公开(公告)号：US20140107186A1

公开(公告)日：2014-04-17

申请号：US14004379

申请日：2012-03-12

Applicant: Luis Garcia ,  Cyriaque Beley ,  Thomas Voit ,  Robert Michael Kotin

Inventor： Luis Garcia ,  Cyriaque Beley ,  Thomas Voit ,  Robert Michael Kotin

IPC: C12N15/86

CPC classification number: C12N15/86 ,  C12N2750/14143 ,  C12N2750/14171 ,  C12N2800/24

Abstract: An isolated linear nucleic acid molecule comprising in this order: a first adeno-associated virus (AAV) inverted terminal repeat (ITR), a nucleotide sequence of interest and a second AAV ITR, wherein said nucleic acid molecule is devoid of AAV capsid protein coding sequences. The said nucleic acid molecule can be applied to a host at repetition without eliciting an immune response. Methods for producing and purifying this nucleic acid molecule, and use of the same for therapeutic purposes are also provided.

Abstract translation: 一种分离的线性核酸分子，其以以下顺序包含：第一腺相关病毒（AAV）倒置末端重复（ITR），目的核苷酸序列和第二AAV ITR，其中所述核酸分子缺乏AAV衣壳蛋白编码 序列。 所述核酸分子可以重复施用于宿主而不引发免疫应答。 还提供了用于产生和纯化该核酸分子的方法及其用于治疗目的的用途。

公开(公告)号：US20130189225A1

公开(公告)日：2013-07-25

申请号：US13636596

申请日：2011-03-22

Applicant: Thomas Voit ,  Luis Garcia ,  Jérôme Denard ,  Fedor Svinartchouk

Inventor： Thomas Voit ,  Luis Garcia ,  Jérôme Denard ,  Fedor Svinartchouk

IPC: A61K48/00 ,  A61K38/00 ,  A61K31/522 ,  A61K38/21

CPC classification number: C04B35/10 ,  A61K31/522 ,  A61K38/00 ,  A61K38/1709 ,  A61K38/21 ,  A61K45/06 ,  A61K48/0008 ,  A61K48/0058 ,  C04B35/14 ,  C04B35/565 ,  C04B35/622 ,  C07K14/005 ,  C07K16/081 ,  C07K16/18 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2750/14151 ,  C12N2810/6027 ,  A61K2300/00

Abstract: Methods for increasing the efficiency of target tissue penetration of an adeno-associated virus (AAV) vector in a patient are provided. In some aspects, the methods involve inhibiting the interaction of the serum protein galectin 3 binding protein (G3BP) with AAV vector. Further provided are methods for reducing tissue distribution of a virus or for neutralizing a virus harbored by an organ destined for transplant, or newly transplanted, by administering a composition comprising G3BP.

Abstract translation: 提供了增加患者中腺相关病毒（AAV）载体靶组织穿透效率的方法。 在某些方面，该方法涉及抑制血清蛋白半乳凝素3结合蛋白（G3BP）与AAV载体的相互作用。 进一步提供的是通过施用包含G3BP的组合物来减少病毒的组织分布或用于中和由目的地移植或新移植的器官所携带的病毒的方法。

公开(公告)号：US08440151B2

公开(公告)日：2013-05-14

申请号：US12673755

申请日：2008-08-18

Applicant: Thomas Voit

Inventor： Thomas Voit

IPC: B01L3/00

CPC classification number: B65B69/0041 ,  B01L3/50853 ,  B01L2200/16 ,  B01L2300/044 ,  B01L2300/0672 ,  B01L2300/0832 ,  B65D77/08 ,  G01N35/1079

Abstract: A system (10) for removing a pipettable substance from a pre-filled container (20), which is closed off by a top (30) having at least one opening area (40), comprises an opening tool (100) having a tube (110), which has a cross-section corresponding substantially to the shape of the opening area and which comprises at a distal end (120) an endpiece (140) extending substantially obliquely relative to the longitudinal axis of the tube, which moves a part of the top (30) located inside the opening area (40) towards the container when the opening tool is applied, so as to form an opening in the top, and a point of attack (150) for a transporting tool (200). The opening tool (100) is designed to remain on the container (20) after use. Moreover, the system comprises a transporting tool (200) for moving the opening tool (100) through the lid, and a cannula (250), which is adapted to be inserted over at least part of its length through the tube (110) into the interior of the container (20), in the position of use of the opening tool (100), and one end of which can be connected to a suction device (300) for sucking a pipettable substance out of the interior of the container (20).

Abstract translation: 一种用于从预填充容器（20）中移除可移液物质的系统（10），其由具有至少一个开口区域（40）的顶部（30）封闭，包括具有管子的开口工具（100） （110），其横截面基本上对应于开口区域的形状，并且在远端（120）处包括相对于管的纵向轴线基本上倾斜地延伸的端部件（140），所述端件（110）移动部件 当打开工具被施加时，位于开口区域（40）内部的顶部（30）朝向容器移动，以便在顶部形成开口以及用于输送工具（200）的攻角（150）。 打开工具（100）设计成在使用后留在容器（20）上。 此外，该系统包括用于使打开工具（100）通过盖移动的输送工具（200）和套管（250），套管（250）适于在其长度的至少一部分上穿过管（110）插入， 所述容器（20）的内部处于使用所述打开工具（100）的位置，并且其一端可以连接到用于从所述容器的内部吸出可移液物质的抽吸装置（300） 20）。

公开(公告)号：US20130045538A1

公开(公告)日：2013-02-21

申请号：US13635356

申请日：2011-03-17

Applicant: Luis Garcia ,  Denis Furling ,  Cyriaque Beley ,  Thomas Voit

Inventor： Luis Garcia ,  Denis Furling ,  Cyriaque Beley ,  Thomas Voit

IPC: C07H21/02 ,  C12N15/113 ,  C12N15/85 ,  C12N15/63 ,  C12N5/10

CPC classification number: C12N15/111 ,  C12N2310/11 ,  C12N2310/531 ,  C12N2320/33 ,  C12N2320/34 ,  C12N2320/50 ,  C12N2330/51

Abstract: The present invention relates to a method to improve the activity of engineered U7 snRNAs used in the context of RNA-based therapeutics; particularly in exon skipping, exon inclusion, and mRNA eradication strategies. The resulting modified snRNAs are useful for treating neuromuscular diseases, in particular Duchenne neuromuscular dystrophy, myotonic dystrophy DM1 and spinal muscular atrophy.

Abstract translation: 本发明涉及一种改善在基于RNA的疗法的上下文中使用的工程化U7 snRNA的活性的方法; 特别是在外显子跳跃，外显子包含和mRNA消除策略。 所产生的修饰的snRNA可用于治疗神经肌肉疾病，特别是Duchenne神经肌肉营养不良症，强直性营养不良DM1和脊髓性肌肉萎缩。

公开(公告)号：US20120149756A1

公开(公告)日：2012-06-14

申请号：US13263949

申请日：2010-04-09

Applicant: Daniel Schümperli ,  Christian Leumann ,  Denis Furling ,  Luis Garcia ,  Thomas Voit

Inventor： Daniel Schümperli ,  Christian Leumann ,  Denis Furling ,  Luis Garcia ,  Thomas Voit

IPC: A61K31/712 ,  A61P21/00 ,  A61P25/00 ,  C07H21/04 ,  C12N5/071

CPC classification number: C12N15/113 ,  A61K31/711 ,  A61K31/712 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/3231 ,  C12N2320/33

Abstract: Provided are tricyclo-DNA (tc-DNA) AON and methods employing tc-DNA AON for modifying splicing events that occur during pre-mRNA processing. Tricyclo-DNA (tc-DNA) AON are described that may be used to facilitate exon skipping or to mask intronic silencer sequences and/or terminal stem-loop sequences during pre-mRNA processing and to target RNase-mediated destruction of processed mRNA. Tc-DNA AON described herein may be used in methods for the treatment of Duchenne Muscular Dystrophy by skipping a mutated exon 23 or exon 51 within a dystrophin gene to restore functionality of a dystrophin protein; in methods for the treatment of Spinal Muscular Atrophy by masking an intronic silencing sequence and/or a terminal stem-loop sequence within an SMN2 gene to yield modified functional SMN2 protein, including an amino acid sequence encoded by exon 7, which is capable of at least partially complementing a non-functional SMN1 protein; and in methods for the treatment of Steinert's Myotonic Dystrophy by targeting the destruction of a mutated DM1 mRNA comprising 3′-terminal CUG repeats.

Abstract translation: 提供三环DNA（tc-DNA）AON和使用tc-DNA AON来改变在mRNA前处理期间发生的剪接事件的方法。 描述了Tricyclo-DNA（tc-DNA）AON，其可用于促进外显子跳跃或在mRNA前处理期间掩盖内含子沉默子序列和/或末端茎环序列，并靶向核糖核酸酶介导的加工mRNA的破坏。 本文所述的Tc-DNA AON可用于通过跳过肌营养不良蛋白基因内突变外显子23或外显子51来恢复肌营养不良蛋白功能的方法来治疗杜氏肌营养不良症; 在通过掩蔽SMN2基因内的内含子沉默序列和/或末端茎环序列来治疗脊髓性肌萎缩的方法中，以产生修饰的功能性SMN2蛋白，包括由外显子7编码的氨基酸序列，其能够在 最少部分补充非功能性SMN1蛋白; 以及用于通过靶向包含3'-末端CUG重复序列的突变的DM1mRNA的破坏来治疗Steinert's强直性营养不良的方法。

公开(公告)号：US11773408B2

公开(公告)日：2023-10-03

申请号：US16707223

申请日：2019-12-09

Applicant: Lucie Carrier ,  Thomas Eschenhagen ,  Thomas Voit ,  Giulia Mearini ,  Oliver Mueller ,  Doreen Stimpel

Inventor： Lucie Carrier ,  Thomas Eschenhagen ,  Thomas Voit ,  Giulia Mearini ,  Oliver Mueller ,  Doreen Stimpel ,  Julia Mourot-Filiatre

IPC: C12N15/86 ,  C07K14/47 ,  A61K35/34 ,  C12N5/074 ,  C12N5/077 ,  A61K48/00 ,  C12N7/00

CPC classification number: C12N15/86 ,  A61K35/34 ,  A61K48/0058 ,  C07K14/4716 ,  C12N5/0657 ,  C12N5/0696 ,  C12N7/00 ,  C12N2506/45 ,  C12N2750/14143 ,  C12N2799/025 ,  C12N2830/007 ,  C12N2830/008

Abstract: The present invention relates to a gene therapy vector which is useful in the treatment or prevention of hypertrophic cardiomyopathy in a subject in need thereof. The gene therapy vector of the invention comprises a nucleic acid sequence encoding a cardiac sarcomeric protein and a cardiomyocyte-specific promoter which is operably linked to said nucleic acid sequence. The invention furthermore relates to a cell which comprises the gene therapy vector. Pharmaceutical compositions which comprise the gene therapy vector and/or a cell comprising said vector are also provided. In another aspect, the invention relates to a method for treating or preventing hypertrophic cardiomyopathy in a subject by introducing the gene therapy vector of the invention into a subject in need of treatment.

公开(公告)号：US08997800B2

公开(公告)日：2015-04-07

申请号：US12673758

申请日：2008-08-18

Applicant: Thomas Voit ,  Andrea Wildhaber

Inventor： Thomas Voit ,  Andrea Wildhaber

IPC: B65B3/04 ,  G01N35/10 ,  B01L3/00

CPC classification number: B65B69/0041 ,  B01L3/50853 ,  B01L2200/16 ,  B01L2300/044 ,  B01L2300/0672 ,  B01L2300/0832 ,  B65D77/08 ,  G01N35/1079

Abstract: A system (10) for removing a pipettable substance from a pre-filled container (20), which is closed off by a top (30) having at least one opening area (40), comprises an opening tool (100) having a tube (110), which has a cross-section corresponding substantially to the shape of the opening area and which comprises at a distal end (120) an endpiece (140) extending substantially obliquely relative to the longitudinal axis of the tube, which moves a part of the top (30) located inside the opening area (40) towards the container when the opening tool is applied, so as to form an opening in the top, and a point of attack (150) for a transporting tool (200). The opening tool (100) is designed to remain on the container (20) after use. Moreover, the system comprises a transporting tool (200) for moving the opening tool (100) through the top, and a cannula (250), which is adapted to be inserted over at least part of its length through the tube (110) into the interior of the container (20), in the position of use of the opening tool (100), and one end of which can be connected to a suction device (300) for sucking a pipettable substance out of the interior of the container (20).

Abstract translation: 一种用于从预填充容器（20）中移除可移液物质的系统（10），其由具有至少一个开口区域（40）的顶部（30）封闭，包括具有管子的开口工具（100） （110），其横截面基本上对应于开口区域的形状，并且在远端（120）处包括相对于管的纵向轴线基本上倾斜地延伸的端部件（140），所述端件（110）移动部件 当打开工具被施加时，位于开口区域（40）内部的顶部（30）朝向容器移动，以便在顶部形成开口以及用于输送工具（200）的攻角（150）。 打开工具（100）设计成在使用后留在容器（20）上。 此外，该系统包括用于使打开工具（100）通过顶部移动的输送工具（200）和适于在其长度的至少一部分上穿过管（110）插入的套管（250），该套管 所述容器（20）的内部处于使用所述打开工具（100）的位置，并且其一端可以连接到用于从所述容器的内部吸出可移液物质的抽吸装置（300） 20）。

公开(公告)号：US08932542B2

公开(公告)日：2015-01-13

申请号：US11992637

申请日：2006-09-26

Applicant: Andreas Schaefer ,  Thomas Voit ,  Walter Tschopp ,  Adrian Geiger ,  Markus Zbinden ,  Harald Hibbing ,  Andreas Karl ,  Frank Eigemeier ,  Volker Behrmann ,  Dietmar Kopp ,  Andreas Schmiede

Inventor： Andreas Schaefer ,  Thomas Voit ,  Walter Tschopp ,  Adrian Geiger ,  Markus Zbinden ,  Harald Hibbing ,  Andreas Karl ,  Frank Eigemeier ,  Volker Behrmann ,  Dietmar Kopp ,  Andreas Schmiede

IPC: B01L9/06 ,  G01N35/00 ,  B04B5/04 ,  B04B13/00 ,  B01L3/00 ,  B04B11/04

CPC classification number: B04B5/02 ,  B01L3/5021 ,  B01L9/06 ,  B01L2300/043 ,  B04B5/0421 ,  B04B7/08 ,  B04B13/00 ,  B04B2011/046 ,  B25J11/00 ,  G01N35/00712 ,  G01N35/00732 ,  G01N35/0099 ,  G01N2035/00801 ,  G01N2035/0436 ,  G01S15/04 ,  G01V8/10

Abstract: A gripper unit for handling a vessel for receiving biological material is proposed, inter alia. The vessel has a lid which can assume an open position and a closed position. The gripper unit comprises a gripper for gripping and releasing the vessel, and a lid holder, for holding a lid in a defined position in relation to the vessel. The defined position is an open position of the lid.

Abstract translation: 提出了用于处理用于接收生物材料的容器的夹持器单元。 容器具有能够承受打开位置和关闭位置的盖子。 夹持器单元包括用于夹紧和释放容器的夹持器和用于将盖保持在相对于容器的限定位置的盖保持器。 定义的位置是盖的打开位置。

公开(公告)号：US08771614B2

公开(公告)日：2014-07-08

申请号：US11992616

申请日：2006-09-26

Applicant: Andreas Schaefer ,  Thomas Voit ,  Markus Zbinden ,  Andreas Schmiede

Inventor： Andreas Schaefer ,  Thomas Voit ,  Markus Zbinden ,  Andreas Schmiede

IPC: B04B5/02 ,  B01L3/00

CPC classification number: B01L3/5021 ,  B01L3/50255 ,  B01L3/56 ,  B01L9/06 ,  B01L2200/023 ,  B01L2400/0409 ,  B04B5/02 ,  B04B5/0421 ,  B04B2005/0435 ,  G01N1/405 ,  G01N1/4077 ,  G01N2035/00485 ,  G01N2035/00504 ,  Y10T436/25375

Abstract: The present invention relates to improved methods for processing fluids and to a fluid processing device (1) for use in a centrifuge comprising: (a) a first holder (14) form-fit to the shape of a first tube (18) for holding said first tube (18) whereby said first tube (18) has a first cross section (A1); and (b) a second holder (22) form-fit to the shape of a second tube (26) for holding said second tube (26) whereby said second tube (26) has a second cross section (A2) that is different from said first cross section (A1). With the fluid processing devices and the methods according to the invention, it is possible to simplify the centrifugal processing steps for a given fluid processing sequence and to automate them.

Abstract translation: 本发明涉及用于处理流体的改进方法和用于离心机的流体处理装置（1），其包括：（a）第一保持器（14），其形状配合到第一管（18）的形状，用于保持 所述第一管（18），由此所述第一管（18）具有第一横截面（A1）; 和（b）与第二管（26）的形状配合的用于保持所述第二管（26）的第二保持器（22），由此所述第二管（26）具有不同于第二管 第一个横截面（A1）。 利用根据本发明的流体处理装置和方法，可以简化给定流体处理顺序的离心处理步骤并使其自动化。

公开(公告)号：US20130026268A1

公开(公告)日：2013-01-31

申请号：US13560304

申请日：2012-07-27

Applicant: Dennis MERTENS ,  Thomas VOIT

Inventor： Dennis MERTENS ,  Thomas VOIT

IPC: C12M1/33 ,  B02C19/16

CPC classification number: B02C17/14

Abstract: The invention relates, for example, to methods for preparing a plant or animal sample for processing, i.e. for example for the isolation of nucleic acids or proteins from the sample, as well as to a pulverizer.

Abstract translation: 本发明涉及例如用于制备用于加工的植物或动物样品的方法，例如用于从样品中分离核酸或蛋白质以及粉碎机。