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
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 (66)
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 |
US20050032841A1 (en) * | 1999-04-20 | 2005-02-10 | Steven Walkley | Therapeutic compositions and methods of treating glycolipid storage related disorders |
DE60138535D1 (en) * | 2000-02-04 | 2009-06-10 | Children S Hospital Res Founda | USE OF LYSOSOMAL ACID LIPASE FOR THE TREATMENT OF ATHEROSCLEROSIS AND SIMILAR DISEASES |
AU2001269923A1 (en) | 2000-06-19 | 2002-01-02 | Genzyme Corporation | 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 |
AU2002241853A1 (en) * | 2001-01-12 | 2002-07-24 | Oxford Glycosciences (Uk) Ltd. | Mucopolysaccharidosis therapies |
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 |
US20030223966A1 (en) * | 2002-04-30 | 2003-12-04 | Fraites Thomas J. | Treatment for pompe disease |
WO2004055157A2 (en) * | 2002-08-13 | 2004-07-01 | Whitley Chester B | Methods of using vectors to treat metabolic disorders |
ES2541785T3 (en) * | 2003-01-31 | 2015-07-24 | The Mount Sinai School Of Medicine Of New York University | Combination therapy to treat protein deficiency disorders |
US7442372B2 (en) | 2003-08-29 | 2008-10-28 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
US20090111812A1 (en) * | 2004-06-14 | 2009-04-30 | Musc Foundation For Research Development | Methods for treating inflammatory disorders |
US9181184B2 (en) * | 2005-05-17 | 2015-11-10 | Amicus Therapeutics, Inc. | Method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives |
WO2007030375A2 (en) * | 2005-09-08 | 2007-03-15 | Children's Hospital Medical Center | Lysosomal acid lipase therapy for nafld and related diseases |
SI1973561T1 (en) * | 2005-12-15 | 2011-07-29 | Chemgen Corp | 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 |
CA2706727A1 (en) | 2007-11-29 | 2009-06-11 | Michael S. Mcgrath | Compositions and methods for treating lysosomal disorders |
EP2271213A4 (en) * | 2008-02-01 | 2011-06-29 | Scripps Research Inst | Methods for treating a condition characterized by dysfunction in protein homeostasis |
JP2011512876A (en) * | 2008-03-12 | 2011-04-28 | アミカス セラピューティックス インコーポレイテッド | Treatment of Pompe disease with specific pharmacological chaperones and monitoring of treatment with surrogate markers |
PT2484371E (en) | 2008-06-26 | 2015-03-03 | Orphazyme Aps | Use of hsp70 as a regulator of enzymatic activity |
BR122020010601B8 (en) | 2008-12-16 | 2021-07-27 | Genzyme Corp | protein-oligosaccharide conjugates, their uses, and pharmaceutical compositions |
CA2793276A1 (en) * | 2009-03-27 | 2010-09-30 | Zacharon Pharmaceuticals, Inc. | Ganglioside biosynthesis modulators |
CN105126086B (en) * | 2009-07-28 | 2019-05-14 | 夏尔人类遗传性治疗公司 | For treating the composition and method of Gaucher disease |
EP2470199B1 (en) * | 2009-08-28 | 2019-05-22 | Icahn School of Medicine at Mount Sinai | Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency |
JP5739898B2 (en) * | 2009-11-17 | 2015-06-24 | ベイラー リサーチ インスティテュートBaylor Research Institute | Urinary triosylceramide (GB3) as a heart disease marker |
US8765698B2 (en) * | 2010-02-03 | 2014-07-01 | University Of Central Florida Research Foundation, Inc. | Methods and products for reawakening retrocyclins |
ES2950358T3 (en) | 2010-04-23 | 2023-10-09 | Alexion Pharma Inc | Lysosomal storage disease enzyme |
WO2012012461A2 (en) | 2010-07-19 | 2012-01-26 | Shire Human Genetic Therapies, Inc. | Mannose receptor c type 1 (mrc1) codon optimized cell line and uses thereof |
EP3650039A1 (en) | 2010-09-09 | 2020-05-13 | Alexion Pharmaceuticals, Inc. | Use of lysosomal acid lipase for treating lysosomal acid lipase deficiency in patients |
ES2753169T3 (en) | 2010-11-30 | 2020-04-07 | Orphazyme As | Procedures to increase intracellular activity of Hsp70 |
WO2012112681A1 (en) | 2011-02-15 | 2012-08-23 | Shire Human Genetic Therapies, Inc. | 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 |
US9265815B2 (en) | 2011-04-29 | 2016-02-23 | Selecta Biosciences, Inc. | Tolerogenic synthetic nanocarriers |
CN103764824B (en) | 2011-06-20 | 2018-04-24 | 西奈山医学院 | The anti-tnf-alpha therapy of mucopolysaccharidosis and other lysosome illnesss |
KR20140138850A (en) | 2012-03-02 | 2014-12-04 | 샤이어 휴먼 지네틱 테라피즈 인크. | Compositions and methods for treating type iii gaucher disease |
KR20230066482A (en) | 2012-03-07 | 2023-05-15 | 아미쿠스 세라퓨틱스, 인코포레이티드 | High concentration alpha-glucosidase compositions for the treatment of pompe disease |
PL2844279T3 (en) | 2012-05-03 | 2021-08-16 | 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 |
US9694056B2 (en) | 2012-07-17 | 2017-07-04 | Amicus Therapeutics, Inc. | α-galactosidase A and 1-deoxygalactonojirimycin co-formulation |
SG11201502989XA (en) | 2012-11-05 | 2015-05-28 | Genzyme Corp | Compositions and methods for treating proteinopathies |
JP7028556B2 (en) | 2013-05-03 | 2022-03-02 | セレクタ バイオサイエンシーズ インコーポレーテッド | Delivery of immunosuppressive agents with antigens for specific pharmacodynamic efficacy and induction of immune tolerance |
MX2017002933A (en) | 2014-09-07 | 2017-05-30 | Selecta Biosciences Inc | Methods and compositions for attenuating gene editing anti-viral transfer vector immune responses. |
BR112017004948A2 (en) | 2014-09-15 | 2017-12-05 | Orphazyme Aps | pharmaceutical formulation. |
SG11201702114TA (en) | 2014-09-30 | 2017-04-27 | Amicus Therapeutics Inc | Highly potent acid alpha-glucosidase with enhanced carbohydrates |
HRP20230468T1 (en) * | 2014-12-22 | 2023-11-10 | Codexis, Inc. | Human alpha-galactosidase variants |
GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
MY198085A (en) | 2015-12-30 | 2023-07-31 | Amicus Therapeutics Inc | Augmented acid alpha-glucosidase for the treatment of pompe disease |
WO2017139381A1 (en) | 2016-02-08 | 2017-08-17 | University Of Iowa Research Foundation | Methods to produce chimeric adeno-associated virus/bocavirus parvovirus |
EP3426787A1 (en) | 2016-03-07 | 2019-01-16 | University of Iowa Research Foundation | Aav-mediated expression using a synthetic promoter and enhancer |
MX2018011679A (en) * | 2016-03-25 | 2019-06-24 | Genzyme Corp | 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 |
CA3019128A1 (en) | 2016-03-30 | 2017-10-05 | Amicus Therapeutics, Inc. | Formulations comprising recombinant acid alpha-glucosidase |
EP3442530A1 (en) | 2016-04-13 | 2019-02-20 | Orphazyme A/S | Heat shock proteins and cholesterol homeostasis |
RU2750154C2 (en) | 2016-04-29 | 2021-06-22 | Орфазим А/С | Arimoclomol for treatment of glucocerebrosidase-associated disorders |
CN109152342A (en) * | 2016-05-12 | 2019-01-04 | 布赖恩.P.汉利 | CRISPR and most of body cell of other gene therapy safe deliveries into human and animal |
NL2017294B1 (en) | 2016-08-05 | 2018-02-14 | Univ Erasmus Med Ct Rotterdam | Natural cryptic exon removal by pairs of antisense oligonucleotides. |
BR112019002719A2 (en) * | 2016-08-11 | 2019-05-14 | Intrabio Ltd | acetyl leucine, or a pharmaceutically acceptable salt thereof, for use in methods related to lysosomal deposition diseases |
EP3509628A1 (en) * | 2016-09-09 | 2019-07-17 | Fondazione Telethon | Combined therapy for mucopolysaccharidosis type vi (maroteaux-lamy-syndrome) |
WO2018169811A1 (en) | 2017-03-11 | 2018-09-20 | Selecta Biosciences, Inc. | Methods and compositions related to combined treatment with anti-inflammatories and synthetic nanocarriers comprising an immunosuppressant |
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 |
EP3823590A4 (en) * | 2018-07-18 | 2022-04-20 | REGENXBIO Inc. | Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idus) |
CA3202568A1 (en) | 2020-11-19 | 2022-05-27 | Zevra Denmark A/S | Processes for preparing arimoclomol citrate and intermediates thereof |
WO2023164060A2 (en) * | 2022-02-25 | 2023-08-31 | 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 |
US5236838A (en) | 1988-12-23 | 1993-08-17 | Genzyme Corporation | Enzymatically active recombinant glucocerebrosidase |
US5549892A (en) | 1988-12-23 | 1996-08-27 | Genzyme Corporation | Enhanced in vivo uptake of 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 |
US5733761A (en) | 1991-11-05 | 1998-03-31 | Transkaryotic Therapies, Inc. | Protein production and protein delivery |
IL105914A0 (en) | 1992-06-04 | 1993-10-20 | Univ California | 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 |
US5910487A (en) | 1994-12-09 | 1999-06-08 | Genzyme Corporation | Cationic amphiphiles and plasmids 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 |
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 |
US5747471A (en) | 1994-12-09 | 1998-05-05 | Genzyme Corporation | Cationic amphiphiles containing steroid lipophilic groups 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 |
US5948767A (en) | 1994-12-09 | 1999-09-07 | Genzyme Corporation | Cationic amphiphile/DNA complexes |
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 |
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 |
AU715543B2 (en) | 1995-09-08 | 2000-02-03 | 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 |
US6177447B1 (en) * | 1996-07-15 | 2001-01-23 | Universiteit Van Amsterdam | Deoxynojirimycin derivatives and their uses as glucosylceramidase inhibitors |
EP1538202B1 (en) | 1996-09-13 | 2014-01-22 | Shire Human Genetic Therapies, Inc. | Production of human alpha-galactosidase A |
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 |
EP1056880A1 (en) | 1998-02-17 | 2000-12-06 | Genzyme Corporation | Methods for purified aav vector production |
WO1999041400A1 (en) | 1998-02-17 | 1999-08-19 | Genzyme Corporation | Methods for pseudoadenoviral vector production |
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 |
GB9909066D0 (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 |
GB9909064D0 (en) | 1999-04-20 | 1999-06-16 | Oxford Glycosciences Uk Ltd | Therapies |
WO2001007078A1 (en) | 1999-07-26 | 2001-02-01 | G.D. Searle & Co. | Use of long-chain n-alkyl derivates of deoxynojirimycin and a glucocerebrosidase enzyme for the manufacture of medicament for the treatment of glycolipid storage diseases |
ES2284545T3 (en) | 1999-11-16 | 2007-11-16 | Genzyme Corporation | VECTORS AND TRANSGINS WITH REGULATING ELEMENTS FOR THE ADMINISTRATION OF LIVER GENES. |
WO2001060377A2 (en) * | 2000-02-17 | 2001-08-23 | Genzyme Corporation | Methods for treatment of lysosomal storage diseases using biphosphonates |
AU2001269923A1 (en) | 2000-06-19 | 2002-01-02 | Genzyme Corporation | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
ES2541785T3 (en) | 2003-01-31 | 2015-07-24 | The Mount Sinai School Of Medicine Of New York University | Combination therapy to treat protein deficiency disorders |
-
2001
- 2001-06-19 AU AU2001269923A patent/AU2001269923A1/en not_active Abandoned
- 2001-06-19 WO PCT/US2001/019579 patent/WO2001097829A2/en active Application Filing
- 2001-06-19 US US09/884,526 patent/US20020095135A1/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 - Lifetime
-
2012
- 2012-03-29 US US13/433,822 patent/US20120183502A1/en not_active Abandoned
Also Published As
Publication number | Publication date |
---|---|
WO2001097829A2 (en) | 2001-12-27 |
US7910545B2 (en) | 2011-03-22 |
WO2001097829A3 (en) | 2002-12-27 |
US20070280925A1 (en) | 2007-12-06 |
US8168587B2 (en) | 2012-05-01 |
US20110142818A1 (en) | 2011-06-16 |
US20120183502A1 (en) | 2012-07-19 |
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 | |
AU1878601A (en) | Enzyme | |
AU3818300A (en) | Treating hair by targeting enzymes | |
AU2001245987A1 (en) | Compositions and methods for gene therapy | |
AU2001245822A1 (en) | Fungal lactate dehydrogenase gene and constructs for the expression thereof | |
AU2001227900A1 (en) | Drug metabolizing enzymes | |
AU2001274981A1 (en) | Drug metabolizing enzymes | |
AU2001236713A1 (en) | Methods for treatment of lysosomal storage diseases | |
AUPQ262599A0 (en) | Gene expression | |
AU2001270303A1 (en) | Drug metabolizing enzymes | |
AU4009899A (en) | Molecules targeting cd40 and tumor cells | |
AUPR311601A0 (en) | Matrix gene expression in chondrogenesis | |
AU2001265096A1 (en) | Short-root gene, promoter, and uses thereof | |
AU2001270478A1 (en) | Gene expression in biological conditions | |
AU7709300A (en) | Drug discovery using gene expression profiling | |
AU2732000A (en) | Reverse gene therapy | |
AU1194201A (en) | Fluorotellurite, amplifier glasses | |
AU2153401A (en) | Gene expression in biological conditions | |
AU2001255324A1 (en) | Drug metabolizing enzymes | |
AU2001235000A1 (en) | Drug metabolizing enzymes | |
AU2001247444A1 (en) | Erbeta-mediated gene expression | |
AU2001278154A1 (en) | Drug metabolizing enzymes | |
AU2352301A (en) | Gene expression in corneal diseases |