| 序号 | 专利名 | 申请号 | 申请日 | 公开（公告）号 | 公开（公告）日 | 发明人 |
|---|---|---|---|---|---|---|
| 1 | 乙酰肝素N-硫酸酯酶CNS递送的方法和组合物 | CN201180040902.2 | 2011-06-25 | CN103260637B | 2016-04-06 | F·纳托里; G·朱; J·特略; Y·江; J·宗; Z·夏若克; B·维纳格利亚; J·潘; R·菲佛; P·加莱斯 |
| 本发明提供了，除了其它的以外，CNS递送溶酶体酶的组合物和方法，以有效治疗溶酶体贮积症。在一些实施方案中，本发明包括直接CNS鞘内施用的稳定制剂，所述稳定制剂包括乙酰肝素N-硫酸酯酶(HNS)蛋白质、盐、和聚山梨醇酯表面活性剂，以治疗圣菲利波(Sanfilippo)综合症类型A。 | ||||||
| 2 | 乙酰肝素N-硫酸酯酶CNS递送的方法和组合物 | CN201180040902.2 | 2011-06-25 | CN103260637A | 2013-08-21 | F·纳托里; G·朱; J·特略; Y·江; J·宗; Z·夏若克; B·维纳格利亚; J·潘; R·菲佛; P·加莱斯 |
| 本发明提供了，除了其它的以外，CNS递送溶酶体酶的组合物和方法，以有效治疗溶酶体贮积症。在一些实施方案中，本发明包括直接CNS鞘内施用的稳定制剂，所述稳定制剂包括乙酰肝素N-硫酸酯酶(HNS)蛋白质、盐、和聚山梨醇酯表面活性剂，以治疗圣菲利波(Sanfilippo)综合症类型A。 | ||||||
| 3 | 修飾リソソームタンパク質とその製造 | JP2018515835 | 2016-09-30 | JP2018535654A | 2018-12-06 | エーリク ノルドリン; パートリック ストレームバリ; ステファン スベンソン ゲリウス |
| 修飾リソソームタンパク質、修飾リソソームタンパク質を調製する方法、および斯かる修飾タンパク質の治療用途を本明細書中に開示する。リソソーム蓄積症に罹患している哺乳動物を治療する方法をさらに本明細書中に開示する。特に、本開示は、修飾リソソームタンパク質を調製する方法であって、グリコシル化リソソームタンパク質を過ヨウ素酸アルカリ金属塩と反応させ、そして、前記リソソームタンパク質を水素化ホウ素アルカリ金属塩と2時間以下反応させること、を含み、それによって、前記リソソームタンパク質のグリカン部分を修飾し、グリカン認識受容体に関して前記リソソームタンパク質の活性を低下させる前記方法に関する。 | ||||||
| 4 | アリールスルファターゼAのCNS送達の方法および組成物 | JP2015246952 | 2015-12-18 | JP6346162B2 | 2018-06-20 | ナジラ サラマット−ミラー; キャサリン テイラー; ポール キャンポリート; ザーラ シャーロク; ジン パン; ローレンス チャーナス; テレサ リア ライト; ペリクルス カリアス |
| 5 | ＣＮＳにおける酵素活性を増大するための方法および組成物 | JP2016529759 | 2014-05-19 | JP2016525545A | 2016-08-25 | ウィリアム・エム・パードリッジ; ルーベン・ジェイ・ボアド |
| 本明細書は、中枢神経系(CNS)における酵素欠損症に罹患した対象を治療するための方法および組成物を提供する。本明細書の二機能性の融合抗体は、内因性血液脳関門(BBB)受容体に対する抗体、および、ムコ多糖症III型(MPS−III)で欠損している酵素を含む。本明細書の融合抗体は、N−スルフォグルコサミンスルフォヒドロラーゼ(SGSH)、α−N−アセチルグルコサミニダーゼ(NAGLU)、ヘパリン−α−グルコサミニドN−アセチルトランスフェラーゼ(HGSNAT)、またはN−アセチルグルコサミン−6−スルファターゼ(GNS)を含む。CNSにおける酵素欠損症の治療方法は、本明細書の融合抗体の全身投与を含む。 | ||||||
| 6 | Anti-Human Transferrin Receptor Antibody Permeating Blood-Brain Barrier | US15739200 | 2016-06-24 | US20180171012A1 | 2018-06-21 | Hiroyuki Sonoda; Kenichi Takahashi |
| Disclosed are a means to convert compounds having physiological or pharmacological activity and unable to pass through the blood-brain barrier into a form that allows them to pass through the blood-brain barrier, and compounds converted thereby. The means is an anti-human transferrin receptor antibody and the converted compounds are molecular conjugates between physiologically active protein or pharmacologically active low-molecular-weight compounds and an anti-human transferrin receptor antibody. | ||||||
| 7 | Modified sulfamidase and production thereof | US14806504 | 2015-07-22 | US09982243B2 | 2018-05-29 | Charlotta Berghard; Erik Nordling; Stefan Svensson Gelius; Agneta Tjernberg |
| Disclosed herein are a modified sulfamidase, a composition comprising a modified sulfamidase, as well as methods for preparing a modified sulfamidase and therapeutic use of such a sulfamidase. In particular, the present disclosure relates to a modified sulfamidase comprising substantially no epitopes for glycan recognition receptors, thereby enabling transportation of said sulfamidase across the blood brain barrier of a mammal, wherein said sulfamidase has catalytic activity in the brain of said mammal. | ||||||
| 8 | MODIFIED SULFAMIDASE AND PRODUCTION THEREOF | US14806504 | 2015-07-22 | US20160230155A1 | 2016-08-11 | Charlotta BERGHARD; Erik NORDLING; Stefan Svensson GELIUS; Agneta TJERNBERG |
| Disclosed herein are a modified sulfamidase, a composition comprising a modified sulfamidase, as well as methods for preparing a modified sulfamidase and therapeutic use of such a sulfamidase. In particular, the present disclosure relates to a modified sulfamidase comprising substantially no epitopes for glycan recognition receptors, thereby enabling transportation of said sulfamidase across the blood brain barrier of a mammal, wherein said sulfamidase has catalytic activity in the brain of said mammal. | ||||||
| 9 | METHODS AND COMPOSITIONS FOR CNS DELIVERY OF IDURONATE-2-SULFATASE | US14938824 | 2015-11-11 | US20160158324A1 | 2016-06-09 | Gaozhong Zhu; Kris Lowe; Zahra Shahrokh; James Christian; Richard Fahrner; Jing Pan; Teresa Leah Wright; Pericles Calias |
| The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising an iduronate-2-sulfatase (I2S) protein, salt, and a polysorbate surfactant for the treatment of Hunters Syndrome. | ||||||
| 10 | THERAPEUTIC STRATEGIES TO TREAT CNS PATHOLOGY IN MUCOPOLYSACCHARIDOSES | US14939700 | 2015-11-12 | US20160122731A1 | 2016-05-05 | Andrea BALLABIO; Alessandro FRALDI |
| The invention provides for nucleotide sequences encoding for a chimeric sulfatase, viral vectors expressing such sequences for gene therapy and pharmaceutical uses of the chimeric expressed protein. The invention is particularly applied in the therapy of mucopolysaccharidosis, preferably type IIIA. | ||||||
| 11 | Methods and compositions for CNS delivery of iduronate-2-sulfatase | US13974457 | 2013-08-23 | US09220677B2 | 2015-12-29 | Gaozhong Zhu; Kris Lowe; Zahra Shahrokh; James Christian; Rick Fahrner; Jing Pan; Teresa Leah Wright; Pericles Calias |
| The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising an iduronate-2-sulfatase (I2S) protein, salt, and a polysorbate surfactant for the treatment of Hunters Syndrome. | ||||||
| 12 | PLANT LECTINS AS CARRIERS OF ASSOCIATED DRUG SUBSTANCES INTO ANIMAL AND HUMAN CELLS | US13906203 | 2013-05-30 | US20130323221A1 | 2013-12-05 | DAVID N. RADIN |
| The current invention involves the use of protein lectins produced by plants including the non-toxic carbohydrate binding subunits (B subunits) of plant “AB toxins” (PTB lectins) as delivery vehicles for mobilizing associated drug substances for delivery to animal and human cells. The resulting protein fusions or conjugates retain lectin carbohydrate specificity for binding to cells and cellular trafficking activity so as to deliver an associated drug compound to the site of disease manifestation. One embodiment of this invention concerns the ability of ricin toxin B subunit, as a model PTB lectin, to deliver enzyme replacement therapeutic drugs to cells of several organs of the body including the brain and central nervous system, eyes, ears, lungs, bone, heart, kidney, liver, and spleen for treating lysosomal diseases. | ||||||
| 13 | PRODUCTS AND METHODS FOR DELIVERY OF POLYNUCLEOTIDES BY ADENO-ASSOCIATED VIRUS FOR LYSOSOMAL STORAGE DISORDERS | US13922915 | 2013-06-20 | US20130323207A1 | 2013-12-05 | Douglas M. McCarty; Haiyan Fu |
| The present invention relates to methods and materials useful for systemically delivering polynucleotides across the blood brain barrier using adeno-associated virus as a vector. For example, the present invention relates to methods and materials useful for systemically delivering α-N-acetylglucosamidinase polynucleotides to the central and peripheral nervous systems, as well as the somatic system. Use of these methods and materials is indicated, for example, for treatment of the lysosomal storage disorder mucopolysaccharidosis IIIB. As another example, the present invention relates to methods and materials useful for systemically delivering N-sulphoglucosamine sulfphohydrolase polynucleotides to the central and peripheral nervous systems, as well as the somatic system. Use of this second type of methods and materials is indicated, for example, for treatment of the lysosomal storage disorder mucopolysaccharidosis IIIA. | ||||||
| 14 | MODIFIED LYSOSOMAL PROTEIN AND PRODUCTION THEREOF | US15764175 | 2016-09-30 | US20180258408A1 | 2018-09-13 | Erik Nordling; Patrick Strömberg; Stefan Svensson Gelius |
| Disclosed herein are a modified lysosomal protein, methods for preparing a modified lysosomal protein and therapeutic use of such a modified protein. Further disclosed herein is a method of treating a mammal afflicted with a lysosomal storage disease. In particular, the present disclosure relates to a method of preparing a modified lysosomal protein, said method comprising reacting a glycosylated lysosomal protein with an alkali metal periodate and reacting said lysosomal protein with an alkali metal borohydride for a time period of no more than 2 h, thereby modifying glycan moieties of the lysosomal protein and reducing the activity of the lysosomal protein with respect to glycan recognition receptors. | ||||||
| 15 | Plant lectins as carriers of associated drug substances into animal and human cells | US14956001 | 2015-12-01 | US09862939B2 | 2018-01-09 | David N. Radin; Carole L. Cramer |
| The current invention involves the use of protein lectins produced by plants including the non-toxic carbohydrate binding subunits (B subunits) of plant “AB toxins” (PTB lectins) as delivery vehicles for mobilizing associated drug substances for delivery to animal and human cells. The resulting protein fusions or conjugates retain lectin carbohydrate specificity for binding to cells and cellular trafficking activity so as to deliver an associated drug compound to the site of disease manifestation. One embodiment of this invention concerns the ability of ricin toxin B subunit, as a model PTB lectin, to deliver enzyme replacement therapeutic drugs to cells of several organs of the body including the brain and central nervous system, eyes, ears, lungs, bone, heart, kidney, liver, and spleen for treating lysosomal diseases. | ||||||
| 16 | CNS DELIVERY OF THERAPEUTIC AGENTS | US15016141 | 2016-02-04 | US20170042978A1 | 2017-02-16 | Pericles Calias; Jing Pan; Jan Powell; Lawrence Charnas; Thomas McCauley; Teresa Leah Wright; Richard Pfeifer; Zahra Shahrokh |
| The present invention provides an effective and less invasive approach for direct delivery of therapeutic agents to the central nervous system (CNS). In some embodiments, the present invention provides methods including a step of administering intrathecally to a subject suffering from or susceptible to a lysosomal storage disease associated with reduced level or activity of a lysosomal enzyme, a composition comprising a replacement enzyme for the lysosomal enzyme. | ||||||
| 17 | METHODS AND COMPOSITIONS FOR CNS DELIVERY OF HEPARAN N-SULFATASE | US15077046 | 2016-03-22 | US20170042977A1 | 2017-02-16 | Farah Natoli; Gaozhong Zhu; Jennifer Terew; Yuan Jiang; Jamie Tsung; Zahra Shahrokh; Brian Vernaglia; Jing Pan; Richard Pfeifer; Pericles Calias |
| The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising a heparan N-sulfatase (HNS) protein, salt, and a polysorbate surfactant for the treatment of Sanfilippo Syndrome Type A. | ||||||
| 18 | Therapeutic strategies to treat CNS pathology in mucopolysaccharidoses | US14939700 | 2015-11-12 | US09487766B2 | 2016-11-08 | Andrea Ballabio; Alessandro Fraldi |
| The invention provides for nucleotide sequences encoding for a chimeric sulfatase, viral vectors expressing such sequences for gene therapy and pharmaceutical uses of the chimeric expressed protein. The invention is particularly applied in the therapy of mucopolysaccharidosis, preferably type IIIA. | ||||||
| 19 | PRODUCTS AND METHODS FOR DELIVERY OF POLYNUCLEOTIDES BY ADENO-ASSOCIATED VIRUS FOR LYSOSOMAL STORAGE DISORDERS | US14950387 | 2015-11-24 | US20160175406A1 | 2016-06-23 | Douglas M. McCarty; Haiyan Fu |
| The present invention relates to methods and materials useful for systemically delivering polynucleotides across the blood brain barrier using adeno-associated virus as a vector. For example, the present invention relates to methods and materials useful for systemically delivering α-N-acetylglucosamidinase polynucleotides to the central and peripheral nervous systems, as well as the somatic system. Use of these methods and materials is indicated, for example, for treatment of the lysosomal storage disorder mucopolysaccharidosis IIIB. As another example, the present invention relates to methods and materials useful for systemically delivering N-sulphoglucosamine sulfphohydrolase polynucleotides to the central and peripheral nervous systems, as well as the somatic system. Use of this second type of methods and materials is indicated, for example, for treatment of the lysosomal storage disorder mucopolysaccharidosis IIIA. | ||||||
| 20 | PLANT LECTINS AS CARRIERS OF ASSOCIATED DRUG SUBSTANCES INTO ANIMAL AND HUMAN CELLS | US14956001 | 2015-12-01 | US20160083707A1 | 2016-03-24 | DAVID N. RADIN |
| The current invention involves the use of protein lectins produced by plants including the non-toxic carbohydrate binding subunits (B subunits) of plant “AB toxins” (PTB lectins) as delivery vehicles for mobilizing associated drug substances for delivery to animal and human cells. The resulting protein fusions or conjugates retain lectin carbohydrate specificity for binding to cells and cellular trafficking activity so as to deliver an associated drug compound to the site of disease manifestation. One embodiment of this invention concerns the ability of ricin toxin B subunit, as a model PTB lectin, to deliver enzyme replacement therapeutic drugs to cells of several organs of the body including the brain and central nervous system, eyes, ears, lungs, bone, heart, kidney, liver, and spleen for treating lysosomal diseases. | ||||||
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