WO2017062666A3 - Enzyme replacement therapy for mucopolysaccharidosis iiid - Google Patents

Enzyme replacement therapy for mucopolysaccharidosis iiid Download PDF

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Publication number
WO2017062666A3
WO2017062666A3 PCT/US2016/055822 US2016055822W WO2017062666A3 WO 2017062666 A3 WO2017062666 A3 WO 2017062666A3 US 2016055822 W US2016055822 W US 2016055822W WO 2017062666 A3 WO2017062666 A3 WO 2017062666A3
Authority
WO
WIPO (PCT)
Prior art keywords
iiid
sanfilippo
seq
replacement therapy
amino acid
Prior art date
Application number
PCT/US2016/055822
Other languages
French (fr)
Other versions
WO2017062666A9 (en
WO2017062666A2 (en
Inventor
Patricia Dickson
Tsui-Fen Chou
Sean Ekins
Shih-Hsin KAN
Steven LE
Derek R. MOEN
Original Assignee
Patricia Dickson
Tsui-Fen Chou
Sean Ekins
Kan Shih-Hsin
Le Steven
Moen Derek R
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 Patricia Dickson, Tsui-Fen Chou, Sean Ekins, Kan Shih-Hsin, Le Steven, Moen Derek R filed Critical Patricia Dickson
Publication of WO2017062666A2 publication Critical patent/WO2017062666A2/en
Publication of WO2017062666A3 publication Critical patent/WO2017062666A3/en
Publication of WO2017062666A9 publication Critical patent/WO2017062666A9/en
Priority to US15/946,505 priority Critical patent/US20180221459A1/en
Priority to US16/919,104 priority patent/US20210015906A1/en

Links

Classifications

    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
    • 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
    • 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)
    • 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)
    • C12Y301/06—Sulfuric ester hydrolases (3.1.6)
    • C12Y301/06014—N-Acetylglucosamine-6-sulfatase (3.1.6.14)

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Biotechnology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)

Abstract

The present disclosure relates to compositions and methods for treating Sanfilippo syndrome (also known as Sanfilippo disease type D, Sanfilippo D, mucopolysaccharidosis type IIID, MPS IIID). The method can entail injecting to the spinal fluid of a MPS IIID patient an effective amount of a composition comprising a recombinant human acetylglucosamine-6-sulfatase (GNS) protein comprising the amino acid sequence of SEQ ID NO: 1 or an amino acid sequence having at least 90% sequence identity to SEQ ID NO: 1 and having the enzymatic activity of the human GNS protein. The composition can be provided in an artificial cerebrospinal fluid.
PCT/US2016/055822 2015-10-06 2016-10-06 Enzyme replacement therapy for mucopolysaccharidosis iiid WO2017062666A2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
US15/946,505 US20180221459A1 (en) 2015-10-06 2018-04-05 Enzyme replacement therapy for mucopolysaccharidosis iiid
US16/919,104 US20210015906A1 (en) 2015-10-06 2020-07-01 Enzyme replacement therapy for mucopolysaccharidosis iiid

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562238024P 2015-10-06 2015-10-06
US62/238,024 2015-10-06

Related Child Applications (1)

Application Number Title Priority Date Filing Date
US15/946,505 Continuation-In-Part US20180221459A1 (en) 2015-10-06 2018-04-05 Enzyme replacement therapy for mucopolysaccharidosis iiid

Publications (3)

Publication Number Publication Date
WO2017062666A2 WO2017062666A2 (en) 2017-04-13
WO2017062666A3 true WO2017062666A3 (en) 2017-05-26
WO2017062666A9 WO2017062666A9 (en) 2017-06-22

Family

ID=58488484

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2016/055822 WO2017062666A2 (en) 2015-10-06 2016-10-06 Enzyme replacement therapy for mucopolysaccharidosis iiid

Country Status (1)

Country Link
WO (1) WO2017062666A2 (en)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20200058510A (en) 2017-10-02 2020-05-27 데날리 테라퓨틱스 인크. Fusion protein containing enzyme replacement therapy enzyme
EP3505181B1 (en) * 2017-12-28 2021-07-07 Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center Preparation of enzyme replacement therapy for mucopolysaccharidosis iiid

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20120003202A1 (en) * 2010-06-25 2012-01-05 Shire Human Genetic Therapies, Inc. Cns delivery of therapeutic agents

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20120003202A1 (en) * 2010-06-25 2012-01-05 Shire Human Genetic Therapies, Inc. Cns delivery of therapeutic agents

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
LUKARE MEDICAL: "Elliotts B(R) Solution: Clinical Data.", 14 August 2015 (2015-08-14), pages 1, Retrieved from the Internet <URL:http://http://www.elliottsbsolution.com/id2.html> [retrieved on 20170316] *
MOK, A ET AL.: "Genomic basis of mucopolysaccharidosis type IIID (MIM 252940) revealed by sequencing of GNS encoding N-acetylglucosamine-6-sulfatase.", GENOMICS, vol. 81, no. 1, January 2003 (2003-01-01), pages 1 - 5, XP026804384 *

Also Published As

Publication number Publication date
WO2017062666A9 (en) 2017-06-22
WO2017062666A2 (en) 2017-04-13

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