Pharmaceutical composition for treatment of duchenne muscular dystrophy
원문보기
IPC분류정보
국가/구분
United States(US) Patent
등록
국제특허분류(IPC7판)
C07H-021/04
C12Q-001/68
C12P-019/34
출원번호
US-0917907
(2001-07-31)
우선권정보
JP-0140930 (1999-05-21)
발명자
/ 주소
Matsuo, Masafumi
출원인 / 주소
JCR Pharmaceuticals Co., Ltd., Matsuo
Masafumi
대리인 / 주소
Greenblum & Bernstein P.L.C.
인용정보
피인용 횟수 :
57인용 특허 :
7
초록▼
A therapeutic pharmaceutical composition for patients of Duchenne muscular dystrophy with entire loss of exon 20 in dystrophin mature mRNA is provided. The composition comprise as an active principle an antisense oligonucleotide consisting of a 20 to 50-nucleotide sequence against exon 19 of the dys
A therapeutic pharmaceutical composition for patients of Duchenne muscular dystrophy with entire loss of exon 20 in dystrophin mature mRNA is provided. The composition comprise as an active principle an antisense oligonucleotide consisting of a 20 to 50-nucleotide sequence against exon 19 of the dystrophin pre-mRNA.
대표청구항▼
1. A method of manufacturing a composition, the method comprising: forming an antisense oligonucleotide consisting of a nucleotide sequence fully complementary to the sequence represented by SEQ ID NO: 1; dissolving the antisense oligonucleotide in a pharmaceutically acceptable injectable medium
1. A method of manufacturing a composition, the method comprising: forming an antisense oligonucleotide consisting of a nucleotide sequence fully complementary to the sequence represented by SEQ ID NO: 1; dissolving the antisense oligonucleotide in a pharmaceutically acceptable injectable medium to form a solution; and sterilizing the solution to form a composition for intravenous administration. 2. The method of claim 1, wherein the antisense oligonucleotide comprises oligoDNA, phosphorothioate oligoDNA, or phosphorothioate oligoRNA. 3. A method of manufacturing a composition comprising: forming an antisense oligonucleotide consisting of a nucleotide sequence fully complementary to the sequence represented by SEQ ID NO: 2; dissolving the antisense oligonucleotide in a pharmaceutically acceptable injectable medium to form a solution; and sterilizing the solution to form a composition for intravenous administration. 4. The method of claim 3, wherein the antisense oligonucleotide comprises oligoDNA, phosphorothioate oligoDNA, or phosphorothioate oligoRNA. 5. A composition comprising: in a pharmaceutically acceptable injectable medium, an antisense oligonucleotide consisting of a nucleotide sequence fully complementary to the nucleotide sequence represented by SEQ ID NO: 1 or SEQ ID NO: 2. 6. The composition of claim 5, wherein the antisense oligonucleotide comprises oligoDNA, phosphorothioate oligoDNA, or phosphorothioate oligoRNA. 7. The composition of claim 5, comprising 0.05-5 μmol/mL of the oligonucleotide, 0.02-10 w/v % of at least one carbohydrate or polyalcohol, and 0.0 1-0.4 w/v % of at least one pharmaceutically acceptable surfactant. 8. The composition of claim 7, wherein the at least one of carbohydrate or polyalcohol comprises carbohydrate comprising monosaccharides or disaccharides. 9. The composition of claim 7, wherein the at least one of carbohydrate or polyalcohol comprises glucose, galactose, mannose, lactose, maltose, mannitol, or sorbitol. 10. The composition of claim 7, wherein the pharmaceutically acceptable surfactant comprises polyoxyethylene sorbitan mono- to tri-ester, alkyl phenyl polyoxyethylene, sodium taurocholate, sodium cholate, or polyalcohol ester. 11. The composition of claim 7, wherein the pharmaceutically acceptable surfactant comprises polyoxyethylene sorbitan ester comprising oleate, laurate, stearate, or palmitate. 12. The composition of claim 5, further comprising 0.03-0.09 M of at least one pharmaceutically neutral salt. 13. The composition of claim 12, wherein the neutral salt is selected from at least one of sodium chloride, potassium chloride, or calcium chloride. 14. The composition of claim 5, further comprising 0.002-0.05 M of a pharmaceutically acceptable buffering agent. 15. The composition of claim 14, wherein the buffering agent is selected from at least one of sodium citrate, sodium glycinate, sodium phosphate, or tris(hydroxymethyl) aminomethane. 16. The composition of claim 5, wherein the composition is in lyophilized form.
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