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Last Updated: April 19, 2024

Claims for Patent: 5,861,488

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Summary for Patent: 5,861,488

Title:	Compositions and methods for treating sickle cell disease
Abstract:	Gene therapy methods and compositions for high level expression of anti-sickling globin proteins in erythroid cells for treating Sickle Cell disease are described.
Inventor(s):	LeBoulch; Philippe (Cambridge, MA), London; Irving M. (Cambridge, MA)
Assignee:	Massachusetts Institute of Technology (Cambridge, MA)
Application Number:	08/935,648
Patent Claims:	1. An anti-sickling .beta.-globin protein having structure of human .beta.-globin and comprising the amino acid sequence shown in SEQ ID NO: 1, wherein at least one amino acid residue of SEQ ID NO: 1 has been substituted with an anti-sickling amino acid residue selected from the group consisting of: 9 Thr, 12 Asn, 22 Ala, 50 Ser, 80 Asp, 86 Ser, 87 Gln, 116 Arg, 117 Asn, 125 Gln and 126 Met, provided that said anti-sickling .beta.-globin protein does not contain all of said anti-sickling residues, wherein said anti-sickling .beta.-globin protein exhibits reduced sickling compared to wild-type .beta.-globin protein when present in a hemoglobin tetramer along with at least one .beta. 6 Val mutated .beta.-globin protein. 2. The anti-sickling .beta.-globin protein of claim 1 wherein said anti-sickling amino acid residue is selected from the group consisting of; 9 Thr, 12 Asn, 22 Ala, 50 Ser, 86 Ser, 87 Gln, 116 Arg, and 117 Asn, 125 Gln and 126 Met. 3. The anti-sickling .beta.-globin protein of claim 1 having the amino acid sequence shown in SEQ ID NO:2. 4. The anti-sickling .beta.-globin protein of claim 1 produced recombinantly in a host cell transformed with an expression vector comprising a nucleic acid encoding the protein. 5. The anti-sickling .beta.-globin protein of claim 4 wherein said host cell is an erythroid cell. 6. The anti-sickling .beta.-globin protein of claim 4 wherein said expression vector further comprises a promoter recognized by a DNA or an RNA polymerase. 7. The anti-sickling .beta.-globin protein of claim 6 wherein said DNA or RNA polymerase is erythroid-specific and wherein said host cell is an erythroid cell. 8. The anti-sickling .beta.-globin protein of claim 6 wherein said promoter is selected from the group consisting of the .beta.-globin promoter and the HPFH promoter. 9. The anti-sickling .beta.-globin protein of claim 6 wherein said expression vector further comprises an enhancer. 10. The anti-sickling .beta.-globin protein of claim 9 wherein said enhancer is the DNAse I hypersensitive site 2 of the human .beta. Locus Control Region and/or the second intron of the .beta.-globin gene. 11. The anti-sickling .beta.-globin protein of claim 4 further comprising a selectable marker.

Details for Patent 5,861,488

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Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Approval Date	Patent No.	Expiredate
Merck Sharp & Dohme Corp.	INTRON A	interferon alfa-2b	For Injection	103132	06/04/1986	⤷ Try a Trial	2014-09-19
Merck Sharp & Dohme Corp.	INTRON A	interferon alfa-2b	For Injection	103132		⤷ Try a Trial	2014-09-19
Merck Sharp & Dohme Corp.	INTRON A	interferon alfa-2b	Injection	103132		⤷ Try a Trial	2014-09-19
>Applicant	>Tradename	>Biologic Ingredient	>Dosage Form	>BLA	>Approval Date	>Patent No.	>Expiredate

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