CA3078464A1 - Gene therapies for lysosomal disorders - Google Patents

Gene therapies for lysosomal disorders Download PDF

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Publication number
CA3078464A1
CA3078464A1 CA3078464A CA3078464A CA3078464A1 CA 3078464 A1 CA3078464 A1 CA 3078464A1 CA 3078464 A CA3078464 A CA 3078464A CA 3078464 A CA3078464 A CA 3078464A CA 3078464 A1 CA3078464 A1 CA 3078464A1
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Canada
Prior art keywords
nucleic acid
isolated nucleic
sequence
seq
protein
Prior art date
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Pending
Application number
CA3078464A
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English (en)
French (fr)
Inventor
Asa Abeliovich
Laura Heckman
Herve Rhinn
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.)
Prevail Therapeutics Inc
Original Assignee
Prevail Therapeutics Inc
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Filing date
Publication date
Application filed by Prevail Therapeutics Inc filed Critical Prevail Therapeutics Inc
Publication of CA3078464A1 publication Critical patent/CA3078464A1/en
Pending legal-status Critical Current

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    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
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    • 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
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    • 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
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    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
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    • A01K2267/0306—Animal model for genetic diseases
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    • 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
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    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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CA3078464A 2017-10-03 2018-10-03 Gene therapies for lysosomal disorders Pending CA3078464A1 (en)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201762567296P 2017-10-03 2017-10-03
US201762567301P 2017-10-03 2017-10-03
US201762567319P 2017-10-03 2017-10-03
US201762567310P 2017-10-03 2017-10-03
US201762567311P 2017-10-03 2017-10-03
US62/567,319 2017-10-03
US62/567,310 2017-10-03
US62/567,311 2017-10-03
US62/567,301 2017-10-03
US62/567,296 2017-10-03
PCT/US2018/054227 WO2019070894A1 (en) 2017-10-03 2018-10-03 GENE THERAPIES FOR LYSOSOMIAL DISORDERS

Publications (1)

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CA3078464A1 true CA3078464A1 (en) 2019-04-11

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CA3078464A Pending CA3078464A1 (en) 2017-10-03 2018-10-03 Gene therapies for lysosomal disorders

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