA2370081 A CA 2370081A CA 2370081 C CA2370081 C CA 2370081C
Authority
CA
Canada
Prior art keywords
lpl
therapeutic
nucleic acid
seq
sequence
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.)
Expired - Fee Related
Application number
CA2370081A
Other languages
English (en)
French (fr)
Other versions
CA2370081A1 (en
Inventor
Michael R. Hayden
John J. P. Kastelein
Katherine Julie Diane Ashbourne Excoffon
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.)
University of British Columbia
ACADEMIC HOSPITAL AT UNIVERSITY OF AMSTERDAM
Amsterdam Molecular Therapeutics AMT BV
Original Assignee
University of British Columbia
ACADEMIC HOSPITAL AT UNIVERSITY OF AMSTERDAM
Amsterdam Molecular Therapeutics AMT BV
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 University of British Columbia, ACADEMIC HOSPITAL AT UNIVERSITY OF AMSTERDAM, Amsterdam Molecular Therapeutics AMT BV filed Critical University of British Columbia
Publication of CA2370081A1 publication Critical patent/CA2370081A1/en
Priority to US12/898,684 priority Critical patent/US20110201673A1/en
Application granted granted Critical
Publication of CA2370081C publication Critical patent/CA2370081C/en
Anticipated expiration legal-status Critical
Expired - Fee Related legal-status Critical Current

Links

Classifications

    • 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)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/18—Carboxylic ester hydrolases (3.1.1)
    • C12N9/20—Triglyceride splitting, e.g. by means of lipase
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/04—Anorexiants; Antiobesity agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/12—Antihypertensives
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/01—Carboxylic ester hydrolases (3.1.1)
    • C12Y301/01034—Lipoprotein lipase (3.1.1.34)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Diabetes (AREA)
  • Microbiology (AREA)
  • Obesity (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Hematology (AREA)
  • Hospice & Palliative Care (AREA)
  • Child & Adolescent Psychology (AREA)
  • Urology & Nephrology (AREA)
  • Vascular Medicine (AREA)
  • Emergency Medicine (AREA)
  • Endocrinology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
CA2370081A 1999-06-24 2000-06-23 Lpl variant therapeutics Expired - Fee Related CA2370081C (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
US12/898,684 US20110201673A1 (en) 1999-06-24 2010-10-05 Lpl variant therapeutics

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP99202048 1999-06-24
EP99202048.7 1999-06-24
PCT/CA2000/000762 WO2001000220A2 (en) 1999-06-24 2000-06-23 Lipoprotein lipase (lpl) variant therapeutics

Publications (2)

Publication Number Publication Date
CA2370081A1 CA2370081A1 (en) 2001-01-04
CA2370081C true CA2370081C (en) 2014-06-03

Family

ID=8240356

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2370081A Expired - Fee Related CA2370081C (en) 1999-06-24 2000-06-23 Lpl variant therapeutics

Country Status (12)

Country Link
US (1) US20110201673A1 (https=)
EP (1) EP1200117B1 (https=)
JP (1) JP5095894B2 (https=)
AT (1) ATE404217T1 (https=)
AU (1) AU5799200A (https=)
CA (1) CA2370081C (https=)
CY (1) CY1108511T1 (https=)
DE (1) DE60039880D1 (https=)
DK (1) DK1200117T3 (https=)
ES (1) ES2311464T3 (https=)
PT (1) PT1200117E (https=)
WO (1) WO2001000220A2 (https=)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20070015518A (ko) * 2004-03-02 2007-02-05 셀렉타, 엘엘씨 암의 진단 및 치료를 위한 포스포리피드 유사체
SI1761273T1 (sl) * 2004-06-21 2008-10-31 Amsterdam Molecular Therapeutics Bv Zdravljenje nealkoholnega steatoznega hepatitisa
EP2311967B1 (en) 2005-10-20 2017-09-20 UniQure IP B.V. Improved AAV vectors produced in insect cells
KR101589259B1 (ko) 2006-06-21 2016-02-01 유니큐어 아이피 비.브이. 곤충세포 내 ａａｖ의 생산에 유용한 ａａｖ-ｒｅｐ78의 번역을 위한 변형된 개시 코돈을 갖는 벡터
SI3093345T1 (sl) 2007-07-26 2019-08-30 Uniqure Ip B.V. Bakulovirusni vektorji, ki vsebujejo ponavljajoča kodirna zaporedja z različnimi kodonskimi preferencami
WO2009030254A1 (en) * 2007-09-04 2009-03-12 Curevac Gmbh Complexes of rna and cationic peptides for transfection and for immunostimulation
EP2432497A1 (en) * 2009-05-18 2012-03-28 Amsterdam Molecular Therapeutics (AMT) IP B.V. Use of lipoprotein lipase (lpl) in therapy
SI3708192T1 (sl) 2009-06-12 2023-12-29 Cellectar, Inc. Eter fosfolipidne spojine za zdravljenje raka in slikanje in detekcijo rakavih izvornih celic
WO2011112089A2 (en) 2010-03-11 2011-09-15 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Mutated rep encoding sequences for use in aav production
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
CA2863964C (en) 2012-02-07 2021-10-26 Global Bio Therapeutics Usa, Inc. Compartmentalized method of nucleic acid delivery and compositions and uses thereof
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
JP2016538829A (ja) 2013-10-03 2016-12-15 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 低密度リポタンパク質受容体をコードするポリヌクレオチド
WO2015137802A1 (en) 2014-03-10 2015-09-17 Uniqure Ip B.V. Further improved aav vectors produced in insect cells
MA41035A (fr) 2014-08-19 2017-08-15 Shire Human Genetic Therapies Lipoprotéine lipase pour le traitement d'affections liées à une hypertriglycéridémie, y compris la pancréatite aiguë
EP3458106A4 (en) 2016-05-18 2020-03-18 Modernatx, Inc. POLYNUCLEOTIDS FOR CODING LIPOPROTEIN LIPASE FOR TREATING HYPERLIPIDEMIA
WO2018109207A1 (en) 2016-12-16 2018-06-21 Uniqure Ip B.V. Immunoadsorption
PT3652326T (pt) 2017-07-10 2024-12-26 Uniqure Ip Bv Meios e métodos para terapia génica de aav em humanos
JP7666922B2 (ja) 2017-07-20 2025-04-22 ユニキュアー アイピー ビー．ブイ． 昆虫細胞における向上したａａｖカプシド産生
CA3084452A1 (en) 2017-12-29 2019-07-04 Uniqure Ip B.V. Modified viral vectors and methods of making and using the same

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0755268A1 (en) * 1994-04-11 1997-01-29 Baylor College Of Medicine Compositions and methods for gene therapy to treat disease
US5658729A (en) * 1994-10-11 1997-08-19 The University Of British Columbia Method, reagent and kit for evaluating susceptibility to premature atherosclerosis

Also Published As

Publication number Publication date
EP1200117B1 (en) 2008-08-13
CA2370081A1 (en) 2001-01-04
JP5095894B2 (ja) 2012-12-12
WO2001000220A3 (en) 2001-07-12
WO2001000220A2 (en) 2001-01-04
DK1200117T3 (da) 2008-11-17
ES2311464T3 (es) 2009-02-16
PT1200117E (pt) 2008-11-26
CY1108511T1 (el) 2014-04-09
DE60039880D1 (en) 2008-09-25
JP2003503355A (ja) 2003-01-28
ATE404217T1 (de) 2008-08-15
US20110201673A1 (en) 2011-08-18
AU5799200A (en) 2001-01-31
EP1200117A2 (en) 2002-05-02

Similar Documents

Publication Publication Date Title
US20110201673A1 (en) Lpl variant therapeutics
Jimenez et al. FGF21 gene therapy as treatment for obesity and insulin resistance
US10894077B2 (en) Synthetic methylmalonyl-CoA mutase transgene for the treatment of MUT class methylmalonic acidemia (MMA)
KR20250044484A (ko) 표적 유전자 억제를 통해 저밀도 지단백질을 감소시키기 위한 조성물, 시스템, 및 방법
US9944918B2 (en) Synthetic methylmalonyl-CoA mutase transgene for the treatment of MUT class methylmalonic acidemia (MMA)
WO2018060097A1 (en) Adenoassociated virus vectors for the treatment of mucopolysaccharidoses
WO2008100789A2 (en) Compositions and methods for altering elastogenesis
WO2012001170A1 (en) Telomerase reverse transcriptase for protection against ageing
WO2010135714A2 (en) Methods for modulating adipocyte expression using microrna compositions
JP2021526023A (ja) ムコ多糖症ｉｖａ型治療用のアデノ随伴ウイルスベクター
US9290751B2 (en) LPL variant therapeutics
WO2019000093A1 (en) PLATINUM TALE AND USE THEREOF TO INCREASE THE EXPRESSION OF FRATAXIN
WO2020176732A1 (en) TREATMENT OF PULMONARY FIBROSIS WITH SERCA2a GENE THERAPY
EP1761273B1 (en) Treatment of non alcoholic steatotic hepatitis (nash)
JP2003530835A (ja) 高トリグリセリド血症を誘発することなくコレステロールレベルを低下するための化合物と方法
US10835620B2 (en) Methods for treating heart failure using beta-ARKnt peptide
US20260002151A1 (en) Compositions and methods for the modification and regulation of liver gene expression
US20260109963A1 (en) Compositions, systems, and methods for reducing adipose tissue
CN102355911A (zh) 调节脂肪生成的Vgll3活性调节剂的用途
US7309606B2 (en) Methods and compositions for treating hypercholesterolemia
JPH10500859A (ja) 組換えウイルス、製造方法および遺伝子治療での使用
WO2007044912A2 (en) Non-native constitutively active osteopontin
MXPA99004301A (es) Adenovirus recombinantes bicistronicos para el tratamiento de patologias relacionadas a las dislipoproteinemias
HK1129842A (en) Diagnosis and treatment of multiple sulfatase deficiency and other using a formylglycine generating enzyme(fge)
HK1090088B (en) Diagnosis and treatment of multiple sulfatase deficiency and other using a formylglycine generating enzyme(fge)

Legal Events

Date Code Title Description
EEER Examination request
MKLA Lapsed

Effective date: 20180626