STABLE FORMULATIONS FOR CNS DELIVERY OF ARYLSULFATASE A
The present invention provides, among oilier things, compositions and methods for CNS delivery of lysosomal enzymes (e.g., recombinant human arylsulfatase A (rhASA)) for effective treatment of lysosomal storage diseases (e.g.. Metachromatic Leukodystrophy Disease), In some embodiments, the presen t...
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| creator | SHAHROKH, ZAHRA CAMPOLIETO, PAUL PAN, JING TAYLOR, KATHERINE BASU, SUJIT WRIGHT, TERESA, LEAH SALAMAT-MILLER, NAZILA CALIAS, PERICLES CHARNAS, LAWRENCE JAIN, KEETHKUMAR |
| description | The present invention provides, among oilier things, compositions and methods for CNS delivery of lysosomal enzymes (e.g., recombinant human arylsulfatase A (rhASA)) for effective treatment of lysosomal storage diseases (e.g.. Metachromatic Leukodystrophy Disease), In some embodiments, the presen t invention includes a stable formulation for intrathecal administration comprising an ASA protein and a poloxamer, wherein less than 3% of the ASA protein exists in aggregated form.
La présente invention concerne, entre autres choses, des compositions et des procédés pour l'administration au système nerveux central d'enzymes lysosomales (par exemple, arylsulfatase A humaine recombinante (rhASA)) pour un traitement efficace de maladies de stockage lysosomal (par exemple, maladie de leucodystrophie métachromatique). Dans certains modes de réalisation, la présente invention comprend une formulation stable pour une administration intrathécale comprenant une protéine ASA et un poloxamère, où moins de 3 % de la protéine ASA existe sous forme agrégée. |
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La présente invention concerne, entre autres choses, des compositions et des procédés pour l'administration au système nerveux central d'enzymes lysosomales (par exemple, arylsulfatase A humaine recombinante (rhASA)) pour un traitement efficace de maladies de stockage lysosomal (par exemple, maladie de leucodystrophie métachromatique). Dans certains modes de réalisation, la présente invention comprend une formulation stable pour une administration intrathécale comprenant une protéine ASA et un poloxamère, où moins de 3 % de la protéine ASA existe sous forme agrégée.</description><language>eng ; fre</language><subject>HUMAN NECESSITIES ; HYGIENE ; MEDICAL OR VETERINARY SCIENCE ; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES</subject><creationdate>2013</creationdate><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktohtml>$$Uhttps://worldwide.espacenet.com/publicationDetails/biblio?FT=D&date=20130627&DB=EPODOC&CC=WO&NR=2013096899A2$$EHTML$$P50$$Gepo$$Hfree_for_read</linktohtml><link.rule.ids>230,308,776,881,25542,76289</link.rule.ids><linktorsrc>$$Uhttps://worldwide.espacenet.com/publicationDetails/biblio?FT=D&date=20130627&DB=EPODOC&CC=WO&NR=2013096899A2$$EView_record_in_European_Patent_Office$$FView_record_in_$$GEuropean_Patent_Office$$Hfree_for_read</linktorsrc></links><search><creatorcontrib>SHAHROKH, ZAHRA</creatorcontrib><creatorcontrib>CAMPOLIETO, PAUL</creatorcontrib><creatorcontrib>PAN, JING</creatorcontrib><creatorcontrib>TAYLOR, KATHERINE</creatorcontrib><creatorcontrib>BASU, SUJIT</creatorcontrib><creatorcontrib>WRIGHT, TERESA, LEAH</creatorcontrib><creatorcontrib>SALAMAT-MILLER, NAZILA</creatorcontrib><creatorcontrib>CALIAS, PERICLES</creatorcontrib><creatorcontrib>CHARNAS, LAWRENCE</creatorcontrib><creatorcontrib>JAIN, KEETHKUMAR</creatorcontrib><title>STABLE FORMULATIONS FOR CNS DELIVERY OF ARYLSULFATASE A</title><description>The present invention provides, among oilier things, compositions and methods for CNS delivery of lysosomal enzymes (e.g., recombinant human arylsulfatase A (rhASA)) for effective treatment of lysosomal storage diseases (e.g.. Metachromatic Leukodystrophy Disease), In some embodiments, the presen t invention includes a stable formulation for intrathecal administration comprising an ASA protein and a poloxamer, wherein less than 3% of the ASA protein exists in aggregated form.
La présente invention concerne, entre autres choses, des compositions et des procédés pour l'administration au système nerveux central d'enzymes lysosomales (par exemple, arylsulfatase A humaine recombinante (rhASA)) pour un traitement efficace de maladies de stockage lysosomal (par exemple, maladie de leucodystrophie métachromatique). Dans certains modes de réalisation, la présente invention comprend une formulation stable pour une administration intrathécale comprenant une protéine ASA et un poloxamère, où moins de 3 % de la protéine ASA existe sous forme agrégée.</description><subject>HUMAN NECESSITIES</subject><subject>HYGIENE</subject><subject>MEDICAL OR VETERINARY SCIENCE</subject><subject>PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES</subject><fulltext>true</fulltext><rsrctype>patent</rsrctype><creationdate>2013</creationdate><recordtype>patent</recordtype><sourceid>EVB</sourceid><recordid>eNrjZDAPDnF08nFVcPMP8g31cQzx9PcLBnEUnIG0i6uPZ5hrUKSCv5uCY1CkT3Coj5tjiGOwq4IjDwNrWmJOcSovlOZmUHZzDXH20E0tyI9PLS5ITE7NSy2JD_c3MjA0NrA0s7C0dDQyJk4VAOv2KVE</recordid><startdate>20130627</startdate><enddate>20130627</enddate><creator>SHAHROKH, ZAHRA</creator><creator>CAMPOLIETO, PAUL</creator><creator>PAN, JING</creator><creator>TAYLOR, KATHERINE</creator><creator>BASU, SUJIT</creator><creator>WRIGHT, TERESA, LEAH</creator><creator>SALAMAT-MILLER, NAZILA</creator><creator>CALIAS, PERICLES</creator><creator>CHARNAS, LAWRENCE</creator><creator>JAIN, KEETHKUMAR</creator><scope>EVB</scope></search><sort><creationdate>20130627</creationdate><title>STABLE FORMULATIONS FOR CNS DELIVERY OF ARYLSULFATASE A</title><author>SHAHROKH, ZAHRA ; CAMPOLIETO, PAUL ; PAN, JING ; TAYLOR, KATHERINE ; BASU, SUJIT ; WRIGHT, TERESA, LEAH ; SALAMAT-MILLER, NAZILA ; CALIAS, PERICLES ; CHARNAS, LAWRENCE ; JAIN, KEETHKUMAR</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-epo_espacenet_WO2013096899A23</frbrgroupid><rsrctype>patents</rsrctype><prefilter>patents</prefilter><language>eng ; fre</language><creationdate>2013</creationdate><topic>HUMAN NECESSITIES</topic><topic>HYGIENE</topic><topic>MEDICAL OR VETERINARY SCIENCE</topic><topic>PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES</topic><toplevel>online_resources</toplevel><creatorcontrib>SHAHROKH, ZAHRA</creatorcontrib><creatorcontrib>CAMPOLIETO, PAUL</creatorcontrib><creatorcontrib>PAN, JING</creatorcontrib><creatorcontrib>TAYLOR, KATHERINE</creatorcontrib><creatorcontrib>BASU, SUJIT</creatorcontrib><creatorcontrib>WRIGHT, TERESA, LEAH</creatorcontrib><creatorcontrib>SALAMAT-MILLER, NAZILA</creatorcontrib><creatorcontrib>CALIAS, PERICLES</creatorcontrib><creatorcontrib>CHARNAS, LAWRENCE</creatorcontrib><creatorcontrib>JAIN, KEETHKUMAR</creatorcontrib><collection>esp@cenet</collection></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext_linktorsrc</fulltext></delivery><addata><au>SHAHROKH, ZAHRA</au><au>CAMPOLIETO, PAUL</au><au>PAN, JING</au><au>TAYLOR, KATHERINE</au><au>BASU, SUJIT</au><au>WRIGHT, TERESA, LEAH</au><au>SALAMAT-MILLER, NAZILA</au><au>CALIAS, PERICLES</au><au>CHARNAS, LAWRENCE</au><au>JAIN, KEETHKUMAR</au><format>patent</format><genre>patent</genre><ristype>GEN</ristype><title>STABLE FORMULATIONS FOR CNS DELIVERY OF ARYLSULFATASE A</title><date>2013-06-27</date><risdate>2013</risdate><abstract>The present invention provides, among oilier things, compositions and methods for CNS delivery of lysosomal enzymes (e.g., recombinant human arylsulfatase A (rhASA)) for effective treatment of lysosomal storage diseases (e.g.. Metachromatic Leukodystrophy Disease), In some embodiments, the presen t invention includes a stable formulation for intrathecal administration comprising an ASA protein and a poloxamer, wherein less than 3% of the ASA protein exists in aggregated form.
La présente invention concerne, entre autres choses, des compositions et des procédés pour l'administration au système nerveux central d'enzymes lysosomales (par exemple, arylsulfatase A humaine recombinante (rhASA)) pour un traitement efficace de maladies de stockage lysosomal (par exemple, maladie de leucodystrophie métachromatique). Dans certains modes de réalisation, la présente invention comprend une formulation stable pour une administration intrathécale comprenant une protéine ASA et un poloxamère, où moins de 3 % de la protéine ASA existe sous forme agrégée.</abstract><oa>free_for_read</oa></addata></record> |
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| subjects | HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES |
| title | STABLE FORMULATIONS FOR CNS DELIVERY OF ARYLSULFATASE A |
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