| 序号 | 专利名 | 申请号 | 申请日 | 公开（公告）号 | 公开（公告）日 | 发明人 |
|---|---|---|---|---|---|---|
| 161 | 중합체인자 ＩＸ 부분의 접합체 | KR1020117005370 | 2005-06-30 | KR101146160B1 | 2012-07-16 | 보사드메리제이; 스티븐슨게일 |
| 본 발명은 인자 IX 부분과 하나 이상의 수용성 중합체의 접합체를 제공한다. 전형적으로, 수용성 중합체는 폴리(에틸렌 글리콜) 또는 그의 유도체이다. 다른 것들 중에서도, 상기 접합체를 포함하는 조성물, 상기 접합체의 제조 방법, 및 상기 접합체를 포함하는 조성물을 환자에게 투여하는 방법 또한 제공된다.
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| 162 | 낮은 수준의 수용성 중합체를 포함하는 개질된 혈액 인자 | KR1020117011049 | 2009-10-16 | KR1020110071012A | 2011-06-27 | 투레켓피터; 시에크만위르겐; 로텐스테이너한스피터 |
| 본 발명은 일반적으로 혈액 인자에 콘주게이션된 낮은 수준의 수용성 중합체 분자를 갖지만, 보다 다수의 수용성 중합체 잔기를 갖는 분자와 유사한 또는 그보다 우수한 생물학적 활성을 나타내는 개질된 혈액 인자의 제조 방법 및 그를 위한 물질에 관한 것이다. | ||||||
| 163 | 재조합에 의해 제조된 인간 인자 Ⅷ 및 Ⅸ | KR1020117003725 | 2009-08-21 | KR1020110057131A | 2011-05-31 | 산드베르그헬레나; 스텐룬드페테르; 슈뢰더카롤라; 까사데문트엘리자베스; 타이메이어마야 |
| 인간 글리코실화 패턴을 갖는 재조합 인간 인자 VIII 또는 IX 단백질에 관한 것이고, 상기 단백질은 N-글리콜릴뉴라민산 및/또는 탄수화물기 Galα-3Gal을 갖지 않는다.
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| 164 | 중합체인자 ＩＸ 부분의 접합체 | KR1020117005370 | 2005-06-30 | KR1020110030712A | 2011-03-23 | 보사드메리제이; 스티븐슨게일 |
| 본 발명은 인자 IX 부분과 하나 이상의 수용성 중합체의 접합체를 제공한다. 전형적으로, 수용성 중합체는 폴리(에틸렌 글리콜) 또는 그의 유도체이다. 다른 것들 중에서도, 상기 접합체를 포함하는 조성물, 상기 접합체의 제조 방법, 및 상기 접합체를 포함하는 조성물을 환자에게 투여하는 방법 또한 제공된다.
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| 165 | 제ＩＸ인자의 부위-지정 변형 | KR1020107025563 | 2009-04-15 | KR1020110015551A | 2011-02-16 | 브룩스,알란,알.; 머피,존,이.; 세토,마리안; 지앙,샤오캬오; 키유리치,데이비드; 파텔,찬드라 |
| 본 발명은 변형된 제IX인자 폴리펩티드 예컨대 하나 이상의 시스테인 부위가 도입된 제IX인자 폴리펩티드에 관한 것이다. 변형된 제IX인자 폴리펩티드는 생체적합성 중합체에 접합될 수 있다. 또한 본 발명은 변형된 제IX인자 폴리펩티드의 제조 방법, 및 변형된 제IX인자 폴리펩티드의 사용 방법, 예를 들어, B형 혈우병에 걸린 환자를 치료하기 위해 사용하는 방법에 관한 것이다.
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| 166 | 아데노-결합 바이러스 (ＡＡＶ) 서열을 검출 및/또는 확인하는 방법 및 그 방법에 의해 확인된 신규한 서열을 분리하는 방법 | KR1020107001837 | 2002-11-12 | KR101014207B1 | 2011-02-14 | 가오권핑; 윌슨제임스엠; 알비라마우리시오 |
| 본 발명에서는 조직 또는 세포로부터 얻어진 DNA의 샘플에서 AAV 서열을 검출하고 분리하기 위한 방법이 제공된다. 본 발명은 또한 이 방법, 이들 서열을 사용하여 구성된 벡터들을 사용하여 확인되는 AAV 서열을 제공한다.
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| 167 | 화학적으로 변형된 제 ９ 인자 | KR1020107014071 | 2008-12-19 | KR1020100110799A | 2010-10-13 | 투레첵페테르; 샤이프링거프리드리히 |
| 본 발명은 활성화 펩티드 영역이 공유 결합형 커플링된 수용성 친수성 중합체를 함유하는 화학적으로 변형된 FIX를 게재한다. | ||||||
| 168 | 트랜스펙션 및 면역자극을 위한 ＲＮＡ 및 양이온성 펩타이드의 복합체 | KR1020107007226 | 2008-09-04 | KR1020100085020A | 2010-07-28 | 포틴-믈레크제크,마리올라; 바움호프,패트릭 |
| The present invention relates to a complexed RNA, comprising at least one RNA complexed with one or more oligopeptides, wherein the oligopeptide, which has the function of cell-penetrating peptide (CPP), has a length of 8 to 15 amino acids and has the empirical formula (Arg);(;Lys);(His);(Om); (Xaa)with the majority of residues being selected from Arg, Lys, His, Om. The invention further relates to a method for transfecting a cell or an organism, thereby applying the inventive complexed RNA. Additionally, pharmaceutical compositions and kits comprising the inventive complexed RNA, as well as the use of the inventive complexed RNA for transfecting a cell, tissue or an organism and/or for modulating, preferably inducing or enhancing, an immune response are disclosed herein. | ||||||
| 169 | 인간 세포주에서 재조합 인간 단백질의 무혈청의 안정한형질감염 및 생산 | KR1020077030563 | 2006-06-29 | KR1020080028891A | 2008-04-02 | 슈뢰더카롤라; 베그만카틀린; 딩하이얜 |
| The present invention relates to an improved method for the serum-free production of an immortalized human cell line stably transfected under serum-free conditions with a specific vector carrying the gene coding for the protein of interest. Furthermore the invention relates to a production cell line obtained by said method, a production method for said protein of interest utilizing said production cell line, and the specific vector carrying the gene of interest itself. | ||||||
| 170 | 혈액 응고 인자의 조직-특이적 발현을 위한 ＤＮＡ-작제물 | KR1020000022862 | 2000-04-28 | KR100681719B1 | 2007-02-15 | 네그리에끌로드; 로드리귀에마리엘렌느; 엔졸라나딸리 |
| 혈액 응고 인자의 아미노산 서열을 암호화하는 DNA 및 조혈세포에서의 발현에 특이적인 프로모터를 암호화하는 DNA를 포함하는, 제9인자와 같은 혈액 응고 인자의 조직-특이적 발현에 적합한 DNA-작제물이 개시되어 있다. 혈액 응고 인자, 조혈세포, 제9인자, 사람 혈소판 당단백질 IIb, 형질감염, 포볼-12-미리스테이트-13-아세테이트 (PMA) | ||||||
| 171 | 글리코페질화 인자 나인 | KR1020067010603 | 2004-12-03 | KR1020060123224A | 2006-12-01 | 디프리스,숀; 베이어,로버트,제이.; 바우어,캐린; 판니어셀밤,크리시나사미 |
| The present invention provides conjugates between Factor IX and PEG moieties. The conjugates are linked via an intact glycosyl linking group interposed between and covalently attached to the peptide and the modifying group. The conjugates are formed from glycosylated peptides by the action of a glycosyltransferase. The glycosyltransferase ligates a modified sugar moiety onto a glycosyl residue on the peptide. Also provided are methods for preparing the conjugates, methods for treating various disease conditions with the conjugates, and pharmaceutical formulations including the conjugates. | ||||||
| 172 | 아데노-결합 바이러스 (ＡＡＶ) 서열을 검출 및/또는확인하는 방법 및 그 방법에 의해 확인된 신규한 서열을분리하는 방법 | KR1020047007245 | 2002-11-12 | KR1020050058234A | 2005-06-16 | 가오권핑; 윌슨제임스엠; 알비라마우리시오 |
| A method for detecting and isolating AAV sequences in a sample of DNA obtained from tissue or cells is provided. The invention further provides AAV sequences identified by this method, and vectors constructed using these sequences. | ||||||
| 173 | 혈액 응고 인자의 조직-특이적 발현을 위한 ＤＮＡ-작제물 | KR1020000022862 | 2000-04-28 | KR1020010020795A | 2001-03-15 | 네그리에끌로드; 로드리귀에마리엘렌느; 엔졸라나딸리 |
| PURPOSE: Disclosed is a new DNA structure for tissue-specific expression of a blood coagulation factor consisting of the DNA encoding the amino acid sequence of the blood coagulation factor and the DNA encoding a promotor specific to the expression in hematopoietic cells, and useful for the genetic treatment of hemophilia B or the like. CONSTITUTION: The new DNA structure for tissue-specific expression of a blood coagulation factor consists of the DNA encoding the amino acid sequence of the blood coagulation factor and the DNA encoding a promotor specific to the expression in hematopoietic cells, and it is useful for the genetic treatment of hemophilia B or the like. The DNA structure is obtained by inserting an promotor which consists of the DNA encoding the amino acid sequence of a blood coagulation factor consisting of the cDNA encoding blood coagulation IX factor or the like, and the DNA encoding human platelet glycoprotein IIb(GPIIb) or the like, and which is specific to the expression in hematopoietic cells, into an appropriate expression vector. | ||||||
| 174 | FACTOR IX GENE THERAPY | US15525836 | 2015-11-12 | US20190070271A1 | 2019-03-07 | Amit Nathwani; Jenny Mcintosh; Nishil Patel |
| The invention relates to a new, more potent, coagulation factor IX (FIX) expression cassette for gene therapy of haemophilia B (HB). Disclosed is a vector for expressing factor IX protein, the vector comprising a promoter, a nucleotide sequence encoding for a functional factor IX protein and an intron sequence, wherein the intron sequence is positioned between exon 1 and exon 2 of the nucleotide sequence encoding for a functional factor IX protein, and wherein the intron sequence has at least 80% identity to the sequence of SEQ ID NO. 1 as disclosed herein. | ||||||
| 175 | RECOMBINANT PROMOTERS AND VECTORS FOR PROTEIN EXPRESSION IN LIVER AND USE THEREOF | US16058808 | 2018-08-08 | US20180344877A1 | 2018-12-06 | Christopher B. Doering; H. Trent Spencer; Harrison C. Brown |
| Disclosed herein are recombinant viral vectors comprising a liver specific promotor in operable combination with a heterologous nucleic acid sequence encoding a protein, such as a clotting factor. Methods of treating a subject with a clotting disorder, such as hemophilia A or hemophilia B, are also provided. | ||||||
| 176 | Factor IX variants with clotting activity in absence of their cofactor and their use for treating bleeding disorders | US13001187 | 2009-07-28 | US10125357B2 | 2018-11-13 | Erhard Seifried; Jörg Schüttrumpf |
| The present invention relates to variants of a vitamin K-dependent serine protease of the coagulation cascade, preferably variants of factor IX (F.IX), wherein the variant is characterized in that it has clotting activity in absence of its cofactor. The present invention furthermore relates to the use of these variants for the treatment and/or prophylaxis of bleeding disorders, in particular hemophilia A and/or hemophilia B or hemophilia caused or complicated by inhibitory antibodies to F.VIII. The present invention also relates to further variants of factor IX (F.IX) which have desired properties and can, thus be tailored for respective specific therapeutic applications. | ||||||
| 177 | Complexes of RNA and cationic peptides for transfection and for immunostimulation | US13904868 | 2013-05-29 | US10111967B2 | 2018-10-30 | Mariola Fotin-Mleczek; Patrick Baumhof |
| The present invention relates to a complexed RNA, comprising at least one RNA complexed with one or more oligopeptides, wherein the oligopeptide, which has the function of cell-penetrating peptide (CPP), has a length of 8 to 15 amino acids and has the empirical formula (Arg)l;(Lys)m;(His)n;(Om)o;(Xaa)x with the majority of residues being selected from Arg, Lys, His, Om. The invention further relates to a method for transfecting a cell or an organism, thereby applying the inventive complexed RNA. Additionally, pharmaceutical compositions and kits comprising the inventive complexed RNA, as well as the use of the inventive complexed RNA for transfecting a cell, tissue or an organism and/or for modulating, preferably inducing or enhancing, an immune response are disclosed herein. | ||||||
| 178 | METHODS AND COMPOSITIONS FOR MODIFIED FACTOR IX FUSION PROTEINS | US15769252 | 2016-10-19 | US20180305439A1 | 2018-10-25 | Tal Kafri |
| The present invention provides Factor IX fusion proteins with higher specific activity and a longer useful clotting function relative to wild type or non-modified Factor IX protein. | ||||||
| 179 | Lipids and lipid nanoparticle formulations for delivery of nucleic acids | US15624548 | 2017-06-15 | US10106490B2 | 2018-10-23 | Xinyao Du |
| Compounds are provided having the following structure: or a pharmaceutically acceptable salt, tautomer or stereoisomer thereof, wherein R1a, R1b, R2a, R2b, R3a, R3b, R4a, R4b, R5, R6, R7, R8, R9, L1, L2, a, b, c, d and e are as defined herein. Use of the compounds as a component of lipid nanoparticle formulations for delivery of a therapeutic agent, compositions comprising the compounds and methods for their use and preparation are also provided. | ||||||
| 180 | Single Chain Fc Fusion Proteins | US15949596 | 2018-04-10 | US20180289825A1 | 2018-10-11 | Juan Alvarez; Demetri T. Moustakas; Heather R. Brodkin; Leslie A. McSweeney |
| The present invention provides novel, single chain Fc fusion proteins having improved properties. The invention provides single chain fusions of soluble proteins fused to the Fc region of an immunoglobulin via a novel linker comprising a constant region of an immunoglobulin light chain linked to a CH1 constant region of an immunoglobulin heavy chain. This novel linker confers favorable properties on the Fc fusion proteins of the invention such as improved bioactivity and increased half-life as compared to non-Fc fusion counterparts or as compared to prior art Fc fusion proteins. The novel Fc fusion protein scaffold as described herein may be designed to include soluble proteins of interest capable of binding or interacting with any target of interest. Preferably, the Fc fusion protein of the invention is a dimer. The dimer preferably forms via a disulfide bond between free cysteine residues in the hinge region of two monomeric Fc fusion proteins of the invention. | ||||||
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