AU2001269923A1 - Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases - Google Patents
Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseasesInfo
- Publication number
- AU2001269923A1 AU2001269923A1 AU2001269923A AU6992301A AU2001269923A1 AU 2001269923 A1 AU2001269923 A1 AU 2001269923A1 AU 2001269923 A AU2001269923 A AU 2001269923A AU 6992301 A AU6992301 A AU 6992301A AU 2001269923 A1 AU2001269923 A1 AU 2001269923A1
- Authority
- AU
- Australia
- Prior art keywords
- therapy
- small molecule
- lysosomal storage
- storage diseases
- enzyme replacement
- 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.)
- Abandoned
Links
- 108090000790 Enzymes Proteins 0.000 title 1
- 102000004190 Enzymes Human genes 0.000 title 1
- 208000015439 Lysosomal storage disease Diseases 0.000 title 1
- 238000001415 gene therapy Methods 0.000 title 1
- 150000003384 small molecules Chemical class 0.000 title 1
- 238000002560 therapeutic procedure Methods 0.000 title 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
- A61K31/445—Non condensed piperidines, e.g. piperocaine
-
- 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)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- 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
-
- 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
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Organic Chemistry (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Vascular Medicine (AREA)
- Urology & Nephrology (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Enzymes And Modification Thereof (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US21237700P | 2000-06-19 | 2000-06-19 | |
| US60/212,377 | 2000-06-19 | ||
| PCT/US2001/019579 WO2001097829A2 (en) | 2000-06-19 | 2001-06-19 | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AU2001269923A1 true AU2001269923A1 (en) | 2002-01-02 |
Family
ID=22790749
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2001269923A Abandoned AU2001269923A1 (en) | 2000-06-19 | 2001-06-19 | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
Country Status (3)
| Country | Link |
|---|---|
| US (4) | US20020095135A1 (en) |
| AU (1) | AU2001269923A1 (en) |
| WO (1) | WO2001097829A2 (en) |
Families Citing this family (72)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6274597B1 (en) | 1998-06-01 | 2001-08-14 | Mount Sinai School Of Medicine Of New York University | Method of enhancing lysosomal α-Galactosidase A |
| GB9909066D0 (en) * | 1999-04-20 | 1999-06-16 | Oxford Glycosciences Uk Ltd | Therapies |
| WO2002055064A2 (en) * | 2001-01-12 | 2002-07-18 | Oxford Glycosciences (Uk) Ltd. | Mucopolysaccharidosis therapies |
| US20050032841A1 (en) * | 1999-04-20 | 2005-02-10 | Steven Walkley | Therapeutic compositions and methods of treating glycolipid storage related disorders |
| BR0108077A (en) | 2000-02-04 | 2002-10-22 | Children S Hospital Res Founda | Process for reducing atherosclerotic plaques in a mammal, method for treating atherosclerosis in a mammal, composition, methods for providing biologically active lipid hydrolyzing protein or polypeptide or mixture thereof, for cells of a mammal having biologically hydrolyzing lipid protein or polypeptide active, to provide biologically active lysosomal acid lipase to cells of a mammal having biologically active lysosomal acid lipase deficiency, to provide biologically active lysosomal acid lipase to cells of a mammal with atherosclerosis, to treat wolman's disease in a mammal , for treatment of cholesterol ester storage disease in a mammal, and for treatment of atherosclerosis in a mammal |
| US20020095135A1 (en) | 2000-06-19 | 2002-07-18 | David Meeker | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
| US7138262B1 (en) * | 2000-08-18 | 2006-11-21 | Shire Human Genetic Therapies, Inc. | High mannose proteins and methods of making high mannose proteins |
| US20020142299A1 (en) * | 2001-01-09 | 2002-10-03 | Davidson Beverly L. | PTD-modified proteins |
| US8241622B2 (en) * | 2001-07-13 | 2012-08-14 | University Of Iowa Research Foundation | Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences |
| EP1336411A1 (en) * | 2002-02-14 | 2003-08-20 | Academisch Medisch Centrum bij de Universiteit van Amsterdam | Compositions and methods for improving enzyme replacement therapy of lysosomal storage diseases |
| AU2003223780A1 (en) * | 2002-04-30 | 2003-11-17 | University Of Florida | Treatment for pompe disease |
| AU2003302724A1 (en) * | 2002-08-13 | 2004-07-09 | Mcivor, Scott, R. | Methods of using vectors to treat metabolic disorders |
| DK1589993T3 (en) | 2003-01-31 | 2015-03-09 | Sinai School Medicine | COMBINATION THERAPY FOR TREATMENT OF PROTEIN FAILURE DISORDERS |
| US7442372B2 (en) | 2003-08-29 | 2008-10-28 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| WO2005123055A2 (en) * | 2004-06-14 | 2005-12-29 | Musc Foundation For Research Development | Methods for treating inflammatory disorders |
| CA2612538C (en) * | 2005-05-17 | 2015-06-30 | Amicus Therapeutics, Inc. | A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives |
| US20090297496A1 (en) * | 2005-09-08 | 2009-12-03 | Childrens Hospital Medical Center | Lysosomal Acid Lipase Therapy for NAFLD and Related Diseases |
| CA2633066C (en) * | 2005-12-15 | 2015-01-20 | Chemgen Corporation | Enzymes for reduced immunological stress |
| ES2391657T3 (en) | 2006-02-07 | 2012-11-28 | Shire Human Genetic Therapies, Inc. | Stabilized protein compositions that have a free thiol moiety |
| AU2008334094B2 (en) | 2007-11-29 | 2015-01-22 | Neuraltus Pharmaceuticals, Inc. | Compositions and methods for treating lysosomal disorders |
| JP2011511007A (en) * | 2008-02-01 | 2011-04-07 | ザ スクリプス リサーチ インスティチュート | Methods for treating conditions characterized by dysfunction of protein homeostasis |
| US20110189710A1 (en) * | 2008-03-12 | 2011-08-04 | Amicus Therapeutics, Inc. | TREATMENT OF POMPE DISEASE WITH SPECIFIC PHARMACOLOGICAL CHAPERONES AND MONITORING TREATMENT USING SURROGATE MARkERS |
| CA3004867C (en) | 2008-06-26 | 2020-09-15 | Orphazyme Aps | Use of hsp70 as a regulator of enzymatic activity |
| MX2011006347A (en) | 2008-12-16 | 2011-09-06 | Genzyme Corp | Oligosaccharide-protein conjugates. |
| EP2411526A4 (en) * | 2009-03-27 | 2012-09-19 | Zacharon Pharmaceuticals Inc | Ganglioside biosynthesis modulators |
| MX379016B (en) * | 2009-07-28 | 2025-03-10 | Takeda Pharmaceuticals Co | COMPOSITIONS AND METHODS FOR TREATING GAUCHER'S DISEASE. |
| DK3482767T3 (en) | 2009-08-28 | 2021-12-20 | Icahn School Med Mount Sinai | DOSAGE RECEPTION ENZYMER SUBSTITUTE TO TREAT ACID SPHINGOMYELINASE DEFICIENCY |
| EP3028701B1 (en) * | 2009-11-17 | 2019-11-13 | Baylor Research Institute | 1-deoxygalactonojirimycin for use in treating a cardiac disease |
| US8765698B2 (en) * | 2010-02-03 | 2014-07-01 | University Of Central Florida Research Foundation, Inc. | Methods and products for reawakening retrocyclins |
| HUE033217T2 (en) | 2010-04-23 | 2017-11-28 | Alexion Pharma Inc | Lysosomal storage disease enzyme |
| US9453847B2 (en) | 2010-07-19 | 2016-09-27 | Shire Human Genetic Therapies, Inc. | Mannose receptor C type 1 (MRC1) codon optimized cell line and uses thereof |
| ME02062B (en) | 2010-09-09 | 2015-05-20 | Synageva Biopharma Corp | Use of lysosomal acid lipase for treating lysosomal acid lipase deficiency in patients |
| JP2013544263A (en) | 2010-11-30 | 2013-12-12 | オルファザイム エーピーエス | Method for increasing intracellular activity of Hsp70 |
| EP2675472A4 (en) | 2011-02-15 | 2014-09-17 | Synageva Biopharma Corp | Methods for treating lysosomal acid lipase deficiency |
| WO2012112677A2 (en) * | 2011-02-15 | 2012-08-23 | Children's Hospital Medical Center | Methods for treating lysosomal acid lipase deficiency |
| CN117205331A (en) | 2011-04-29 | 2023-12-12 | 西莱克塔生物科技公司 | Tolerogenic synthetic nanocarriers for reducing antibody responses |
| EP2721151B1 (en) | 2011-06-20 | 2017-12-06 | Mount Sinai School Of Medicine | Anti-tnf-therapy for the mucopolysaccharidoses and other lysosomal disorders |
| CA2865614A1 (en) | 2012-03-02 | 2013-09-06 | Shire Human Genetic Therapies, Inc. | Compositions and methods for treating type iii gaucher disease |
| KR20140135222A (en) | 2012-03-07 | 2014-11-25 | 아미쿠스 세라퓨틱스, 인코포레이티드 | High concentration alpha-glucosidase compositions for the treatment of pompe disease |
| SI2844279T1 (en) | 2012-05-03 | 2021-04-30 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of pompe disease |
| US10155027B2 (en) | 2012-07-17 | 2018-12-18 | Amicus Therapeutics, Inc. | Alpha-galactosidase A and 1-deoxygalactonojirimycin co-formulation for the treatment of fabry disease |
| CA2917995C (en) | 2012-07-17 | 2021-01-26 | Amicus Therapeutics, Inc. | Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation |
| EP2914281A1 (en) | 2012-11-05 | 2015-09-09 | Genzyme Corporation | Compositions and methods for treating proteinopathies |
| CN110639024A (en) | 2013-05-03 | 2020-01-03 | 西莱克塔生物科技公司 | Methods and compositions for enhancing CD4+ regulatory T cells |
| BR112017001601A2 (en) | 2014-09-07 | 2017-11-21 | Selecta Biosciences Inc | methods and compositions for attenuating immune responses of the modulating antiviral transfer vector of gene expression |
| JP6678676B2 (en) | 2014-09-15 | 2020-04-08 | オーファザイム エー/エス | Arimoclomol formulation |
| HUE070443T2 (en) | 2014-09-30 | 2025-06-28 | Amicus Therapeutics Inc | Highly potent acid alpha-glucosidase with enhanced carbohydrates |
| JP6650943B2 (en) | 2014-12-22 | 2020-02-19 | コデクシス, インコーポレイテッド | Human α-galactosidase variant |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| AU2016381832B2 (en) | 2015-12-30 | 2024-01-25 | Amicus Therapeutics, Inc. | Augmented acid alpha-glucosidase for the treatment of Pompe disease |
| US11702672B2 (en) | 2016-02-08 | 2023-07-18 | University Of Iowa Research Foundation | Methods to produce chimeric adeno-associated virus/bocavirus parvovirus |
| US11684679B2 (en) | 2016-03-07 | 2023-06-27 | University Of Iowa Research Foundation | AAV-mediated expression using a synthetic promoter and enhancer |
| CA3018745A1 (en) * | 2016-03-25 | 2017-09-28 | Genzyme Corporation | Biomarkers of proteopathies and uses thereof |
| WO2017173059A1 (en) | 2016-03-30 | 2017-10-05 | Amicus Therapeutics, Inc. | Method for selection of high m6p recombinant proteins |
| KR102618519B1 (en) | 2016-03-30 | 2023-12-28 | 아미쿠스 세라퓨틱스, 인코포레이티드 | Formulations comprising recombinant acid alpha-glucosidase |
| EP3442530A1 (en) | 2016-04-13 | 2019-02-20 | Orphazyme A/S | Heat shock proteins and cholesterol homeostasis |
| PT3448382T (en) | 2016-04-29 | 2020-11-20 | Orphazyme As C/O Cobis As | Arimoclomol for treating glucocerebrosidase associated disorders |
| CN109152342A (en) * | 2016-05-12 | 2019-01-04 | 布赖恩.P.汉利 | CRISPR and other gene therapies safely delivered to most somatic cells in humans and animals |
| WO2017205739A1 (en) | 2016-05-26 | 2017-11-30 | University Of Iowa Research Foundation | cis AND trans REQUIREMENTS FOR TERMINAL RESOLUTION OF HUMAN BOCAVIRUS 1 |
| NL2017294B1 (en) | 2016-08-05 | 2018-02-14 | Univ Erasmus Med Ct Rotterdam | Natural cryptic exon removal by pairs of antisense oligonucleotides. |
| MA43876B1 (en) * | 2016-08-11 | 2019-04-30 | Intrabio Ltd | Pharmaceutical compositions and their uses against lysosomal storage diseases |
| WO2018046737A1 (en) * | 2016-09-09 | 2018-03-15 | Fondazione Telethon | Combined therapy for mucopolysaccharidosis type vi (maroteaux-lamy-syndrome) |
| WO2018132747A1 (en) | 2017-01-13 | 2018-07-19 | University Of Iowa Research Foundation | Bocaparvovirus small noncoding rna and uses thereof |
| MX2019010757A (en) | 2017-03-11 | 2020-01-20 | Selecta Biosciences Inc | METHODS AND COMPOSITIONS RELATED TO THE COMBINED TREATMENT WITH ANTI-INFLAMMATORY AND SYNTHETIC NANPORTERS THAT INCLUDE AN IMMUNOSUPPRESSOR. |
| EP3624831B1 (en) | 2017-05-15 | 2023-03-29 | Amicus Therapeutics, Inc. | Recombinant human acid alpha-glucosidase |
| BR112019025888A2 (en) | 2017-06-07 | 2020-06-30 | Regeneron Pharmaceuticals, Inc. | polynucleotide encoding a therapeutic multi-domain protein, gene therapy vector, recombinant multi-domain therapeutic protein, expression method, methods to reduce the accumulation of glycogen in a tissue in a patient in need, to reduce the accumulation of glycogen in a tissue in a patient in need and to treat enzyme deficiency in a patient in need and / or tolerate the patient to the enzyme for which he is deficient, anti-cd63 antibody or antigen binding fragment, and pharmaceutical composition |
| AU2019218892B2 (en) | 2018-02-07 | 2025-08-14 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for therapeutic protein delivery |
| SG11202011232VA (en) | 2018-05-17 | 2020-12-30 | Regeneron Pharma | Anti-cd63 antibodies, conjugates, and uses thereof |
| BR112021000830A2 (en) * | 2018-07-18 | 2021-04-13 | Regenxbio Inc. | TREATMENT OF MUCOPOLYSACARIDOSIS I WITH ALUMINUM-L-IDURONIDASE (IDUS) HUMAN TOTALLY HUMAN AND GLYCOSILATED |
| MY207075A (en) | 2018-12-20 | 2025-01-28 | Codexis Inc | Human alpha-galactosidase variants |
| IL303026A (en) | 2020-11-19 | 2023-07-01 | Zevra Denmark As | Processes for preparing arimoclomol citrate and its intermediates |
| US20250163457A1 (en) * | 2022-02-25 | 2025-05-22 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus for treating pompe disease with long term cessation of gaa enzyme replacement therapy |
Family Cites Families (47)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5851991A (en) * | 1987-08-31 | 1998-12-22 | The Regents Of The University Of California | Therapeutic use of the retinoblastoma susceptibility gene product |
| US5549892A (en) * | 1988-12-23 | 1996-08-27 | Genzyme Corporation | Enhanced in vivo uptake of glucocerebrosidase |
| US5236838A (en) * | 1988-12-23 | 1993-08-17 | Genzyme Corporation | Enzymatically active recombinant glucocerebrosidase |
| US5179023A (en) * | 1989-03-24 | 1993-01-12 | Research Corporation Technologies, Inc. | Recombinant α-galactosidase a therapy for Fabry disease |
| US5272071A (en) * | 1989-12-22 | 1993-12-21 | Applied Research Systems Ars Holding N.V. | Method for the modification of the expression characteristics of an endogenous gene of a given cell line |
| US5670488A (en) * | 1992-12-03 | 1997-09-23 | Genzyme Corporation | Adenovirus vector for gene therapy |
| US5236938A (en) * | 1991-04-30 | 1993-08-17 | Rhone-Poulenc Inc. | Pesticidal 1-aryl-5-(substituted alkylideneimino)pyrazoles |
| US5968502A (en) * | 1991-11-05 | 1999-10-19 | Transkaryotic Therapies, Inc. | Protein production and protein delivery |
| JP3285355B2 (en) * | 1992-06-04 | 2002-05-27 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | Methods and compositions for in vivo gene therapy |
| US5834266A (en) * | 1993-02-12 | 1998-11-10 | President & Fellows Of Harvard College | Regulated apoptosis |
| US5836905A (en) * | 1994-06-20 | 1998-11-17 | Lemelson; Jerome H. | Apparatus and methods for gene therapy |
| US5767099A (en) * | 1994-12-09 | 1998-06-16 | Genzyme Corporation | Cationic amphiphiles containing amino acid or dervatized amino acid groups for intracellular delivery of therapeutic molecules |
| US5840710A (en) * | 1994-12-09 | 1998-11-24 | Genzyme Corporation | Cationic amphiphiles containing ester or ether-linked lipophilic groups for intracellular delivery of therapeutic molecules |
| US5747471A (en) * | 1994-12-09 | 1998-05-05 | Genzyme Corporation | Cationic amphiphiles containing steroid lipophilic groups for intracellular delivery of therapeutic molecules |
| US5948767A (en) * | 1994-12-09 | 1999-09-07 | Genzyme Corporation | Cationic amphiphile/DNA complexes |
| US5910487A (en) * | 1994-12-09 | 1999-06-08 | Genzyme Corporation | Cationic amphiphiles and plasmids for intracellular delivery of therapeutic molecules |
| US5650096A (en) * | 1994-12-09 | 1997-07-22 | Genzyme Corporation | Cationic amphiphiles for intracellular delivery of therapeutic molecules |
| US5939401A (en) * | 1994-12-09 | 1999-08-17 | Genzyme Corporation | Cationic amphiphile compositions for intracellular delivery of therapeutic molecules |
| US5719131A (en) * | 1994-12-09 | 1998-02-17 | Genzyme Corporation | Cationic amphiphiles containing dialkylamine lipophilic groups for intracellular delivery of therapeutic molecules |
| US6492332B1 (en) * | 1995-12-12 | 2002-12-10 | Omeros Corporation | Irrigation solution and methods for inhibition of tumor cell adhesion, pain and inflammation |
| US5707618A (en) * | 1995-03-24 | 1998-01-13 | Genzyme Corporation | Adenovirus vectors for gene therapy |
| US5756283A (en) * | 1995-06-05 | 1998-05-26 | The Trustees Of The University Of Pennsylvania | Method for improved production of recombinant adeno-associated viruses for gene therapy |
| EP0850313B8 (en) | 1995-09-08 | 2009-07-29 | Genzyme Corporation | Improved aav vectors for gene therapy |
| WO1997010817A1 (en) * | 1995-09-20 | 1997-03-27 | The Regents Of The University Of Michigan | Amino ceramide-like compounds and therapeutic methods of use |
| US5840702A (en) * | 1996-03-22 | 1998-11-24 | Uab Research Foundation | Cystic fibrosis treatment |
| US5935936A (en) * | 1996-06-03 | 1999-08-10 | Genzyme Corporation | Compositions comprising cationic amphiphiles and co-lipids for intracellular delivery of therapeutic molecules |
| IL128036A (en) * | 1996-07-15 | 2005-08-31 | Univ Amsterdam | Deoxynojirimycin derivatives comprising a polyclic alcohol group, pharmaceutical composition comprising a deoxynojirimycin derivative and uses thereof |
| PL190041B1 (en) | 1996-09-13 | 2005-10-31 | Transkaryotic Therapies | Therapy against alpha-galactosidase a deficiency |
| US5861397A (en) * | 1996-10-03 | 1999-01-19 | Vical Incorporated | Piperazine based cytofectins |
| US5925628A (en) * | 1997-03-31 | 1999-07-20 | Genzyme Corporation | Cationic amphiphiles for intracellular delivery of therapeutic molecules |
| US5912239A (en) * | 1997-04-04 | 1999-06-15 | Genzyme Corporation | Imidazole-containing cationic amphiphiles for intracellular delivery of therapeutic molecules |
| US5948925A (en) * | 1997-05-06 | 1999-09-07 | Genzyme Corporation | Cationic amphiphiles containing linkers derived from neutral or positively charged amino acids |
| US5942634A (en) * | 1997-05-09 | 1999-08-24 | Genzyme Corporation | Cationic amphiphiles for cell transfections |
| EP2147681A1 (en) * | 1997-10-29 | 2010-01-27 | Genzyme Corporation | Compositions and methods for treating lysosomal storage disease |
| US6465488B1 (en) * | 1997-12-11 | 2002-10-15 | Chancellor, Masters & Scholars Of The University Of Oxford | Inhibition of glycolipid biosynthesis |
| JP2002538758A (en) | 1998-02-17 | 2002-11-19 | ジェンザイム・コーポレイション | Method for producing pseudo adenovirus vector |
| US6303371B1 (en) | 1998-02-17 | 2001-10-16 | Genzyme Corporation | Method of purified rAAV vector production in non-human cell line transfected with cocksackie and adenovirus receptor |
| WO1999057296A1 (en) | 1998-05-01 | 1999-11-11 | Genzyme Corporation | Partially deleted adenoviral vectors |
| US6610703B1 (en) * | 1998-12-10 | 2003-08-26 | G.D. Searle & Co. | Method for treatment of glycolipid storage diseases |
| GB9909064D0 (en) | 1999-04-20 | 1999-06-16 | Oxford Glycosciences Uk Ltd | Therapies |
| GB0100889D0 (en) * | 2001-01-12 | 2001-02-21 | Oxford Glycosciences Uk Ltd | Compounds |
| GB9909066D0 (en) * | 1999-04-20 | 1999-06-16 | Oxford Glycosciences Uk Ltd | Therapies |
| DE60001745T2 (en) * | 1999-07-26 | 2004-03-04 | G.D. Searle & Co., Chicago | USE OF LONG-CHAIN N-ALKYL DERIVATIVES OF DEOXYNOJIRIMYCIN WITH GLUCOCEREBROSIDASE ENZYMES FOR PRODUCING A MEDICINE FOR TREATING DISEASES RELATING TO GLYCOLIPID ACCUMULATION |
| ES2284545T3 (en) * | 1999-11-16 | 2007-11-16 | Genzyme Corporation | VECTORS AND TRANSGINS WITH REGULATING ELEMENTS FOR THE ADMINISTRATION OF LIVER GENES. |
| US20010031741A1 (en) * | 2000-02-17 | 2001-10-18 | Robin Ziegler | Methods for treatment of lysosomal storage diseases |
| US20020095135A1 (en) | 2000-06-19 | 2002-07-18 | David Meeker | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
| DK1589993T3 (en) * | 2003-01-31 | 2015-03-09 | Sinai School Medicine | COMBINATION THERAPY FOR TREATMENT OF PROTEIN FAILURE DISORDERS |
-
2001
- 2001-06-19 US US09/884,526 patent/US20020095135A1/en not_active Abandoned
- 2001-06-19 WO PCT/US2001/019579 patent/WO2001097829A2/en not_active Ceased
- 2001-06-19 AU AU2001269923A patent/AU2001269923A1/en not_active Abandoned
-
2007
- 2007-06-13 US US11/762,689 patent/US7910545B2/en not_active Expired - Fee Related
-
2011
- 2011-02-23 US US13/033,344 patent/US8168587B2/en not_active Expired - Fee Related
-
2012
- 2012-03-29 US US13/433,822 patent/US20120183502A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| US7910545B2 (en) | 2011-03-22 |
| WO2001097829A3 (en) | 2002-12-27 |
| WO2001097829A2 (en) | 2001-12-27 |
| US20110142818A1 (en) | 2011-06-16 |
| US8168587B2 (en) | 2012-05-01 |
| US20120183502A1 (en) | 2012-07-19 |
| US20070280925A1 (en) | 2007-12-06 |
| US20020095135A1 (en) | 2002-07-18 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| AU2001269923A1 (en) | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases | |
| EP1463512A4 (en) | Use of p97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes | |
| AU2001280767A1 (en) | Peptide-mediated delivery of molecules into cells | |
| AU3818300A (en) | Treating hair by targeting enzymes | |
| AU2001245987A1 (en) | Compositions and methods for gene therapy | |
| AU2001227900A1 (en) | Drug metabolizing enzymes | |
| AU2001236713A1 (en) | Methods for treatment of lysosomal storage diseases | |
| AUPR311601A0 (en) | Matrix gene expression in chondrogenesis | |
| AU2001274981A1 (en) | Drug metabolizing enzymes | |
| AUPQ262599A0 (en) | Gene expression | |
| AU2001270303A1 (en) | Drug metabolizing enzymes | |
| AU2002312624A1 (en) | Drug metabolizing enzymes | |
| AU7709300A (en) | Drug discovery using gene expression profiling | |
| AU2001255324A1 (en) | Drug metabolizing enzymes | |
| AU2001235000A1 (en) | Drug metabolizing enzymes | |
| AU2732000A (en) | Reverse gene therapy | |
| AU1194201A (en) | Fluorotellurite, amplifier glasses | |
| AU2153401A (en) | Gene expression in biological conditions | |
| AU2001247444A1 (en) | Erbeta-mediated gene expression | |
| AU2001278154A1 (en) | Drug metabolizing enzymes | |
| AU2352301A (en) | Gene expression in corneal diseases | |
| AU4144600A (en) | Cyp2a6 gene judgment methods | |
| AU2001284680A1 (en) | Haplotypes of the alas2 gene | |
| AU4552700A (en) | Gene therapy | |
| AU2002230890A1 (en) | Targeted enzyme prodrug therapy |