公开(公告)号：US20230270884A1

公开(公告)日：2023-08-31

申请号：US18005504

申请日：2021-07-13

Applicant: The Trustees of the University of Pennsylvania

Inventor： James M. Wilson ,  Christian Hinderer ,  Eileen Workman

IPC: A61K48/00 ,  C12N15/86 ,  C12N15/113 ,  C12N9/14 ,  A61P25/00

CPC classification number: A61K48/005 ,  C12N15/86 ,  C12N15/1137 ,  C12N9/14 ,  A61P25/00 ,  C12Y306/05

Abstract: Provided herein are rAAV and other vectors and compositions useful for treating a patient having CMT2 comprising: (a) a recombinant nucleic acid sequence encoding an engineered human mitofusin 2 coding sequence operably linked to regulatory sequences which direct expression thereof in a human target cell. Also provided are rAAV and other vectors and compositions useful for treating a patient having CMT2 comprising: (b) a nucleic acid sequence encoding at least one miRNA specific for an endogenous human mitofusin 2 sequence in a human CMT2A subject, wherein the miRNA coding sequence is operably linked to regulatory sequences which direct expression thereof in the subject. Further provided are compositions containing both the engineered hMfn2 coding sequence and the at least one miRNA coding sequence, wherein the engineered human mitofusin 2 coding sequence has a sequence which differs from endogenous human mitofusin 2 in the CMT2A patient in the target site of the encoded miRNA.

公开(公告)号：US20180036385A1

公开(公告)日：2018-02-08

申请号：US15699262

申请日：2017-09-08

Applicant: The Trustees of the University of Pennsylvania ,  University of Florida Research Foundation, Inc.

Inventor： William A. BELTRAN ,  Gustavo D. AGUIRRE ,  Samuel G. JACOBSON ,  Artur V. CIDECIYAN ,  Alfred S. LEWIN ,  Sanford L. BOYE ,  William W. HAUSWIRTH ,  Wen-Tao DENG

IPC: A61K38/46 ,  C12N15/86 ,  A61K48/00 ,  C07K14/47 ,  A61K38/17 ,  A61K38/18

CPC classification number: A61K38/46 ,  A61K38/1709 ,  A61K38/185 ,  A61K48/00 ,  A61K48/005 ,  C07K14/4702 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2830/008 ,  C12Y306/05

Abstract: Described herein are methods of preventing, arresting progression of or ameliorating vision loss and other conditions associated with retinitis pigmentosa and x-linked retinitis pigmentosa in a subject. The methods include administering to said subject an effective concentration of a composition comprising a recombinant adeno-associated virus (AAV) carrying a nucleic acid sequence encoding a normal retinitis pigmentosa GTPase regulator (RPGR gene), or fragment thereof, under the control of regulatory sequences which express the product of the gene in the photoreceptor cells of the subject, and a pharmaceutically acceptable carrier.

公开(公告)号：US20160032264A1

公开(公告)日：2016-02-04

申请号：US14883133

申请日：2015-10-14

Applicant: ASK Diagnostics, Inc.

Inventor： Geoffrey Schmidt

IPC: C12N9/14 ,  G01N33/573

CPC classification number: C12N9/14 ,  C07K16/40 ,  C07K2317/34 ,  C12Y306/05 ,  G01N33/573 ,  G01N2333/914

Abstract: Disclosed are therapeutic formulations comprising antibodies against the PEKRAEKIWK (SEQ ID NO:1) epitope of the monomeric isoform of A-protein and a physiologically acceptable carrier. Methods for the treatment of subjects using these therapeutic formulations are also disclosed.

Abstract translation: 公开了包含抗A蛋白的单体同种型的PEKRAEKIWK（SEQ ID NO：1）表位的抗体和生理上可接受的载体的治疗制剂。 还公开了使用这些治疗制剂治疗受试者的方法。

公开(公告)号：US20190247480A1

公开(公告)日：2019-08-15

申请号：US16389676

申请日：2019-04-19

Applicant: RhoVac ApS

Inventor： Mads Hald Andersen ,  Per Thor Straten

IPC: A61K39/00 ,  C07K14/82 ,  C07K14/47 ,  C12N9/14

CPC classification number: A61K39/0011 ,  A61K38/00 ,  C07K14/4702 ,  C07K14/82 ,  C12N9/14 ,  C12Y306/05

Abstract: The present invention relates generally to the field of prophylaxis and therapy of metastatic cancer. In particular there is provided a protein; Ras Homology gene family, member C (RhoC) or peptide fragments thereof that are capable of eliciting anti-cancer immune responses. Specifically, the invention relates to use of RhoC or peptides derived thereof or RhoC specific T-cells for treatment of metastatic cancer. Hence, the invention in one aspect relates to RhoC specific T-cells adoptively transferred or induced in vivo by vaccination as a treatment of cancer.Also the use of RhoC and immunogenic peptide fragments hereof in cancer treatment, diagnosis and prognosis is provided.

公开(公告)号：US20180080926A1

公开(公告)日：2018-03-22

申请号：US15710696

申请日：2017-09-20

Applicant: The Board of Trustees of the Leland Stanford Junior University ,  Washington University

Inventor： Daria Mochly-Rosen ,  Gerald W. Dorn, II

IPC: G01N33/50 ,  G01N33/58 ,  C07K14/705 ,  C07K7/08 ,  G01N33/547 ,  G01N33/566

CPC classification number: G01N33/5079 ,  A61K38/00 ,  C07K7/08 ,  C07K14/705 ,  C07K2319/10 ,  C12N9/14 ,  C12Y306/05 ,  C12Y310/01 ,  G01N33/547 ,  G01N33/566 ,  G01N33/582 ,  G01N2500/00

Abstract: Mitofusin modulatory peptides are described which may function as activators or inhibitors of mitochondrial fusion. The sequences and compositions comprising the sequences are useful for treating diseases or disorders associated with mitofusin 1 (Mfn1) and/or mitofusin 2 (Mfn2) and mitochondrial dysfunction. Methods of treatment, pharmaceutical formulations and methods of identifying compounds that mimic the activity of the peptides for use in screening assays are also described.

公开(公告)号：US20150273026A1

公开(公告)日：2015-10-01

申请号：US14741578

申请日：2015-06-17

Applicant: Rapid Pathogen Screening, Inc.

Inventor： Robert W. VanDine ,  Robert P. Sambursky ,  Uma Mahesh Babu

IPC: A61K38/46

CPC classification number: A61K38/46 ,  C12N9/14 ,  C12N9/16 ,  C12Y301/04 ,  C12Y306/05 ,  G01N33/56983

Abstract: Full length MxA constructs and truncated MxA constructs produce human MxA protein in E. coli. The full length MxA and truncated MxA constructs are preferably E. coli codon-optimized to optimize the amount of protein made using the constructs. T5 or T7 promoters can each be used in combination with either the full length MxA or the truncated MxA constructs. In one preferred embodiment, the MxA protein produced by the full length MxA or truncated MxA constructs is used in a control prep or external control. In other preferred embodiments, the MxA protein is used as a therapeutic.

Abstract translation: 全长MxA构建体和截短的MxA构建体在大肠杆菌中产生人MxA蛋白。 全长MxA和截短的MxA构建体优选是经密码子优化的E.coli，以优化使用构建体制备的蛋白质的量。 T5或T7启动子各自可以与全长MxA或截短的MxA构建体组合使用。 在一个优选实施方案中，由全长MxA或截短的MxA构建体产生的MxA蛋白用于对照制备或外部对照。 在其它优选的实施方案中，MxA蛋白用作治疗剂。

公开(公告)号：US20150202269A1

公开(公告)日：2015-07-23

申请号：US14413884

申请日：2013-01-23

Applicant: The Trustees of the University of Pennsylvania ,  University of Florida Research Foundation, Inc.

Inventor： William A Beltran ,  Gustavo D Aguirre ,  Samuel G Jacobson ,  Artur V Cideciyan ,  Alfred S Lewin ,  Sanford L Boye ,  William W Hauswirth ,  Wen-Tao Deng

IPC: A61K38/46 ,  A61K38/18 ,  C12N15/86

CPC classification number: A61K38/46 ,  A61K38/1709 ,  A61K38/185 ,  A61K48/00 ,  A61K48/005 ,  C07K14/4702 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2830/008 ,  C12Y306/05

Abstract: Described herein are methods of preventing, arresting progression of or ameliorating vision loss and other conditions associated with retinitis pigmentosa and x-linked retinitis pigmentosa in a subject. The methods include administering to said subject an effective concentration of a composition comprising a recombinant adeno-associated virus (AAV) carrying a nucleic acid sequence encoding a normal retinitis pigmentosa GTPase regulator (RPGR gene), or fragment thereof, under the control of regulatory sequences which express the product of the gene in the photoreceptor cells of the subject, and a pharmaceutically acceptable carrier.

Abstract translation: 本文描述的是预防，阻止视力丧失进展或改善与受试者中色素性视网膜炎和色素连带性视网膜炎相关的其它病症的方法。 所述方法包括向所述受试者施用有效浓度的组合物，所述组合物包含携带编码正常视网膜色素变性GTP酶调节剂（RPGR基因）或其片段的核酸序列的重组腺相关病毒（AAV），其在调节序列的控制下 其表达该受试者的感光细胞中的基因的产物和药学上可接受的载体。

公开(公告)号：US20140370079A1

公开(公告)日：2014-12-18

申请号：US14271117

申请日：2014-05-06

Applicant: Korea Advanced Institute of Science and Technology (KAIST)

Inventor： Gou Young KOH ,  Chan Kim ,  Hanseul Yang ,  Won Do Heo ,  Injune Kim ,  Sangyong Jon ,  Akiyoshi Uemura

IPC: C12N15/113 ,  A61K31/661 ,  A61K33/24 ,  A61K45/06

CPC classification number: C12N15/1137 ,  A61K9/1271 ,  A61K31/661 ,  A61K31/7088 ,  A61K33/24 ,  A61K45/06 ,  C12N2310/14 ,  C12N2320/31 ,  C12Y306/05 ,  A61K2300/00

Abstract: The present application describes a method of inhibiting tumor growth including contacting the tumor with a compound that inhibits activity of RhoJ protein.

Abstract translation: 本申请描述了抑制肿瘤生长的方法，包括使肿瘤与抑制RhoJ蛋白活性的化合物接触。

公开(公告)号：US20240344041A1

公开(公告)日：2024-10-17

申请号：US18517417

申请日：2023-11-22

Applicant: PRESIDENT AND FELLOWS OF HARVARD COLLEGE

Inventor： Pascal S. KAESER ,  Chao TAN ,  Shan Shan WANG

IPC: C12N9/14 ,  A61K38/00 ,  A61P25/28 ,  C07K14/705

CPC classification number: C12N9/14 ,  A61P25/28 ,  C07K14/705 ,  C12Y306/05 ,  A61K38/00

Abstract: Technology described herein relates to a fusion protein comprising a zinc-finger domain (ZNF) of Regulating Synaptic Membrane Exocytosis Protein (RIMS); and a CaVβ Ca2+ channel subunit. Compositions comprising the fusion protein and method of treatment utilizing the fusion protein are also provided herein.

公开(公告)号：US10011600B2

公开(公告)日：2018-07-03

申请号：US15508387

申请日：2015-09-25

Applicant: ARAXES PHARMA LLC

Inventor： Liansheng Li ,  Jun Feng ,  Yun Oliver Long ,  Pingda Ren ,  Yi Liu

IPC: C07D471/04 ,  A61K45/06 ,  C07D239/94 ,  A61K31/517 ,  C07D403/04 ,  C07D401/04 ,  C07D403/12 ,  C07D401/14 ,  C07D405/12 ,  A61K9/00 ,  C12N9/14

CPC classification number: C07D471/04 ,  A61K9/0019 ,  A61K9/0053 ,  A61K31/517 ,  A61K45/06 ,  C07D239/94 ,  C07D239/95 ,  C07D401/04 ,  C07D401/14 ,  C07D403/04 ,  C07D403/12 ,  C07D403/14 ,  C07D405/12 ,  C12N9/14 ,  C12Y306/05

Abstract: Inhibitors of Ras protein, methods to modulate the activity of Ras protein, and methods of treatment of disorders mediated by Ras protein are provided. A method for regulating activity of a K-Ras, H-Ras or N-Ras mutant protein with a compound is described. Disorders that can be treated include cancer, such as hematological cancer, pancreatic cancer, MYH associated polyposis, colorectal cancer, or lung cancer.