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    METHODS, COMPOUNDS AND COMPOSITIONS FOR MODULATING BLOOD BRAIN BARRIER INTEGRITY AND RE-MYELINATION
    4.
    发明申请
    METHODS, COMPOUNDS AND COMPOSITIONS FOR MODULATING BLOOD BRAIN BARRIER INTEGRITY AND RE-MYELINATION 审中-公开
    方法，化合物和组合物，用于调节血液脑阻滞剂的完整性和再释放

    公开(公告)号：WO2017049411A1

    公开(公告)日：2017-03-30

    申请号：PCT/CA2016/051122

    申请日：2016-09-23

    Applicant: UNIVERSITY HEALTH NETWORK

    Inventor： MONNIER, Philippe Patrick ,  TASSEW, Nardos G. ,  BAGLAENKO, Yuriy

    IPC: A61K38/17 ,  A61K39/395 ,  A61K49/00 ,  A61P21/00 ,  A61P25/28 ,  C07K14/47

    CPC classification number: A61K38/179 ,  A61K38/1709 ,  A61K39/395 ,  C07K14/47

    Abstract: Disclosed herein are methods and compositions for increasing and decreasing the permeability of the blood brain barrier for the treatment of diseases and conditions and to facilitate the delivery of agents to the brain, as well as methods and compositions for promoting re-myelination and preventing de-myelination. Compositions include RGMa, soluble RGMa, and functional fragments and variants thereof, RGMc, soluble RGMc, and functional fragments and variants thereof, and Neogenin peptides including 4Ig.

    Abstract translation: 本文公开了用于增加和减少用于治疗疾病和病症的血脑屏障的渗透性并促进药物递送至脑的方法和组合物，以及用于促进再髓鞘化和预防脱发的方法和组合物， 髓鞘形成。 组合物包括RGMa，可溶性RGMa及其功能片段及其变体，RGMc，可溶性RGMc及其功能片段及变体，以及包含4Ig的Neogenin肽。

    TARGETING GIV-GEF-GI SIGNALING FOR TREATING DIVERSE DISEASES
    6.
    发明申请
    TARGETING GIV-GEF-GI SIGNALING FOR TREATING DIVERSE DISEASES 审中-公开
    指导GIV-GEF-GI信号治疗多种疾病

    公开(公告)号：WO2016168702A1

    公开(公告)日：2016-10-20

    申请号：PCT/US2016/027897

    申请日：2016-04-15

    Applicant: THE REGENTS OF THE UNIVERSITY OF CALIFORNIA ,  GHOSH, Pradipta

    Inventor： GHOSH, Pradipta

    IPC: A61K38/00 ,  C07K14/00 ,  C07K19/00 ,  C12N15/63

    CPC classification number: A61K38/179 ,  A61K38/00 ,  A61K38/1741 ,  A61P35/04 ,  C07K7/06 ,  C07K7/08 ,  C07K14/005 ,  C07K14/4703 ,  C07K14/4705 ,  C07K19/00 ,  C07K2319/02 ,  C07K2319/03 ,  C07K2319/10 ,  C07K2319/21 ,  C07K2319/23 ,  C07K2319/33 ,  C07K2319/43 ,  C07K2319/74 ,  C07K2319/81 ,  C12N15/63

    Abstract: The invention provides cell-permeable Ga-lnteracting Vesicle associated protein (GlV)-derived peptides and their use for treatment of diverse diseases. The invention further provides C-terminus of Ga-lnteracting Vesicle associated protein (GIV-CT)-based peptides and GIV-CT encoding vectors and methods of use thereof, for successfully manipulating the diverse pathophysiologic processes in which GIV has been implicated. In one aspect, the invention provides cell-permeable peptides PTD-GIV-CT comprising (i) a peptide transduction domain (PTD) and (ii) a C-terminus of Ga-lnteracting Vesicle associated protein (GIV-CT) or a mutant thereof. In one embodiment, said.peptides are capable of activating Gi ownstream of receptor tyrosine kinases (RTKs). The invention also provides pharmaceutical compositions comprising one or more of the peptides of the invention as well as vectors encoding such peptides.

    Abstract translation: 本发明提供了细胞可渗透的与Ga相互作用的囊泡相关蛋白（GlV）衍生的肽及其用于治疗多种疾病的用途。 本发明进一步提供了用于成功地操纵其中涉及GIV的多种病理生理过程的Ga反应性泡泡相关蛋白（GIV-CT）基于肽和GIV-CT编码载体的C末端及其使用方法。 一方面，本发明提供了细胞可渗透肽PTD-GIV-CT，其包含（i）肽转导结构域（PTD）和（ii）Ga-反应性泡泡相关蛋白（GIV-CT）的C末端或突变体 它们。 在一个实施方案中，所述肽能够激活受体酪氨酸激酶（RTK）的自身流。 本发明还提供包含一种或多种本发明的肽的药物组合物以及编码这些肽的载体。

    TREATMENT OF FIBRODYSPLASIA OSSIFICANS PROGRESSIVA
    7.
    发明申请
    TREATMENT OF FIBRODYSPLASIA OSSIFICANS PROGRESSIVA 审中-公开
    纤维肉质软骨细胞进程的处理

    公开(公告)号：WO2016039796A3

    公开(公告)日：2016-06-16

    申请号：PCT/US2015000100

    申请日：2015-09-14

    Applicant: REGENERON PHARMA

    Inventor： HATSELL SARAH J ,  ECONOMIDES ARIS N ,  IDONE VINCENT J

    IPC: C07K16/22 ,  A61K31/519 ,  A61K38/17 ,  A61P19/00 ,  C07K16/28

    CPC classification number: C07K16/18 ,  A61K38/179 ,  A61K39/39533 ,  A61K2039/505 ,  C07K14/705 ,  C07K16/22 ,  C07K16/28 ,  C07K16/2863 ,  C07K2317/21 ,  C07K2317/24 ,  C07K2317/33 ,  C07K2319/30

    Abstract: Methods for treating Fibrodysplasia Ossificans Progressiva (FOP) are provided. Such methods involve administering to a subject having FOP an effective regime of an activin receptor type 2A (ACVR2A) and/or an activin receptor type 2B (ACVR2B) antagonist or an activin receptor type 1 (ACVRl) antagonist. Antagonists include fusion proteins of one or more extracellular domains (ECDs) of ACVR2A, ACVR2B and/or ACVRl and the Fc domain of an immunoglobulin heavy chain, and antibodies against ACVR2A, ACVR2B, ACVRl or Activin A.

    Abstract translation: 提供治疗骨纤维增生异常进展（FOP）的方法。 此类方法涉及向具有FOP的受试者施用2A型活化素受体（ACVR2A）和/或2B型活化素受体（ACVR2B）拮抗剂或1型活化素受体（ACVR1）拮抗剂的有效方案。 拮抗剂包括ACVR2A，ACVR2B和/或ACVR1的一个或多个细胞外结构域（ECD）和免疫球蛋白重链的Fc结构域的融合蛋白，以及针对ACVR2A，ACVR2B，ACVR1或激活素A的抗体。

    TREATMENT OF CARDIOVASCULAR DISEASE USING ACTRII LIGAND TRAPS
    8.
    发明申请
    TREATMENT OF CARDIOVASCULAR DISEASE USING ACTRII LIGAND TRAPS 审中-公开
    使用ACTRII LIGAND TRAPS治疗心血管疾病

    公开(公告)号：WO2016069234A1

    公开(公告)日：2016-05-06

    申请号：PCT/US2015/054674

    申请日：2015-10-08

    Applicant: CELGENE CORPORATION ,  WASHINGTON UNIVERSITY

    Inventor： HRUSKA, Keith ,  FANG, Yifu ,  SMITH, William ,  CHEN, Nianhang

    IPC: A61P9/00 ,  A61K38/00

    CPC classification number: A61K38/179 ,  A61K38/00 ,  A61P9/00 ,  A61P9/10 ,  A61P19/10 ,  C07K14/71 ,  C07K2319/30

    Abstract: Provided herein are methods of treating diseases associated with vascular calcification and/or cardiovascular disease, and/or bone resorption in a subject, by using the level of a biomarker, in particular, snail homolog I (Snail), phosphosmad2, phosphosmad3, urinary protein, dickkopf homolog I (Dkkl), collagen type 1 alphal (Collal), activin (i.e.free activin), runt-related transcription factor 2 (Runx2 ), alkaline phosphatase (Alp), bone-specific alkaline phosphatase (BSAP), C-terninal type 1 collagen telopeptide (CTX), osterix, Klotho, alpha-smooth muscle actin (alpha-SMA), myocardin (JVIYOCD), activin receptor type 2A (ActRIIA), axis inhibition protein 2 (Axin2), and/or smooth muscle protein 22-alpha (Sm22-alpha), as an indicator(s) of responsiveness of the subject to the treatment, efficacy of the treatment, or appropriate dosage tor the treatment with an activin type II receptor signaling inhibitor.

    Abstract translation: 本文提供了通过使用生物标志物的水平，特别是蜗牛同源物I（Snail），磷磷酸2，磷磷酸3，尿蛋白质来治疗与受试者血管钙化和/或心血管疾病和/或骨吸收相关的疾病的方法 ，dickkopf同源物I（Dkkl），1型胶原（Collal），激活素（即免疫激活素），runt相关转录因子2（Runx2），碱性磷酸酶（Alp），骨特异性碱性磷酸酶（BSAP），C- 1型胶原端肽（CTX），osterix，Klotho，α-平滑肌肌动蛋白（α-SMA），心肌素（JVIYOCD），2A型激活素受体（ActRIIA），轴抑制蛋白2（Axin2）和/或平滑肌 蛋白22-α（Sm22-α）作为受试者对治疗的反应性的指标，治疗的功效或用激活素II型受体信号传导抑制剂治疗的适当剂量。

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