Recombinant α-galactosidase A therapy for Fabry disease

Details Recombinant α-galactosidase A therapy for Fabry disease Recombinant α-galactosidase A therapy for Fabry disease

2006-03-14

2006-03-14

Wax, Robert A. (Department: 1653)

Drug, bio-affecting and body treating compositions

Enzyme or coenzyme containing

Hydrolases

C435S208000, C435S320100

Reexamination Certificate

active

07011831

ABSTRACT:
Fabry disease results from an X-linked deficiency in the enzyme α-galactosidase A. The present invention is directed to recombinant human α-galactosidase A and provides baculovirus expression vectors and recombinant virus that provide stable expression of extracellular and intracellular levels of this enzyme in an insect cell culture. The recombinant-derived enzyme can be used in enzyme replacement therapy to treat Fabry patients. Composition useful in therapeutic administration of α-galactosidase A are also provided.

REFERENCES:
patent: 4497797 (1985-02-01), Ebata et al.
patent: 5179023 (1993-01-01), Calhoun et al.
patent: 5356804 (1994-10-01), Desnick et al.
patent: 5401650 (1995-03-01), Desnick et al.
patent: 5658567 (1997-08-01), Calhoun et al.
Bishop, D..F. ,et al. ,“Affinity Purification of alpha-Galactosidase A from Human Spleen, Placenta, and Plasma with Elimination of Pyrogen Contamination”,The Journal of Biological Chemistry, 256(3), (Feb. 10, 1981),1307-1316.
Bishop, D..F. ,et al. ,“Human alpha-galactosidase A: Nucleotide Sequence of a cDNA Clone Encoding the Mature Enzyme”,Proc. Natl. Acad. Sci. USA, 83, (Jul. 1986),4859-4863.
Bishop, D..F. ,et al. ,“Structural organization of the human alpha-galactosidase A gene: Further evidence for the absence of a 3' untranslated region”,Proc. Natl. Acad. Sci. USA, 85,(Jun. 1988),pp. 3903-3907.
Brady, R..O. ,et al. ,“Replacement Therapy For Inherited Enzyme Deficiency”,The New England Journal Of Medicine, 289(1), (Jul. 1973),pp. 9-14.
Butters, T..D. ,et al. ,“Steps in the Biosynthesis of Mosquito Cell Membrane Glycoproteins and the Effects of Tunicamycin”,Biochemica et Biophysica Acta, 640, (1981),672-686.
Calhoun, D..H. ,et al. ,“Fabry Disease: Isolation of a cDNA Clone Encoding Human alpha-galactosidase A”,Proc. Natl. Acad. Sci. USA, 82, (Nov. 1985),7364-7368.
Coppola, G..,et al. ,“Characterization of Glycosylated and Catalytically Active Recombinant Human alpha-Galactosidase A Using a Baculovirus Vector”,Gene, 144, (1994), 197-203.
Davidson, D..J. , et al. ,“alpha-Mannosidase-Catalyzed Trimming of High-Mannose Glycans in Noninfected and Baculovirus-InfectedSpodoptera frugiperdaCells (IPLB-SF-21AE). A Possible Contributing Regulatory Mechanism for Assembly of Complex-Type Oligosaccharides in Infected Cells”,Biochemistry,30, (Oct. 15, 1991),9811-9815.
Davidson, D..J. , et al. ,Asparagine-Linked Oligosaccharide Processing in Lepidopteran Insect Cells. Temporal Dependence of the Nature of the Oligosaccharides Assemmbled on Asparagine-289 of Recombinant Human.
Plasminogen Produced in Baculovirus Vector InfectedSpodoptera, Biochemistry,30, (1991),6167-6174.
Davidson, D..J. ,et al. ,“Oligosaccharide Processing in the Expression of Human Plasminogen cDNA by Lepidopteran Insect (Spodoptera Frugiperda) Cells”,Biochemistry,29, (1990),5584-5590.
Davidson, D..J. ,et al. ,“Oligosaccharide Structures Present on Asparagine-289 of Recombinant Human Plasminogen Expressed in a Chinese Hamster Ovary Cell Line”,Biochemistry, 30, (1991),625-633.
Davidson, D..J. ,et al. ,“Plasminogen Activator Activities of Equimolar Complexes of Streptokinase with Variant Recombinant Plasminogens”,Biochemistry, 29, (1990),3585-3590.
Desnick, Robert.J. ,et al. ,“Enzyme therapy in Fabry disease: Differential in vivo plasma clearance and metabolic effectiveness of plasma and splenic alpha-galactosidase A isozymes”,Proc. of the Nat'l Acad. of Sci, USA,76(10), (Oct. 1979),5326-5330.
Desnick, R..J. ,et al. ,“Fabry Disease: alpha-Galactosidase Deficiency; Schindler Disease: alpha-N-Acetylgalactosaminidase Deficiency”,The Metabolic Basis of Inherited Disease II, Sixth Edition,(1989),pp. 1751-1796.
ENG, C..M. ,et al. ,“Fabry disease: twenty-three mutations including sense and antisense CpG alterations and identification of a deletional hot-spot in the alpha-galactosidase A gene”,Human Molecular Genetics, 3(10), (1994),pp. 1795-1799.
Goochee, C..F. ,et al. ,“Environmental Effects on Protein Glycosylation”,Bio/Technology, 8, (May 1990),421-427.
Goto, M..,et al. ,“Production of Recombinant Human Erythropoietin in Mammalian Cells: Host-Cell Dependency of the Biological Activity of the Cloned Glycoprotein”,Bio/Technology, 6, (1988),67-71.
Greenfield, C..,et al. ,“Expression of the Human EGF Receptor with Ligand-Stimulatable Kinase Activitive in Insect Cells Using a Baculovirus Vector”,The EMBO Journal, 7, (1988), 139-146.
Hantzopoulos, P..A. ,et al. ,“Expression of the Human alpha-Galactosidase A inEscherichia ColiK-12”,Gene, 57, (1987),159-169.
Hantzopoulos, Petros.A. ,“Molecular Cloning and Expression inE. coliof the human alpha-galactosidase A gene”,Dissertations Abstracts International, 48(5), (Nov. 1987),p. 1250.
Jarvis, D..L. ,et al. ,“Glycosylation and Secretion of Human Tissue Plasminogen Activator in Recobinant Baculovirus-Infected Insect Cells”,Molecular and Cellular Biology, 9, (Jan. 1989),214-223.
Kuroda, K..,et al., “Expression of the Influenza Virus Haemagglutinin in Insect Cells by a Baculovirus Vector”,The EMBO Journal, 5, (1986), 1359-1365.
Luckow, V..A. ,et al. ,“Trends in the Development of Baculovirus Expression Vectors”,Bio/Technology, 6, (Jan. 1988), 47-55.
Mapes, C..A. ,et al. ,“Enzyme Replacement in Fabry's Disease, an Inborn Error of Metabolism”,Science, 169(3949), Sep. 1970),pp. 987-989.
Martin, B..M. ,et al. ,“Glycosylation and Processing of High Levels of Active Human Glucocerebrosidase in Invertebrate Cells Using a Baculovirus Expression Vector”,DNA, 7, (1988),99-106.
Meaney, C..,et al. ,“A nonsense mutation (R220X) in the alpha-galactosidase A gene detected in a female carrier of Fabry disease”,Human Molecular Genetics, 3(6), (1994),pp. 1019-1020.
Miller, L..K. ,“Baculoviruses as Gene Expression Vectors”,Ann. Rev. Microbiol., 42, (1988),177-199.
Miyamura, N..,et al. ,“A Carboxy-terminal Truncation of Human alpha-Galactosidase A in a Heterozygous Female with Fabry Disease and Modification of the Enzymatic Activity by the Carboxy-terminal Domain”,J. Clin. Invest., The American Society for Clinical Investigation, Inc., 98(8), (Oct. 1996),pp. 1809-1817.
Nagao, Y..,et al. ,“Hypertrophic cardiomyopathy in late-onset variant of Fabry disease with high residual activity of alpha-galactosidase A”,Clinical Genetics, 39, (1991),pp. 233-237.
Nakao, S..,et al. ,“An Atypical Variant of Fabry's Disease in Men with Left Ventricular Hypertrophy”,The New England Journal of Medicine,333(5), (Aug. 1995),pp. 288-293.
Page, M..J. ,“p36C: An Improved Baculovirus Expression Vector for Producing High Levels of Mature Recombinant Proteins”,Nucleic Acids Research, 17(1), (1989),2 pages.
Quinn, M..,et al. ,“A Genomic Clone Containing the Promoter for the Gene Encoding the Human Lysosomal Enzyme, alpha-Galactosidase A”,Gene, 58, (1987),177-188.
Rademacher, T..W. ,et al. ,“Glycobiology”,Ann Rev. Biochem, 57, (1988),785-838.
Rankin, C..,et al. ,“Eight Base Pairs Encompassing the Transcriptional Start Point are the Major Determinant for Baculovirus Polyhedrin Gene Expression”,Gene, 70, (1988),39-49.
Ryan, R..O. ,et al. ,“Arylphorin fromManduca sexta:Carbohydrate Structure and Immunological Studies”,Archieves of Biochemistry and Biophysics, 243, (1985),115-124.
Sakuraba, H..,et al. ,“Identification of Point Mutations in the alpha-Galatosidase A Gene in Classical and Atypical Hemizygotes with Fabry Disease”,The American Journal of Human Genetics, 47(5), (Nov. 1990),pp. 784-786.
Scheidt, W..,et al. ,“Brief Report. An Atypical Variant Of Fabry's Disease With Manifestations Confined To The Myocardium”,New England Journal of Medicine, 324(6), (Feb 1991),pp. 395-399.
Shear

Affiliated with

Also associated with

Rating

Recombinant α-galactosidase A therapy for Fabry disease does not yet have a rating. At this time, there are no reviews or comments for this patent.

If you have personal experience with Recombinant α-galactosidase A therapy for Fabry disease, we encourage you to share that experience with our LandOfFree.com community. Your opinion is very important and Recombinant α-galactosidase A therapy for Fabry disease will most certainly appreciate the feedback.

Rate now

Comments { 0 }

Profile ID: LFUS-PAI-O-3592006