Abstract: In one aspect, described herein is a recognition element for splicing modifier (REMS) that can be recognized by a compound provided herein. In another aspect, described herein are methods for modulating the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene contains a REMS, and the methods utilizing a compound described herein. More particularly, described herein are methods for modulating the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene comprises a REMS, and the methods utilizing a compound described herein. In another aspect, provided herein are artificial gene constructs comprising a REMS, and uses of those artificial gene constructs to modulate functional protein production.
Abstract: Amine substituted triazine compounds and forms thereof that inhibit the function and reduce the level of B-cell specific Moloney murine leukemia virus integration site 1 (Bmi-1) protein and methods for their use to inhibit Bmi-1 function and reduce the level of Bmi-1 to treat a cancer mediated by Bmi-1 are described herein.
Type:
Grant
Filed:
November 21, 2013
Date of Patent:
May 22, 2018
Assignee:
PTC THERAPEUTICS, INC.
Inventors:
Hiroo Koyama, Ramil Baiazitov, Wu Du, Chang-sun Lee, Young-Choon Moon, Steven D. Paget, Hongyu Ren, Nadiya Sydorenko
Abstract: The present invention provides compounds of formula (I) wherein A, R1, R2 and R3 are as described herein, as well as pharmaceutically acceptable salts thereof. Further the present invention is concerned with the manufacture of the compounds of formula (I), pharmaceutical compositions comprising them and their use as medicaments.
Type:
Grant
Filed:
November 14, 2016
Date of Patent:
May 15, 2018
Assignees:
HOFFMANN-LA ROCHE INC., PTC THERAPEUTICS, INC.
Inventors:
Hasane Ratni, Luke Green, Marla L. Weetall, Nikolai A. Naryshkin
Abstract: A method, non-transitory computer readable medium, and data manager computing device comprises retrieving a time series data of a monitored asset based on one or more tags in response to a request. Further, a heterogeneous data based on the one or more tags is retrieved. Furthermore, a cause of an anomaly period in retrieved time series data of the monitored asset is determined based on the retrieved heterogeneous data. Finally, the determined cause of the anomaly period in the time series data in the monitored asset is provided.
Abstract: A computer-implemented method processes a simple object access protocol (SOAP) command. The method includes interpreting an XML script to perform a function contained in the XML script, the XML script containing the SOAP command, parsing the SOAP command from the XML script, and passing the SOAP command to a SOAP interpreter for execution.
Abstract: A system and method of message routing messages via connections in a distributed computing environment. The method includes providing a platform server, a set of intermediary servers, and a set of edge servers. The method includes receiving, by a port at the platform server, a first data message from a first end-point device over a first persistent connection. The message has been routed through a an intermediate server over a second persistent connection. The method includes receiving a second data message from a second end-point device over a third persistent connection where the second data message has been routed through another intermediate server over a fourth persistent connection. The method includes servicing, by a processor at the platform server, the first data message and the second data message where the intermediate servers manage connectivity between the end-point devices and the platform servers.
Type:
Grant
Filed:
March 21, 2014
Date of Patent:
May 1, 2018
Assignee:
PTC Inc.
Inventors:
Mike Mahoney, Bob DeRemer, Rick Bullotta
Abstract: Provided herein are compounds of Formula (I): and forms thereof, including compositions thereof and uses therewith for treating spinal muscular atrophy.
Type:
Grant
Filed:
August 26, 2016
Date of Patent:
January 30, 2018
Assignees:
PTC Therapeutics, Inc., F. Hoffmann-La Roche AG
Inventors:
Hongyan Qi, Soongyu Choi, Amal Dakka, Gary Mitchell Karp, Jana Narasimhan, Nikolai Naryshkin, Anthony A. Turpoff, Marla L. Weetall, Ellen Welch, Matthew G. Woll, Tianle Yang, Nanjing Zhang, Xiaoyan Zhang, Xin Zhao, Luke Green, Emmanuel Pinard, Hasane Ratni
Abstract: The present invention relates to specific doses of and dosing regimens for using a 1,2,4-oxadiazole benzoic acid compound in treating or preventing diseases associated with nonsense mutations. In particular, the invention relates to specific doses and dosing regimens for the use of 3-[5-(2-fluoro-phenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid in mammals having diseases associated with nonsense mutations.
Abstract: Provided herein are pharmaceutical compositions, which comprise a 1,2,4-oxadiazole benzoic acid or a pharmaceutically acceptable salt thereof. Further provided herein are certain pharmaceutically acceptable salts of a 1,2,4-oxadiazole benzoic acid and methods for making the same. Further provided herein are methods of treating or preventing a disease associated with a nonsense mutation or a premature stop codon, comprising administering such pharmaceutical compositions or pharmaceutically acceptable salts to a patient having a disease associated with a nonsense mutation or a premature stop codon.
Type:
Grant
Filed:
March 5, 2015
Date of Patent:
January 23, 2018
Assignee:
PTC Therapeutics, Inc.
Inventors:
Marla L. Weetall, Ellen Welch, Mandar V. Dali, James Takasugi
Abstract: Novel 1,2,4-oxadiazole benzoic acid compounds, methods of using and pharmaceutical compositions comprising an 1,2,4-oxadiazole benzoic acid derivative are disclosed. The methods include methods of treating or preventing a disease ameliorated by modulation of premature translation termination or nonsense-mediated mRNA decay, or ameliorating one or more symptoms associated therewith.
Type:
Grant
Filed:
November 13, 2015
Date of Patent:
January 9, 2018
Assignee:
PTC Therapeutics, Inc.
Inventors:
Gary Mitchell Karp, Seongwoo Hwang, Guangming Chen, Neil Gregory Almstead, Young-Choon Moon
Abstract: In a first aspect, the present invention relates to new compositions comprising new bacteriophages allowing the prophylactic or therapeutic treatment of Salmonella infections in mammals and birds. Moreover, the present invention relates to the method for fighting Salmonella comprising the step of treating matter suspected to be afflicted with Salmonella with the bacteriophages according to the present invention. Moreover, the present invention relates to a method of treating or preventing Salmonella infection, in particular, in livestock.
Abstract: An integrated circuit packaging system and method of manufacture thereof including: providing a substrate; forming contact pads on top of the substrate; forming a protection layer on top of the contact pads and the substrate; exposing the contact pads from the protection layer; printing under bump metallization (UBM) layers over the exposed contact pads extended over the protection layer with conductive inks; and forming bumps on top of the under bump metallization layers. It also including: printing an adhesion layer using conductive ink, wherein the adhesion layer comprises interconnected adhesion layer pads; forming additional under bump metallization (UBM) layers and bumps on top of the adhesion layer pads utilizing an electro-deposition process; and removing connections among the interconnected adhesion layer pads.
Type:
Grant
Filed:
November 30, 2015
Date of Patent:
January 9, 2018
Assignee:
STATS ChipPAC Ptc. Ltd.
Inventors:
Il Kwon Shim, Kyung Moon Kim, HeeJo Chi, JunMo Koo, Bartholomew Liao Chung Foh, Zigmund Ramirez Camacho
Abstract: This technology generates a plurality of instances of things each including a dynamically generated interface structure and services associated with and properties of the corresponding one of the things. The services associated with and the properties of one of the plurality of instances of things for a selected one of the plurality of instances of things are retrieved and provided. A service definition for a selected one of the services is retrieved and provided based on the retrieved and provided services associated with and properties for the selected one of the plurality of instances of things. A requested consumption call for the selected one of the services is executed based on the retrieved service definition. A defined result set for the executed consumption call is provided in the dynamically generated interface structure associated with the requested one of the plurality of instances of things.
Abstract: A system and method thereof of a communication device. The device includes a port configured to receive a plurality of binary data streams having a binary header and a binary body. The device includes a memory storing a first structural description of the binary header and the binary body and a second structural description of a metadata construct of the message. The device includes a processor configured to parse a received binary data stream using the first structural description to determine the binary header and the binary body. The processor parses the binary body using the second structural description to determine the one or more groups of description values forming the metadata construct where the processor uses a portion of the determined description values of the metadata construct to determine the one or more groups of data values of the message construct.
Type:
Grant
Filed:
March 21, 2014
Date of Patent:
September 12, 2017
Assignee:
PTC Inc.
Inventors:
Rick Bullotta, John Canosa, Bob DeRemer, Mike Mahoney
Abstract: The present invention relates to specific doses of and dosing regimens for using a 1,2,4-oxadiazole benzoic acid compound in treating or preventing diseases associated with nonsense mutations. In particular, the invention relates to specific doses and dosing regimens for the use of 3-[5-(2-fluoro-phenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid in mammals having diseases associated with nonsense mutations.
Abstract: The present invention relates to methods, compounds, and compositions for treating or preventing diseases associated with nonsense mutations in an mRNA by administering the compounds or compositions of the present invention. More particularly, the present invention relates to methods, compounds, and compositions for suppressing premature translation termination associated with a nonsense mutation in an mRNA.
Type:
Application
Filed:
March 31, 2017
Publication date:
July 20, 2017
Applicant:
PTC Therapeutics, Inc.
Inventors:
Neil Almstead, Guangming Chen, Gary M. Karp, Ellen Welch, Richard Wilde, Jeffrey A. Campbell
Abstract: A process performed on a server includes configuring the server to enable script for a Web service to be defined dynamically, where the Web service includes an application program interface (API) for enabling access by, and interaction with, a computer program executing on a device other than the server. The process also includes compiling the script to produce machine-executable code for the Web service, receiving a call from the computer program to the Web service, executing the machine-executable code in response to the call to produce an output, and sending the output to the device.
Abstract: A method manages configurations of devices in a system that communicates information between a device and an enterprise. The method includes building a defined configuration for a device type. The defined configuration includes a set of value requirements. An actual configuration having values associated with the device is compared to the defined configurations. The actual configuration and defined configuration are stored in a database of the enterprise. The method also includes determining, in the enterprise, if the values of actual configuration match the corresponding value requirements of the defined configurations. The method runs business logic associated with the device based on a result from the step of determining if the values of the actual configuration match the corresponding values of the defined configurations. The matched configurations are stored for subsequent use.
Type:
Grant
Filed:
April 6, 2016
Date of Patent:
July 18, 2017
Assignee:
PTC, Inc.
Inventors:
John Louis Taylor, Timothy A. Rochte, Kiran Bhageshpur, David Patrick Hart
Abstract: The present invention provides compounds of formula (I) wherein A, R1, R2 and R3 are as described herein, as well as pharmaceutically acceptable salts thereof. Further the present invention is concerned with the manufacture of the compounds of formula (I), pharmaceutical compositions comprising them and their use as medicaments.
Type:
Application
Filed:
November 14, 2016
Publication date:
July 13, 2017
Applicants:
Hoffmann-La Roche Inc., PTC Therapeutics, Inc.
Inventors:
Hasane Ratni, Luke Green, Marla L. Weetall, Nikolai A. Naryshkin