CA2365532A1 - Arpe-19 as a platform cell line for encapsulated cell-based delivery - Google Patents
Arpe-19 as a platform cell line for encapsulated cell-based delivery Download PDFInfo
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- CA2365532A1 CA2365532A1 CA002365532A CA2365532A CA2365532A1 CA 2365532 A1 CA2365532 A1 CA 2365532A1 CA 002365532 A CA002365532 A CA 002365532A CA 2365532 A CA2365532 A CA 2365532A CA 2365532 A1 CA2365532 A1 CA 2365532A1
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- 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
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
- A61K9/0024—Solid, semi-solid or solidifying implants, which are implanted or injected in body tissue
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- 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
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/18—Growth factors; Growth regulators
- A61K38/185—Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
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- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
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- 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
- A61K9/0048—Eye, e.g. artificial tears
- A61K9/0051—Ocular inserts, ocular implants
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
- A61P11/06—Antiasthmatics
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
- A61P27/06—Antiglaucoma agents or miotics
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
- A61P27/12—Ophthalmic agents for cataracts
-
- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
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- 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
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- 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
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- 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
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- 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
- C12N5/0618—Cells of the nervous system
- C12N5/0621—Eye cells, e.g. cornea, iris pigmented cells
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- 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
- A61K2035/126—Immunoprotecting barriers, e.g. jackets, diffusion chambers
- A61K2035/128—Immunoprotecting barriers, e.g. jackets, diffusion chambers capsules, e.g. microcapsules
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- 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
-
- 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
Abstract
ARPE-19 cells were evaluated as a platform cell line for encapsulated and unencapsulated cell-based delivery technology. ARPE-19 cells were found to be hardy (the cell line is viable under stringent conditions, such as in central nervous system or intra-ocular environment); can be genetically modified to secrete the protein of choice; has a long life span; is of human origin; has goodin vivo device viability; delivers efficacious quantity of growth factor;
triggers no or low level host immune reaction, and is non-tumorigenic.
triggers no or low level host immune reaction, and is non-tumorigenic.
Claims (30)
1. An implantable cell culture device, the device comprising:
(a) a semipermeable membrane permitting the diffusion of a growth factor therethrough; and (b) at least one ARPE-19 cell disposed within the semipermeable membrane.
(a) a semipermeable membrane permitting the diffusion of a growth factor therethrough; and (b) at least one ARPE-19 cell disposed within the semipermeable membrane.
2. The device of claim 1, wherein the semipermeable membrane is immunoisolatory.
3. The device of claim 1, wherein the semipermeable membrane is microporous.
4. The device of claim 1, wherein the semipermeable membrane permits the diffusion of a substance of therapeutic interest
5. The device of claim 1, wherein the device further comprises a matrix disposed within the semipermeable membrane.
6. The device of claim 1, wherein the device further comprises a tether anchor.
7. The device of claim 1, wherein the ARPE-19 cell is genetically engineered.
8. The device of claim 7, wherein the ARPE-19 cell is genetically engineered to produce Green Fluorescent Protein (GFP).
9. The device of claim 7, wherein the ARPE-19 cell is genetically engineered to secrete a desired factor.
10. The device of claim 9, wherein the desired factor is ciliary neurotrophic factor (CNTF).
11. The device of claim 9, wherein the desired factor is glial cell derived neurotrophic factor (GDNF).
12. A method of delivering a desired factor to a recipient host, comprising the steps of (a) encapsulating an ARPE-19 cell within a semipermeable membrane, said membrane allowing the diffusion of the growth factor; and (b) implanting the encapsulated cell into a target region within the recipient host, such that the encapsulated ARPE-19 cell secretes the desired factor to the target region.
13. The method of claim 12, wherein the target region is the central nervous system (CNS).
14. The method of claim 12, wherein the target region is the eye.
15. The method of claim 13, wherein the target region is a subcutaneous site, an intraperitoneal site, or another implantation locations.
16. The method of claim 12, wherein the desired factor is selected from the group consisting of neurotrophins, interleukins, cytokines, anti-apoptotic, angiogenic, and anti-angiogenic factors, and antigens.
17. The method of claim 12, wherein the desired factor is selected from the group consisting of BDNF, NT-4, CNTF, Axokine, bFGF, IGF I, IGF II, TGFb II, Midkine, IL-1.beta., TNF, NGF, IL-2/3, ILF, IL-6, NTN, Neublastin, VEGF, GDNF, PDGF, LEDGF, PEDF.
18. The method of claim 12, wherein the desired factor is a therapeutic protein.
19. The method of claim 12, wherein the desired factor is ciliary neurotrophic factor (CNTF).
20. The method of claim 12, wherein the desired factor is glial cell derived neurotrophic factor (GDNF).
21. The method of claim 12, wherein the desired factor is an antigenic factor or an antibody.
22. The method of claim 12, wherein the desired factor is a gene transfer vector.
23. The method of claim 12, wherein the desired factor is a therapeutic factor for treating cancer and cancer related disorders, cardio-vascular deseases, asthma, metabolic diseases, and other relevant pathologies.
24. The method of claim 12, wherein the recipient host has a degenerative disease.
25. The method of claim 24, wherein the degenerative disease is selected from the group consisting of Parkinson's Disease, Huntington's Disease, ALS, Alzheimer's Disease, Spinal Cord Injury, Retinopathy of Prematurity, Diabetic Retinopathy, Age-Related Macular Degeneration, Glaucoma, Retinitis Pigmentosa, Cataract Formation, Retinoblastoma, Retinal Ischemia.
26. A method for treating degenerative disease, comprising implanting into the corresponding tissue of a recipient host an implantable cell culture device, the device comprising:
(a) a semipermeable membrane permitting the diffusion of a therapeutic protein therethrough; and (b) at least one ARPE-19 cell disposed within the semipermeable membrane, wherein the ARPE-19 cell is genetically engineered to secrete therapeutic protein;
wherein the device secretes a therapeutically effective amount of protein into the targeted tissue of the host receipient.
(a) a semipermeable membrane permitting the diffusion of a therapeutic protein therethrough; and (b) at least one ARPE-19 cell disposed within the semipermeable membrane, wherein the ARPE-19 cell is genetically engineered to secrete therapeutic protein;
wherein the device secretes a therapeutically effective amount of protein into the targeted tissue of the host receipient.
27. A method for treating retinal degradation, comprising implanting into the eye of a recipient host with retinosa pigmentosa an implantable cell culture device, the device comprising:
(a) a semipermeable membrane permitting the diffusion of a growth factor therethrough; and (b) at least one ARPE-19 cell disposed within the semipermeable membrane, wherein the ARPE-19 cell is genetically engineered to secrete ciliary neurotrophic factor (CNTF);
wherein the device secretes a therapeutically effective amount of CNTF into the eye of the host receipient.
(a) a semipermeable membrane permitting the diffusion of a growth factor therethrough; and (b) at least one ARPE-19 cell disposed within the semipermeable membrane, wherein the ARPE-19 cell is genetically engineered to secrete ciliary neurotrophic factor (CNTF);
wherein the device secretes a therapeutically effective amount of CNTF into the eye of the host receipient.
28. The method of claim 27, wherein the therapeutically effective amount of CNTF is between about levels between about 100-350 ng/million cells/24 hr (ng/M/d)
29. The method of claim 27, wherein the therapeutically effective amount of CNTF is about 1.62~0.4 ng/device/24 hr.
30. The method of claim 27, wherein the retinal degradation is caused by a disorder selected from the group consisting of Retinopathy of Prematurity, Diabetic Retinopathy, Age-Related Macular Degeneration, Glaucoma, Retinitis Pigmentosa, Cataract Formation, Retinoblastoma, Retinal Ischemia.
Applications Claiming Priority (5)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US12792699P | 1999-04-06 | 1999-04-06 | |
US60/127,926 | 1999-04-06 | ||
US09/543,119 | 2000-04-05 | ||
US09/543,119 US6361771B1 (en) | 1999-04-06 | 2000-04-05 | ARPE-19 as a platform cell line for encapsulated cell-based delivery |
PCT/US2000/009150 WO2000060051A2 (en) | 1999-04-06 | 2000-04-06 | Arpe-19 as a platform cell line for encapsulated cell-based delivery |
Publications (2)
Publication Number | Publication Date |
---|---|
CA2365532A1 true CA2365532A1 (en) | 2000-10-12 |
CA2365532C CA2365532C (en) | 2012-11-06 |
Family
ID=26826092
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CA2365532A Expired - Lifetime CA2365532C (en) | 1999-04-06 | 2000-04-06 | Arpe-19 as a platform cell line for encapsulated cell-based delivery |
Country Status (12)
Country | Link |
---|---|
US (2) | US6361771B1 (en) |
EP (1) | EP1171573B1 (en) |
JP (2) | JP4954374B2 (en) |
AT (1) | ATE269715T1 (en) |
AU (1) | AU773387B2 (en) |
BR (1) | BR0009562A (en) |
CA (1) | CA2365532C (en) |
DE (1) | DE60011761T2 (en) |
DK (1) | DK1171573T3 (en) |
ES (1) | ES2219335T3 (en) |
NO (1) | NO20014856D0 (en) |
WO (1) | WO2000060051A2 (en) |
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-
2000
- 2000-04-05 US US09/543,119 patent/US6361771B1/en not_active Expired - Lifetime
- 2000-04-06 AU AU43319/00A patent/AU773387B2/en not_active Expired
- 2000-04-06 JP JP2000609543A patent/JP4954374B2/en not_active Expired - Lifetime
- 2000-04-06 WO PCT/US2000/009150 patent/WO2000060051A2/en active IP Right Grant
- 2000-04-06 BR BRPI0009562-1A patent/BR0009562A/en not_active IP Right Cessation
- 2000-04-06 DE DE60011761T patent/DE60011761T2/en not_active Expired - Lifetime
- 2000-04-06 CA CA2365532A patent/CA2365532C/en not_active Expired - Lifetime
- 2000-04-06 ES ES00923144T patent/ES2219335T3/en not_active Expired - Lifetime
- 2000-04-06 EP EP00923144A patent/EP1171573B1/en not_active Expired - Lifetime
- 2000-04-06 US US09/958,040 patent/US7115257B1/en not_active Expired - Lifetime
- 2000-04-06 DK DK00923144T patent/DK1171573T3/en active
- 2000-04-06 AT AT00923144T patent/ATE269715T1/en active
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2001
- 2001-10-05 NO NO20014856A patent/NO20014856D0/en not_active Application Discontinuation
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2010
- 2010-11-16 JP JP2010256357A patent/JP2011074079A/en not_active Withdrawn
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ATE269715T1 (en) | 2004-07-15 |
NO20014856D0 (en) | 2001-10-05 |
JP2011074079A (en) | 2011-04-14 |
JP2003524621A (en) | 2003-08-19 |
CA2365532C (en) | 2012-11-06 |
AU4331900A (en) | 2000-10-23 |
EP1171573A1 (en) | 2002-01-16 |
BR0009562A (en) | 2007-05-29 |
US7115257B1 (en) | 2006-10-03 |
DK1171573T3 (en) | 2004-09-27 |
EP1171573B1 (en) | 2004-06-23 |
ES2219335T3 (en) | 2004-12-01 |
DE60011761D1 (en) | 2004-07-29 |
AU773387B2 (en) | 2004-05-27 |
JP4954374B2 (en) | 2012-06-13 |
US6361771B1 (en) | 2002-03-26 |
WO2000060051A8 (en) | 2001-04-05 |
WO2000060051A3 (en) | 2002-03-28 |
WO2000060051A2 (en) | 2000-10-12 |
DE60011761T2 (en) | 2005-08-04 |
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