公开(公告)号：WO2019133632A1

公开(公告)日：2019-07-04

申请号：PCT/US2018/067536

申请日：2018-12-26

Applicant: ST. JUDE CHILDREN'S RESEARCH HOSPITAL ,  COA THERAPEUTICS, INC.

Inventor： JACKOWSKI, Suzanne ,  ROCK, Charles, O. ,  LEE, Richard, E. ,  SHARMA, Lalit, Kumar ,  YUN, Mi, Kyung ,  SUBRAMANIAN, Chitra ,  TANGALLAPALLY, Rajendra, P. ,  EDWARDS, Anne, V. ,  ZAMBONI, Robert ,  REDDY, T., Jagadeeswar ,  LIU, Jiuyu

IPC: A61K38/43 ,  A61P35/00

Abstract: The present disclosure relates to methods of treating a coenzyme A reduction, elevation, sequestration, toxicity, or redistribution (CASTOR) disease such as, for example, defects in fatty acid oxidation enzymes, methylmalonic acidemia, glutaric acidemia, propionic academia, and HMG-CoA lyase, via small molecule modulators of CoA levels. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present invention.

公开(公告)号：WO2019116392A1

公开(公告)日：2019-06-20

申请号：PCT/IN2018/050837

申请日：2018-12-13

Applicant: GANGAGEN, INC. ,  AMBADY, Anisha

Inventor： AMBADY, Anisha ,  APPAIAH, Chemira Biddappa ,  BALASUBRAMANIAN, Deepak ,  PAUL, Vivek Daniel ,  SARAVANAN, R. Sanjeev ,  SHARMA, Umender Kumar

IPC: A01N63/02 ,  A61K38/16 ,  A61K38/43 ,  A61K38/50 ,  A61K39/085 ,  A61K39/108 ,  C07K14/195 ,  C07K14/21 ,  C07K14/245

Abstract: The present invention provides methods and compositions to reduce growth of microbial colonies, including infections, and includes therapeutic compositions, methods for treatment of infections, and methods for identifying additional such compositions.

公开(公告)号：WO2019089898A1

公开(公告)日：2019-05-09

申请号：PCT/US2018/058665

申请日：2018-11-01

Applicant: DANISCO US INC

Inventor： BECKER, Nathaniel T. ,  MCAULIFFE, Joseph C. ,  MOSLEMY, Peyman ,  TAN, Jared Enwei ,  WHITE, Luther

IPC: A61K9/16 ,  A61K38/43

CPC classification number: A61K9/1694 ,  A61K9/1611 ,  A61K9/1617 ,  A61K9/1623 ,  C12N9/96

Abstract: The present disclosure is generally related to freezing point depressed solid matrix (FPDSM) systems, methods thereof and formulations thereof, wherein the FPDSM system stabilizes solid enzyme compositions thereof. In certain embodiments, the disclosure is related to methods for inhibiting the degradation of solid enzyme compositions formulated with one or more FPDSM systems of the disclosure.

公开(公告)号：WO2019068098A1

公开(公告)日：2019-04-04

申请号：PCT/US2018/053787

申请日：2018-10-01

Applicant: VANDERBILT UNIVERSITY

Inventor： DUVALL, Craig L. ,  KAVANAUGH, Taylor ,  DAILING, Eric ,  DOLLINGER, Bryan ,  DEJULIUS, Carlisle

IPC: A61K38/44 ,  A61K38/43 ,  A61K45/00 ,  A61P19/00

Abstract: Provided herein are a reactive oxygen species (ROS) scavenging composition, a method of forming a ROS scavenging composition, and a method of treating a subject having a (ROS) perpetuated disease. The ROS scavenging composition includes a co-polymer of a hydrophilic monomer and a grafting monomer, and a ROS scavenging compound grafted to the grafting monomer. The method of forming a ROS scavenging composition includes forming a co-polymer of a hydrophilic monomer and a grafting monomer, and grafting ROS scavenging compound onto the co-polymer. The method of treating a subject having a ROS perpetuated disease includes administering a therapeutically effective amount of an ROS scavenging composition comprising a co-polymer of a hydrophilic monomer and a grafting monomer, and a ROS scavenging compound grafted to the grafting monomer, to the subject in need thereof.

公开(公告)号：WO2019060746A1

公开(公告)日：2019-03-28

申请号：PCT/US2018/052247

申请日：2018-09-21

Applicant: THE BROAD INSTITUTE, INC. ,  MASSACHUSETTS INSTITUTE OF TECHNOLOGY ,  PRESIDENT AND FELLOWS OF HARVARD COLLEGE

Inventor： ZHANG, Feng ,  GOOTENBERG, Jonathan ,  COX, David Benjamin Turitz ,  ABUDAYYEH, Omar ,  KANNAN, Soumya

IPC: A61K38/43 ,  C12N15/09 ,  C12N15/10 ,  C12N9/16

Abstract: The disclosure provides for systems, methods, and compositions for targeting and editing nucleic acids. In particular, the invention provides non-naturally occurring or engineered RNA-targeting systems comprising a RNA-targeting Casl3 protein, at least one guide molecule, and at least one adenosine deaminase protein or catalytic domain thereof.

公开(公告)号：WO2018215780A1

公开(公告)日：2018-11-29

申请号：PCT/GB2018/051415

申请日：2018-05-24

Applicant: AMMUN LIMITED

Inventor： NICOLSON, Tamara

IPC: A61K31/00 ,  A61K38/43 ,  C12N5/10 ,  A61P3/00

Abstract: The present invention relates to the use of glutamine synthetase as a protein therapy (such as enzyme replacement protein therapy) for the treatment of hyperammonemia. In particular the invention relates to the systemic administration of glutamine synthetase. The glutamine synthetase may be provided in conjugated or fusion form, to increase its half-life in the circulation. Also provided is a pharmaceutical composition comprising glutamine synthetase. The invention also relates to the uses, methods and compositions involving a combination of the glutamine synthetase protein and an ammonia lowering agent, such as a nitrogen scavenger.

公开(公告)号：WO2018191666A1

公开(公告)日：2018-10-18

申请号：PCT/US2018/027568

申请日：2018-04-13

Applicant: REGENXBIO INC.

Inventor： YOO, Stephen ,  REINHARDT, Rickey, Robert ,  SIMPSON, Curran, Matthew ,  WU, Zhuchun

IPC: A61K38/43 ,  A61K47/26 ,  A61K48/00 ,  A61P25/00 ,  C12N15/86

Abstract: Compositions and methods are described for the delivery of recombinant human iduronate-2-sulfatase (IDS) produced by human neuronal or glial cells to the cerebrospinal fluid of the central nervous system (CNS) of a human subject diagnosed with mucopolysaccharidosis II (MPS II).

公开(公告)号：WO2018169811A1

公开(公告)日：2018-09-20

申请号：PCT/US2018/021897

申请日：2018-03-11

Applicant: SELECTA BIOSCIENCES, INC.

Inventor： JOHNSTON, Lloyd

IPC: A61K45/00 ,  A61K38/43 ,  A61P29/00

Abstract: Provided herein are methods and compositions, such as kits, related to compositions comprising synthetic nanocarriers comprising an immunosuppressant and compositions comprising an uricase and a composition comprising an anti-inflammatory therapeutic. Also provided herein are methods and compositions for the treatment of subjects in need of administration or treatment with the uricase.

公开(公告)号：WO2018161017A1

公开(公告)日：2018-09-07

申请号：PCT/US2018/020741

申请日：2018-03-02

Applicant: OBSIDIAN THERAPEUTICS, INC.

Inventor： SURI, Vipin ,  BRISKIN, Michael Joseph ,  DOLINSKI, Brian ,  ELPEK, Kutlu Goksu ,  LI, Dan Jun ,  HELLER, Scott Francis ,  OLS, Michelle Lynn ,  SUN, Dexue ,  KOSMIDER, Nicole ,  KULKARNI, Abhishek ,  BALAKRISHNAN, Vijaya ,  EZELL, Tucker

IPC: C07K14/505 ,  C07K14/705 ,  C07K14/725 ,  C07K16/18 ,  C07K16/28 ,  C07K16/30 ,  C07K16/46 ,  A61K38/17 ,  A61K38/43 ,  A61K35/14

Abstract: The present invention provides biocircuit systems, effector modules and compositions for cancer immunotherapy. Methods for inducing anti-cancer immune responses in a subject are also provided.

公开(公告)号：WO2018145147A1

公开(公告)日：2018-08-16

申请号：PCT/AU2018/050078

申请日：2018-02-06

Applicant: CHIN, David

Inventor： CHIN, David

IPC: A61K38/49 ,  A61K38/36 ,  A61K38/43 ,  A61P17/02 ,  A61P19/04

CPC classification number: A61P17/02 ,  A61K9/00 ,  A61K9/0019 ,  A61K31/573 ,  A61K31/58 ,  A61K38/4846 ,  A61K38/4853 ,  A61K38/49 ,  A61K45/06 ,  A61P19/04 ,  C12Y304/21031 ,  C12Y304/21034 ,  C12Y304/21038 ,  C12Y304/21068 ,  A61K2300/00

Abstract: The present invention relates to a method of treating a fibrotic disease condition characterized by excessive collagen formation in a subject including a human. The fibrotic disease includes fascial fibromatosis and a keloid scar. The method comprises the administration of an activator of a collagenase pathway such as tissue plasminogen activator (tPA), urokinase plasminogen activator (uPA), kallikrein or Factor XII or a functional analog thereof. If the fibrotic condition is a fascial fibromatosis, the activator is given in combination with a steroid. The present invention is further directed to formulations and protocols for the treatment of fibrotic conditions.