Abstract: The present application provides materials and methods for treating a patient with Usher Syndrome Type 2A, both ex vivo and in vivo; materials and methods for editing a USH2A gene in a human cell; materials and methods for editing an USH2A gene containing an IVS40 mutation; materials and methods for treating a patient with an USH2A gene containing an IVS40 mutation; and a method for deleting a sequence comprising an IVS40 mutation within a USH2A gene of a cell. The present application also provides one or more gRNAs or sgRNAs for editing an USH2A gene containing an IVS40 mutation. The present application provides a therapeutic for treating a patient with Usher Syndrome Type 2A. The present application also provides a kit for treating a patient with Usher Syndrome Type 2A.
Abstract: Provided include compositions, methods, and systems for modulating the expression, function, and/or activity of a target gene, for example a blood-clotting protein such as Factor VIII (FVIII), in a cell by genome editing. Also provided include compositions, methods, and systems for treating a subject having or suspected of having a disorder or health condition, e.g., Hemophilia A, employing ex vivo and/or in vivo genome editing.
Abstract: Aspects of this invention, inter alia, relate to novel systems for targeting, editing or manipulating DNA in a cell, using novel synthetic RNA-guided nucleases (sRGNs). The sRGNs are derived from wildtype or parental small type II CRISPR Cas9 endonucleases.
Inventors:
Andre Cohnen, Moritz Schmidt, Wayne Coco, Michael Biag Gamalinda, Ashish Gupta, Christian Pitzler, Florian Richter, Jan Tebbe, Christopher Cheng, Ryo Takeuchi, Caroline W. Reiss
Abstract: The invention relates to novel amides of acetic and propionic acids, methods for production and use thereof for the production of medicaments for the treatment and/or prophylaxis of diseases and for improving perception, concentration, learning ability and memory.
Type:
Application
Filed:
August 16, 2013
Publication date:
December 19, 2013
Applicant:
BAYER HEALTHCARE AG
Inventors:
Joachim Luithle, Frank-Gerhard Bob, Christina Erb, Katrin Schnizler, Timo Flessner, Marja Van Kampen, Christoph Methfessel
Abstract: The invention relates to novel amides of acetic and propionic acids, methods for production and use thereof for the production of medicaments for the treatment and/or prophylaxis of diseases and for improving perception, concentration, learning ability and memory.
Type:
Grant
Filed:
June 29, 2009
Date of Patent:
August 27, 2013
Assignee:
Bayer Healthcare AG
Inventors:
Joachim Luithle, Frank-Gerhard Böb, Christina Erb, Katrin Schnitzler, Timo Flessner, Marja Van Kampen, Christoph Methfessel
Abstract: An aqueous or powder composition includes anti-gram-negative antibiotic or salt thereof being present at an amount ranging from about 100 mg/ml to about 200 mg/ml. Another aqueous or powder composition includes anti-gram-positive antibiotic or salt thereof being present at a concentration ranging from about 0.6 to about 0.9 of the water solubility limit, at 25° C. and 1.0 atmosphere, of the anti-gram-positive antibiotic or salt thereof. Other embodiments include unit doses, kits, and methods.
Abstract: The invention relates to N-biarylamides useful for the prophylaxis and/or treatment of impairments of perception, concentration, learning and/or memory, and to processes for preparing them, and pharmaceutical compositions containing them.
Type:
Application
Filed:
September 19, 2011
Publication date:
June 21, 2012
Applicant:
Bayer HealthCare AG
Inventors:
Timo FLEßNER, Frank-Gerhard BÖß, Frank-Thorsten HAFNER, Joachim LUITHLE, Christoph METHFESSEL, Leila TELAN
Abstract: A process for the quantitative optical analysis of fluorescently labeled biological cells involves contacting a cell layer on a transparent support at the bottom of a reaction vessel with a solution containing the fluorescent dye. This process can also be used for improving the sensitivity in the quantitative optical analysis of a luminescent biological cell layer. Analogously, these process principles can also be used in receptor studies for the masking of the interfering background radiation in the quantitative optical analysis of fluorescently or luminescently labelled reaction components. In this case, a receptor layer at the bottom of a reaction vessel is in contact with a solution in which a fluorescent or luminescent ligand is dissolved.
Type:
Grant
Filed:
September 28, 2001
Date of Patent:
May 15, 2012
Assignee:
Bayer Healthcare AG
Inventors:
Thoams Krahn, Wolfgang Paffhausen, Andreas Schade, Martin Bechem, Delf Schmidt
Abstract: A method for assessing the state of Alzheimer's disease in patients is disclosed. A method for monitoring the progression of Alzheimer's disease in patients is also disclosed. The method applies detection of specific peptide markers, e.g., using mass spectrometric analysis.
Abstract: This invention relates to novel compounds of formula (I). Formula (I) in which the variable groups are as defined in the specification and claims, to pharmaceutical compositions containing them, and to a method of treatment using them for treatment of cancer.
Type:
Application
Filed:
November 2, 2006
Publication date:
December 1, 2011
Applicant:
BAYER HEALTHCARE AG
Inventors:
Stephen O'Connor, Jacques Dumas, Wendy Lee, Julie Dixon, David Cantin, David Gunn, Jennifer Burke, Barton Phillips, Derek Lowe, Tatiana Shelekhin, Gan Wang, Xin Ma, Shihong Ying, Andrea McClure, Furahi Achebe, Mario Lobell, Frederick Ehrgott, Christiana Iwuagwu, Kyle Parcella
Abstract: The present application relates to a novel cyclopenta[b]benzofuran derivatives, processes for their preparation and their use for the manufacture of medicaments, in particular for the prophylaxis and/or therapy of acute or chronic disorders characterized by elevated cellular stress, by local or systemic inflammatory processes or by hyperproliferation.
Type:
Grant
Filed:
May 14, 2005
Date of Patent:
October 4, 2011
Assignee:
Bayer HealthCare AG
Inventors:
Nicole Diedrichs, Thomas Fahrig, Irene Gerlach, Jaques Ragot, Joachim Schuhmacher, Kai Thede, Ervin Horváth
Abstract: The invention provides a human PRCP which is associated with the cardiovascular diseases, hematological diseases, neurological diseases and cancer. The invention also provides assays for the identification of compounds useful in the treatment or prevention of cardiovascular diseases, hematological diseases, neurological diseases and cancer. The invention also features compounds which bind to and/or activate or inhibit the activity of PRCP as well as pharmaceutical compositions comprising such compounds.
Type:
Application
Filed:
May 25, 2007
Publication date:
February 10, 2011
Applicant:
BAYER HEALTHCARE AG
Inventors:
Stefan Golz, Ulf Brüggemeier, Adreas Geerts
Abstract: This invention relates to compounds of Formula (I), wherein the variables are as disclosed in the specification, to pharmaceutical compositions containing them, to methods of making the compounds and pharmaceutical compositions, and to methods of using the compounds and pharmaceutical compositions for treating or preventing disorders, in particular cancer.
Type:
Application
Filed:
March 20, 2007
Publication date:
November 25, 2010
Applicant:
BAYER HEALTHCARE AG
Inventors:
Chengzhi Zhang, Kanwar Sidhu, Mario Lobell, Gaeten Ladouceur, Qian Zhao, Zheng Liu, Kristen Allegue, Chetan Darne, Jason Newcom
Abstract: The present invention relates to the use of selective factor Xa inhibitors, in particular of oxazolidinones of the formula (I) for the treatment and/or prophylaxis of microangiopathies and also their use for the production of medicaments for the treatment and/or prophylaxis of microangiopathies.
Abstract: The present invention relates to solid pharmaceutical dosage forms which can be administered orally and comprise 5-chloro-N-({(5S)-2-oxo-3-[4-(3-oxo-4-morpholinyl)phenyl]-1,3-oxazolidin-5-yl}methyl)-2-thiophenecarboxamide in amorphous form and/or thermodynamically metastable crystal modification and have rapid release of active ingredient, and to process for the production thereof, the use thereof as medicament, the use thereof for the prophylaxis, secondary prophylaxis and/or treatment of disorders, and to the use thereof for producing a medicament for the prophylaxis, secondary prophylaxis and/or treatment of disorders.
Abstract: The present invention relates to a novel polymorphic form and the amorphous form of 5-chloro-N-({(5S)-2-oxo-3-[4-(3-oxo-4-morpholinyl)-phenyl]-1,3-oxazolidin-5-yl}-methyl)-2-thiophene-carboxamide, processes for their preparation, medicaments comprising these forms, and their use in the control of diseases.
Type:
Application
Filed:
September 22, 2006
Publication date:
June 17, 2010
Applicant:
BAYER HEALTHCARE AG
Inventors:
Alfons Grunenberg, Jana Lenz, Gerhard Arnold Braun, Birgit Keil, Christian R. Thomas
Abstract: The present application relates to novel 3-tetrazolylindazole and 3-tetrazolylpyrazolo[3,4-b]-pyridine derivatives, processes for their preparation, their use alone or in combination for the treatment and/or prophylaxis of diseases, and their use for producing medicaments for the treatment and/or prophylaxis of diseases, especially for the treatment and/or prevention of cardiovascular disorders.
Type:
Application
Filed:
April 30, 2007
Publication date:
May 6, 2010
Applicant:
BAYER HEALTHCARE AG
Inventors:
Chantal Fürstner, Hartmut Schirok, Nils Griebenow, Joachim Mittendorf, Johannes-Peter Stasch, Frank Wunder
Abstract: The present invention relates to pharmaceutical compositions and combinations for treating, preventing or managing pulmonary hypertension comprising small molecule heterocyclic pharmaceuticals, and more particularly, substituted pyridines and pyridazines optionally combined with at least one additional therapeutic agent.
Type:
Application
Filed:
April 3, 2007
Publication date:
May 6, 2010
Applicant:
BAYER HEALTHCARE AG
Inventors:
Martina Klein, Peter Sandner, Reiner Frey, Bernd Riedl, Olaf Christensen
Abstract: The invention relates to substituted indazoles and methods for production thereof and use thereof for the production of medicinal products for the treatment and/or prophylaxis of diseases, especially of cardiovascular diseases, preferably of thromboembolic diseases.
Type:
Application
Filed:
July 2, 2007
Publication date:
April 29, 2010
Applicant:
BAYER HEALTHCARE AG
Inventors:
Stephan Siegel, Dirk Schneider, Anja Buchmüller, Elke Dittrich-Wengenroth, Chrisstoph Gerdes, Mark Jean Gnoth, Stefan Heitmeier, Martin Hendrix, Ulrich Rester, Uwe Saatmann