SG11202010645VA - Use of lentivector-transduced t-rapa cells for amelioration of lysosomal storage disorders - Google Patents
Use of lentivector-transduced t-rapa cells for amelioration of lysosomal storage disordersInfo
- Publication number
- SG11202010645VA SG11202010645VA SG11202010645VA SG11202010645VA SG11202010645VA SG 11202010645V A SG11202010645V A SG 11202010645VA SG 11202010645V A SG11202010645V A SG 11202010645VA SG 11202010645V A SG11202010645V A SG 11202010645VA SG 11202010645V A SG11202010645V A SG 11202010645VA
- Authority
- SG
- Singapore
- Prior art keywords
- lentivector
- transduced
- amelioration
- lysosomal storage
- storage disorders
- Prior art date
Links
- 208000015439 Lysosomal storage disease Diseases 0.000 title 1
Classifications
-
- 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
- A61K38/44—Oxidoreductases (1)
- A61K38/443—Oxidoreductases (1) acting on CH-OH groups as donors, e.g. glucose oxidase, lactate dehydrogenase (1.1)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- 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
-
- 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
- A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
- A61K31/5375—1,4-Oxazines, e.g. morpholine
- A61K31/5377—1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/14—Blood; Artificial blood
- A61K35/17—Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes
-
- 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
- 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
- A61K38/54—Mixtures of enzymes or proenzymes covered by more than a single one of groups A61K38/44 - A61K38/46 or A61K38/51 - A61K38/53
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K40/00—Cellular immunotherapy
- A61K40/10—Cellular immunotherapy characterised by the cell type used
- A61K40/11—T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K40/00—Cellular immunotherapy
- A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0634—Cells from the blood or the immune system
- C12N5/0636—T lymphocytes
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K40/00
- A61K2239/31—Indexing codes associated with cellular immunotherapy of group A61K40/00 characterized by the route of administration
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K40/00
- A61K2239/38—Indexing codes associated with cellular immunotherapy of group A61K40/00 characterised by the dose, timing or administration schedule
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2510/00—Genetically modified cells
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16041—Use of virus, viral particle or viral elements as a vector
- C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y101/00—Oxidoreductases acting on the CH-OH group of donors (1.1)
- C12Y101/01—Oxidoreductases acting on the CH-OH group of donors (1.1) with NAD+ or NADP+ as acceptor (1.1.1)
- C12Y101/01205—IMP dehydrogenase (1.1.1.205)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01022—Alpha-galactosidase (3.2.1.22)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Epidemiology (AREA)
- Immunology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Cell Biology (AREA)
- Microbiology (AREA)
- Hematology (AREA)
- Molecular Biology (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Developmental Biology & Embryology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201862663786P | 2018-04-27 | 2018-04-27 | |
PCT/US2019/029639 WO2019210301A1 (en) | 2018-04-27 | 2019-04-29 | Use of lentivector-transduced t-rapa cells for amelioration of lysosomal storage disorders |
Publications (1)
Publication Number | Publication Date |
---|---|
SG11202010645VA true SG11202010645VA (en) | 2020-11-27 |
Family
ID=68295811
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG11202010645VA SG11202010645VA (en) | 2018-04-27 | 2019-04-29 | Use of lentivector-transduced t-rapa cells for amelioration of lysosomal storage disorders |
Country Status (7)
Country | Link |
---|---|
US (1) | US12239693B2 (en) |
EP (2) | EP3784695B1 (en) |
JP (1) | JP2021522278A (en) |
KR (1) | KR20210005167A (en) |
CN (1) | CN112513072A (en) |
SG (1) | SG11202010645VA (en) |
WO (1) | WO2019210301A1 (en) |
Families Citing this family (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2020005341A1 (en) * | 2018-06-29 | 2020-01-02 | Icahn School Of Medicine At Mount Sinai | Anc80 encoding sphingolipid-metabolizing proteins |
CN115379863A (en) * | 2020-04-14 | 2022-11-22 | 吉尼松公司 | Carrier for treating acid ceramidase deficiency |
CN118715316A (en) * | 2022-10-08 | 2024-09-27 | 凌意(杭州)生物科技有限公司 | Polynucleotides for treating GCase deficiency-related diseases |
Family Cites Families (31)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US6326007B1 (en) | 1995-07-20 | 2001-12-04 | The Regents Of The University Of California | Attenuated lentivirus vectors expressing interferon |
US6013516A (en) | 1995-10-06 | 2000-01-11 | The Salk Institute For Biological Studies | Vector and method of use for nucleic acid delivery to non-dividing cells |
GB9621680D0 (en) | 1996-10-17 | 1996-12-11 | Oxford Biomedica Ltd | Lentiviral vectors |
WO1998039463A2 (en) | 1997-03-06 | 1998-09-11 | Ueberla Klaus | Lentivirus based vector and vector system |
US6207455B1 (en) | 1997-05-01 | 2001-03-27 | Lung-Ji Chang | Lentiviral vectors |
KR100556864B1 (en) | 1997-05-13 | 2006-03-10 | 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 | Lentivirus-originated gene transfer vector |
EP1003894A2 (en) | 1997-07-18 | 2000-05-31 | Chiron Corporation | Lentiviral vectors |
ES2337429T3 (en) | 1999-10-11 | 2010-04-23 | Institut Pasteur | LENTIVIRAL VECTORS FOR THE PREPARATION OF IMMUNOTHERAPEUTIC COMPOSITIONS. |
AU784988B2 (en) | 1999-11-10 | 2006-08-17 | Uab Research Foundation, The | Lentiviral vector transduction of hematopoietic stem cells |
US20020014242A1 (en) | 2000-07-31 | 2002-02-07 | Abraham Scaria | Use of rapamycin to inhibit immune response and induce tolerance to gene therapy vector and encoded transgene products |
US6627442B1 (en) | 2000-08-31 | 2003-09-30 | Virxsys Corporation | Methods for stable transduction of cells with hiv-derived viral vectors |
US7575924B2 (en) | 2000-11-13 | 2009-08-18 | Research Development Foundation | Methods and compositions relating to improved lentiviral vectors and their applications |
EP1446014A4 (en) | 2001-10-01 | 2004-12-08 | Univ Emory | INTRODUCTION OF THE WLDS GENE FOR THE PREVENTION OF AXONAL DEGENERATION IN NEUROLOGICAL DISEASES |
CA2462628C (en) | 2001-10-02 | 2019-08-20 | Institut Clayton De La Recherche | Restricted expression lentiviral vectors |
US8568709B2 (en) | 2008-03-20 | 2013-10-29 | University Health Network | Thymidylate kinase fusions and uses thereof |
KR20180108886A (en) | 2010-03-23 | 2018-10-04 | 인트렉손 코포레이션 | Vectors Conditionally Expressing Therapeutic Proteins, Host Cells Comprising the Vectors, and Uses Thereof |
RU2013125923A (en) | 2010-11-30 | 2015-01-10 | Орфазиме Апс | METHODS FOR INCREASING THE EXTRACELLULAR ACTIVITY OF HSP70 |
US9387236B2 (en) | 2011-06-10 | 2016-07-12 | Prothera Inc. | Pharmaceutical compositions containing protease and methods for the treatment of lysosomal storage diseases |
ES2632444T5 (en) * | 2011-09-30 | 2022-03-02 | Bluebird Bio Inc | Compounds that enhance viral transduction |
WO2013151672A2 (en) | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of oncology-related proteins and peptides |
CA2930784C (en) | 2013-11-22 | 2023-01-31 | Cellectis | Method for generating batches of allogeneic t cells with averaged potency |
CN105828811B (en) * | 2013-12-23 | 2022-04-08 | Bcn肽类股份有限公司 | Bicalutamide analogs or (S) -bicalutamide as exocytosis activating compounds for use in the treatment of lysosomal storage disorders or glycogen storage diseases |
GB201407322D0 (en) | 2014-04-25 | 2014-06-11 | Ospedale San Raffaele | Gene therapy |
MX2016014773A (en) * | 2014-05-14 | 2017-05-04 | Esteve Labor Dr | Adenoassociated virus vectors for the treatment of lysosomal storage disorders. |
BR112017013300A2 (en) | 2014-12-22 | 2018-02-27 | Codexis Inc | recombinant alpha galactosidase a and / or biologically active recombinant alpha galactosidase fragment, composition, recombinant polynucleotide sequence, expression vector, host cell, methods for producing a variant alpha galactosidase ae for treating and / or preventing symptoms of fabry, pharmaceutical composition, and, use of compositions. |
WO2016183593A2 (en) | 2015-05-14 | 2016-11-17 | The Regents Of The University Of California | Prenatal therapy |
WO2017093464A1 (en) | 2015-12-04 | 2017-06-08 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Method of amplifying a population of antigen-specific memory cd4+ t cells using artificial presenting cells expressing hla class ii molecules |
EP3293259A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
JP7576293B2 (en) | 2017-01-13 | 2024-10-31 | アイカーン スクール オブ メディシン アット マウント サイナイ | Compositions and methods for treating Farber disease |
US11597917B2 (en) | 2017-07-06 | 2023-03-07 | The Medical College Of Wisconsin, Inc. | In vitro and in vivo enrichment strategy targeting lymphocytes derived from vector transduced HSCs for therapy of disorders |
WO2019046815A1 (en) | 2017-08-31 | 2019-03-07 | Poseida Therapeutics, Inc. | Transposon system and methods of use |
-
2019
- 2019-04-29 SG SG11202010645VA patent/SG11202010645VA/en unknown
- 2019-04-29 KR KR1020207034012A patent/KR20210005167A/en active Pending
- 2019-04-29 WO PCT/US2019/029639 patent/WO2019210301A1/en active Application Filing
- 2019-04-29 EP EP19793684.2A patent/EP3784695B1/en active Active
- 2019-04-29 CN CN201980043179.XA patent/CN112513072A/en active Pending
- 2019-04-29 JP JP2020560341A patent/JP2021522278A/en active Pending
- 2019-04-29 US US17/050,949 patent/US12239693B2/en active Active
- 2019-04-29 EP EP23190055.6A patent/EP4285921A3/en active Pending
Also Published As
Publication number | Publication date |
---|---|
WO2019210301A1 (en) | 2019-10-31 |
EP3784695B1 (en) | 2023-08-09 |
EP3784695A4 (en) | 2022-02-23 |
EP3784695A1 (en) | 2021-03-03 |
US20210322472A1 (en) | 2021-10-21 |
JP2021522278A (en) | 2021-08-30 |
CN112513072A (en) | 2021-03-16 |
KR20210005167A (en) | 2021-01-13 |
US12239693B2 (en) | 2025-03-04 |
EP4285921A2 (en) | 2023-12-06 |
EP4285921A3 (en) | 2024-03-06 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
GB2575360B (en) | Expandable sockets for use with portable media players | |
EP3332438B8 (en) | Redox flow cell for the storage of electrical energy and use thereof | |
EP3200591A4 (en) | Central memory t cells for adoptive t cell therapy | |
SG10202005898PA (en) | Cell culture medium | |
EP3155620A4 (en) | Memory cell and an array of memory cells | |
IL248538A0 (en) | Adenoassociated virus vectors for the treatment of lysosomal storage disorders | |
EP3401981A4 (en) | Electrode for energy storage devices | |
EP3134409A4 (en) | Compounds and use thereof in the expansion of hematopoietic stem cells and/or hematopoietic progenitor cells | |
GB201715930D0 (en) | Cell Culture Medium | |
EP3569697B8 (en) | Stem cell therapy in endometrial pathologies | |
EP3145543A4 (en) | Treatment of eosinophil or mast cell related disorders | |
SG11202010645VA (en) | Use of lentivector-transduced t-rapa cells for amelioration of lysosomal storage disorders | |
EP3380102A4 (en) | Telomere extension and anti-inflammatory agents for cell regeneration | |
EP3122278A4 (en) | Expandable catheter devices electrode array | |
EP3128003A4 (en) | Medium for stem cell use | |
EP3765044A4 (en) | Methods for treating non-cancerous disorders using hematopoietic cells | |
SG11202009770TA (en) | Improving cell access procedure | |
EP3313386A4 (en) | Methods and compositions for treating lysosomal storage disorders | |
EP3189155A4 (en) | Methods relating to testing for lysosomal storage disorders | |
EP3178318A4 (en) | Composition for promoting storage stability of stem cells | |
SG11202010878QA (en) | Cell culture medium | |
GB201706854D0 (en) | Therapeutic agents for lysosomal storage disorders | |
GB201702552D0 (en) | Therapeutic agents for lysosomal storage disorders | |
EP3672590A4 (en) | Methods for inducing hematopoietic stem cell specificity | |
GB201613828D0 (en) | Therapeutic agents for lysosomal storage disorders |