CA2250478A1 - Spray-dried microparticles as therapeutic vehicles for use in gene therapy - Google Patents
Spray-dried microparticles as therapeutic vehicles for use in gene therapy Download PDFInfo
- Publication number
- CA2250478A1 CA2250478A1 CA002250478A CA2250478A CA2250478A1 CA 2250478 A1 CA2250478 A1 CA 2250478A1 CA 002250478 A CA002250478 A CA 002250478A CA 2250478 A CA2250478 A CA 2250478A CA 2250478 A1 CA2250478 A1 CA 2250478A1
- Authority
- CA
- Canada
- Prior art keywords
- microparticles
- microparticles according
- agent
- spray
- excipient
- 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
- 239000011859 microparticle Substances 0.000 title claims abstract description 51
- 238000001415 gene therapy Methods 0.000 title claims abstract description 13
- 230000001225 therapeutic effect Effects 0.000 title description 4
- 239000002245 particle Substances 0.000 claims abstract description 30
- 239000000203 mixture Substances 0.000 claims abstract description 20
- 239000000546 pharmaceutical excipient Substances 0.000 claims abstract description 19
- 239000003795 chemical substances by application Substances 0.000 claims abstract description 13
- 108090000623 proteins and genes Proteins 0.000 claims abstract description 11
- 239000003814 drug Substances 0.000 claims description 29
- 238000001694 spray drying Methods 0.000 claims description 17
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- 125000002091 cationic group Chemical group 0.000 claims description 10
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- HIHOWBSBBDRPDW-PTHRTHQKSA-N [(3s,8s,9s,10r,13r,14s,17r)-10,13-dimethyl-17-[(2r)-6-methylheptan-2-yl]-2,3,4,7,8,9,11,12,14,15,16,17-dodecahydro-1h-cyclopenta[a]phenanthren-3-yl] n-[2-(dimethylamino)ethyl]carbamate Chemical compound C1C=C2C[C@@H](OC(=O)NCCN(C)C)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HIHOWBSBBDRPDW-PTHRTHQKSA-N 0.000 description 5
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- KSXTUUUQYQYKCR-LQDDAWAPSA-M 2,3-bis[[(z)-octadec-9-enoyl]oxy]propyl-trimethylazanium;chloride Chemical compound [Cl-].CCCCCCCC\C=C/CCCCCCCC(=O)OCC(C[N+](C)(C)C)OC(=O)CCCCCCC\C=C/CCCCCCCC KSXTUUUQYQYKCR-LQDDAWAPSA-M 0.000 description 4
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- GUBGYTABKSRVRQ-XLOQQCSPSA-N Alpha-Lactose Chemical compound O[C@@H]1[C@@H](O)[C@@H](O)[C@@H](CO)O[C@H]1O[C@@H]1[C@@H](CO)O[C@H](O)[C@H](O)[C@H]1O GUBGYTABKSRVRQ-XLOQQCSPSA-N 0.000 description 3
- WQZGKKKJIJFFOK-GASJEMHNSA-N Glucose Natural products OC[C@H]1OC(O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-GASJEMHNSA-N 0.000 description 3
- 108010020346 Polyglutamic Acid Proteins 0.000 description 3
- WQZGKKKJIJFFOK-VFUOTHLCSA-N beta-D-glucose Chemical compound OC[C@H]1O[C@@H](O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-VFUOTHLCSA-N 0.000 description 3
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- WGCNASOHLSPBMP-UHFFFAOYSA-N Glycolaldehyde Chemical compound OCC=O WGCNASOHLSPBMP-UHFFFAOYSA-N 0.000 description 2
- TWRXJAOTZQYOKJ-UHFFFAOYSA-L Magnesium chloride Chemical compound [Mg+2].[Cl-].[Cl-] TWRXJAOTZQYOKJ-UHFFFAOYSA-L 0.000 description 2
- 108091034117 Oligonucleotide Proteins 0.000 description 2
- DLRVVLDZNNYCBX-UHFFFAOYSA-N Polydextrose Polymers OC1C(O)C(O)C(CO)OC1OCC1C(O)C(O)C(O)C(O)O1 DLRVVLDZNNYCBX-UHFFFAOYSA-N 0.000 description 2
- 239000000654 additive Substances 0.000 description 2
- 230000000996 additive effect Effects 0.000 description 2
- OIRDTQYFTABQOQ-KQYNXXCUSA-N adenosine Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O OIRDTQYFTABQOQ-KQYNXXCUSA-N 0.000 description 2
- 238000005054 agglomeration Methods 0.000 description 2
- 230000002776 aggregation Effects 0.000 description 2
- 239000000074 antisense oligonucleotide Substances 0.000 description 2
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- 208000006673 asthma Diseases 0.000 description 2
- 235000014633 carbohydrates Nutrition 0.000 description 2
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 description 2
- 239000012050 conventional carrier Substances 0.000 description 2
- 238000004090 dissolution Methods 0.000 description 2
- 239000000839 emulsion Substances 0.000 description 2
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- 230000035515 penetration Effects 0.000 description 2
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- 229920000642 polymer Polymers 0.000 description 2
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- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 1
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- 239000002126 C01EB10 - Adenosine Substances 0.000 description 1
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- 102000003839 Human Proteins Human genes 0.000 description 1
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- WHUUTDBJXJRKMK-VKHMYHEASA-N L-glutamic acid Chemical compound OC(=O)[C@@H](N)CCC(O)=O WHUUTDBJXJRKMK-VKHMYHEASA-N 0.000 description 1
- 229920000881 Modified starch Polymers 0.000 description 1
- 102000016943 Muramidase Human genes 0.000 description 1
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- 108010062010 N-Acetylmuramoyl-L-alanine Amidase Proteins 0.000 description 1
- 241000283973 Oryctolagus cuniculus Species 0.000 description 1
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- 108010039918 Polylysine Proteins 0.000 description 1
- 241000125945 Protoparvovirus Species 0.000 description 1
- 108700008625 Reporter Genes Proteins 0.000 description 1
- 102000007562 Serum Albumin Human genes 0.000 description 1
- 108010071390 Serum Albumin Proteins 0.000 description 1
- 241000710960 Sindbis virus Species 0.000 description 1
- 229930006000 Sucrose Natural products 0.000 description 1
- CZMRCDWAGMRECN-UGDNZRGBSA-N Sucrose Chemical compound O[C@H]1[C@H](O)[C@@H](CO)O[C@@]1(CO)O[C@@H]1[C@H](O)[C@@H](O)[C@H](O)[C@@H](CO)O1 CZMRCDWAGMRECN-UGDNZRGBSA-N 0.000 description 1
- 239000007983 Tris buffer Substances 0.000 description 1
- 241000700618 Vaccinia virus Species 0.000 description 1
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- 238000011049 filling Methods 0.000 description 1
- 229920000370 gamma-poly(glutamate) polymer Polymers 0.000 description 1
- 229920000159 gelatin Polymers 0.000 description 1
- 239000008273 gelatin Substances 0.000 description 1
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- 235000011852 gelatine desserts Nutrition 0.000 description 1
- 238000001476 gene delivery Methods 0.000 description 1
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- 238000003306 harvesting Methods 0.000 description 1
- 150000005828 hydrofluoroalkanes Chemical class 0.000 description 1
- 230000002163 immunogen Effects 0.000 description 1
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- 229910001629 magnesium chloride Inorganic materials 0.000 description 1
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- 125000001436 propyl group Chemical group [H]C([*])([H])C([H])([H])C([H])([H])[H] 0.000 description 1
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- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 description 1
- 239000003104 tissue culture media Substances 0.000 description 1
- CYRMSUTZVYGINF-UHFFFAOYSA-N trichlorofluoromethane Chemical compound FC(Cl)(Cl)Cl CYRMSUTZVYGINF-UHFFFAOYSA-N 0.000 description 1
- LENZDBCJOHFCAS-UHFFFAOYSA-N tris Chemical compound OCC(N)(CO)CO LENZDBCJOHFCAS-UHFFFAOYSA-N 0.000 description 1
- 241001529453 unidentified herpesvirus Species 0.000 description 1
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Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/007—Pulmonary tract; Aromatherapy
- A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
- A61K9/0075—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy for inhalation via a dry powder inhaler [DPI], e.g. comprising micronized drug mixed with lactose carrier particles
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/10—Dispersions; Emulsions
- A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
- A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
- A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/10—Dispersions; Emulsions
- A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
- A61K9/1277—Preparation processes; Proliposomes
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Dispersion Chemistry (AREA)
- Molecular Biology (AREA)
- Otolaryngology (AREA)
- Pulmonology (AREA)
- Biotechnology (AREA)
- Genetics & Genomics (AREA)
- Biophysics (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GB9607035.4 | 1996-04-03 | ||
| GBGB9607035.4A GB9607035D0 (en) | 1996-04-03 | 1996-04-03 | Spray-dried microparticles as therapeutic vehicles |
| GB9601379 | 1996-06-07 | ||
| WOPCT/GB96/01379 | 1996-06-07 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA2250478A1 true CA2250478A1 (en) | 1997-10-09 |
Family
ID=26308516
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA002250478A Abandoned CA2250478A1 (en) | 1996-04-03 | 1997-04-03 | Spray-dried microparticles as therapeutic vehicles for use in gene therapy |
Country Status (14)
| Country | Link |
|---|---|
| EP (1) | EP0936902B1 (cg-RX-API-DMAC7.html) |
| JP (1) | JP2000507568A (cg-RX-API-DMAC7.html) |
| AR (1) | AR007493A1 (cg-RX-API-DMAC7.html) |
| AT (1) | ATE243027T1 (cg-RX-API-DMAC7.html) |
| AU (1) | AU715512B2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA2250478A1 (cg-RX-API-DMAC7.html) |
| DE (1) | DE69722952T2 (cg-RX-API-DMAC7.html) |
| DK (1) | DK0936902T3 (cg-RX-API-DMAC7.html) |
| ES (1) | ES2201283T3 (cg-RX-API-DMAC7.html) |
| GB (1) | GB9607035D0 (cg-RX-API-DMAC7.html) |
| NO (1) | NO984462L (cg-RX-API-DMAC7.html) |
| PT (1) | PT936902E (cg-RX-API-DMAC7.html) |
| WO (1) | WO1997036578A1 (cg-RX-API-DMAC7.html) |
| ZA (1) | ZA972837B (cg-RX-API-DMAC7.html) |
Families Citing this family (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6051256A (en) | 1994-03-07 | 2000-04-18 | Inhale Therapeutic Systems | Dispersible macromolecule compositions and methods for their preparation and use |
| US6270795B1 (en) | 1995-11-09 | 2001-08-07 | Microbiological Research Authority | Method of making microencapsulated DNA for vaccination and gene therapy |
| DE69608122T3 (de) | 1995-11-09 | 2011-06-16 | Microbiological Research Authority Camr, Salisbury | Mikroverkapselte dna zur impfung und gentherapie |
| US6565885B1 (en) | 1997-09-29 | 2003-05-20 | Inhale Therapeutic Systems, Inc. | Methods of spray drying pharmaceutical compositions |
| US6309623B1 (en) | 1997-09-29 | 2001-10-30 | Inhale Therapeutic Systems, Inc. | Stabilized preparations for use in metered dose inhalers |
| US6946117B1 (en) | 1997-09-29 | 2005-09-20 | Nektar Therapeutics | Stabilized preparations for use in nebulizers |
| US6433040B1 (en) | 1997-09-29 | 2002-08-13 | Inhale Therapeutic Systems, Inc. | Stabilized bioactive preparations and methods of use |
| US20060165606A1 (en) | 1997-09-29 | 2006-07-27 | Nektar Therapeutics | Pulmonary delivery particles comprising water insoluble or crystalline active agents |
| GB9810236D0 (en) | 1998-05-13 | 1998-07-08 | Microbiological Res Authority | Improvements relating to encapsulation of bioactive agents |
| US6406719B1 (en) | 1998-05-13 | 2002-06-18 | Microbiological Research Authority | Encapsulation of bioactive agents |
| US7871598B1 (en) | 2000-05-10 | 2011-01-18 | Novartis Ag | Stable metal ion-lipid powdered pharmaceutical compositions for drug delivery and methods of use |
| PT1280520E (pt) | 2000-05-10 | 2014-12-16 | Novartis Ag | Pós à base de fosfolípidos para administração de fármacos |
| US7575761B2 (en) | 2000-06-30 | 2009-08-18 | Novartis Pharma Ag | Spray drying process control of drying kinetics |
| WO2002015880A2 (en) * | 2000-08-25 | 2002-02-28 | Merck Patent Gmbh | Powdered mannitol and mannitol-containing compositions |
| PT1458360E (pt) | 2001-12-19 | 2011-07-13 | Novartis Ag | Derivados de indole como agonistas do recetor s1p1 |
| US9339459B2 (en) | 2003-04-24 | 2016-05-17 | Nektar Therapeutics | Particulate materials |
| EP1765294B1 (en) | 2004-05-12 | 2008-09-24 | Baxter International Inc. | Nucleic acid microspheres, production and delivery thereof |
| CA2566199C (en) | 2004-05-12 | 2013-10-22 | Baxter International Inc. | Delivery of as-oligonucleotide microspheres to induce dendritic cell tolerance for the treatment of autoimmune type 1 diabetes |
| EP1834651A1 (en) * | 2006-03-15 | 2007-09-19 | Universiteit Gent | Compositions and methods for veterinary vaccination |
| CN101500616A (zh) | 2006-08-04 | 2009-08-05 | 巴克斯特国际公司 | 预防和/或逆转新发作自身免疫糖尿病的基于微球的组合物 |
| CN101686939B (zh) | 2007-04-17 | 2013-03-27 | 巴克斯特国际公司 | 用于肺部投送的核酸微粒 |
| DE102009033196A1 (de) * | 2009-07-14 | 2011-01-20 | Beiersdorf Ag | Kosmetische Zubereitung mit einem Gehalt an dispergierten Mikrosphären |
| JP7422977B2 (ja) * | 2018-07-23 | 2024-01-29 | トランスレイト バイオ， インコーポレイテッド | メッセンジャーｒｎａ用乾燥粉末製剤 |
| AU2020206984A1 (en) * | 2019-01-09 | 2021-07-15 | Ziccum Ab | Stabilized non-enveloped virus compositions |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB9107628D0 (en) * | 1991-04-10 | 1991-05-29 | Moonbrook Limited | Preparation of diagnostic agents |
| CA2115065C (en) * | 1992-06-12 | 2000-10-03 | Kiyoyuki Sakon | Ultrafine particle powder for inhalation and method for production thereof |
| KR100208979B1 (ko) * | 1992-06-12 | 1999-07-15 | 야스이 쇼사꾸 | 기도내 투여용 제제 |
| AU6637394A (en) * | 1993-04-19 | 1994-11-08 | Medisorb Technologies International L.P. | Long-acting treatment by slow-release delivery of antisense oligodeoxyribonucleotides from biodegradable microparticles |
| MX9605717A (es) * | 1994-05-18 | 1998-05-31 | Inhale Therapeutic Syst | Metodos y composiciones para la formulacion de polvo seco de interferones. |
| NZ292980A (en) * | 1994-09-29 | 1999-02-25 | Andaris Ltd | Smooth, spherical water-soluble microparticles as therapeutic or diagnostic vehicles |
| WO1996027393A1 (en) * | 1995-03-07 | 1996-09-12 | University Of Pittsburgh | A dry powder formulation for gene therapy |
| EP0840623B1 (en) * | 1995-07-21 | 2007-07-18 | Brown University Research Foundation | Compositions for gene therapy comprising nucleic acid loaded polymeric microparticles |
-
1996
- 1996-04-03 GB GBGB9607035.4A patent/GB9607035D0/en active Pending
-
1997
- 1997-04-03 ZA ZA9702837A patent/ZA972837B/xx unknown
- 1997-04-03 DK DK97915601T patent/DK0936902T3/da active
- 1997-04-03 DE DE69722952T patent/DE69722952T2/de not_active Expired - Fee Related
- 1997-04-03 EP EP97915601A patent/EP0936902B1/en not_active Expired - Lifetime
- 1997-04-03 PT PT97915601T patent/PT936902E/pt unknown
- 1997-04-03 AT AT97915601T patent/ATE243027T1/de not_active IP Right Cessation
- 1997-04-03 AR ARP970101333A patent/AR007493A1/es not_active Application Discontinuation
- 1997-04-03 WO PCT/GB1997/000953 patent/WO1997036578A1/en not_active Ceased
- 1997-04-03 ES ES97915601T patent/ES2201283T3/es not_active Expired - Lifetime
- 1997-04-03 CA CA002250478A patent/CA2250478A1/en not_active Abandoned
- 1997-04-03 AU AU23020/97A patent/AU715512B2/en not_active Ceased
- 1997-04-03 JP JP9535066A patent/JP2000507568A/ja active Pending
-
1998
- 1998-09-25 NO NO984462A patent/NO984462L/no not_active Application Discontinuation
Also Published As
| Publication number | Publication date |
|---|---|
| GB9607035D0 (en) | 1996-06-05 |
| DE69722952D1 (de) | 2003-07-24 |
| DK0936902T3 (da) | 2003-10-06 |
| PT936902E (pt) | 2003-11-28 |
| WO1997036578A1 (en) | 1997-10-09 |
| AR007493A1 (es) | 1999-11-10 |
| ATE243027T1 (de) | 2003-07-15 |
| DE69722952T2 (de) | 2003-12-11 |
| EP0936902A1 (en) | 1999-08-25 |
| EP0936902B1 (en) | 2003-06-18 |
| ZA972837B (en) | 1998-04-03 |
| AU715512B2 (en) | 2000-02-03 |
| AU2302097A (en) | 1997-10-22 |
| NO984462D0 (no) | 1998-09-25 |
| JP2000507568A (ja) | 2000-06-20 |
| NO984462L (no) | 1998-09-25 |
| ES2201283T3 (es) | 2004-03-16 |
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| EEER | Examination request | ||
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