43 patents
Utility
Methods of Treating Achondroplasia
11 Jan 24
Provided herein are methods of treating a skeletal dysplasia such as hypochondroplasia or achondroplasia in a pediatric patient by administering to the patient infigratinib or a pharmaceutically acceptable salt thereof.
Riccardo Panicucci, Susan Arangio, Carl Dambkowski, Michael Henderson, Daniela Rogoff, Michael Monteith, Lihua Zhang
Filed: 17 Dec 21
Utility
Methods of Preparing Gene Therapies
4 Jan 24
The disclosure provides methods for purifying gene therapy compositions using anion exchange chromatography.
Ashton LAVOIE, Jeffrey Zugates
Filed: 17 May 23
Utility
Collagen 7 Compositions and Methods of Using the Same
28 Dec 23
The present disclosure relates to compositions comprising collagen 7 and methods of using the same to treat DEB.
Igor QUINONES-GARCIA, Lin Guey, Kris Lowe, Vinh Nguyen, Bing He, Amey Bandekar, Sujit Basu
Filed: 15 Feb 23
Utility
Topical Phosphoinositide 3-KINASE Inhibitors
28 Dec 23
Ahmed F. ABDEL-MAGID, Agis KYDONIEUS, Thomas ROSSI, Hock S. TAN
Filed: 10 Apr 23
Utility
Methods of Treating Disorders Associated with Castor
12 Oct 23
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.
Suzanne JACKOWSKI, Charles O. ROCK, Richard E. LEE, Lalit Kumar SHARMA, Mi Kyung YUN, Chitra SUBRAMANIAN, Rajendra P. TANGALLAPALLY, Anne V. EDWARDS, Robert ZAMBONI, T. Jagadeeswar REDDY, Jiuyu LIU
Filed: 6 Dec 22
Utility
Topical phosphoinositide 3-kinase inhibitors
30 May 23
Ahmed F. Abdel-Magid, Agis Kydonieus, Thomas Rossi, Hock S. Tan
Filed: 5 Feb 20
Utility
Triazole Glycolate Oxidase Inhibitors
11 May 23
The present invention provides triazole carboxylic acids and related compounds, as well as pharmaceutically acceptable salts thereof, which are useful as glycolate oxidase inhibitors.
Hans MAAG, Miguel Xavier FERNANDES, Robert ZAMBONI, Elham AKBARIROMANI, Marc-Andre BEAULIEU, Yves LEBLANC, Pallavi THAKUR
Filed: 17 Jun 22
Utility
Benzoxazinone compounds as KLK5/7 dual inhibitors
9 May 23
Andreas Betz, Robert Zamboni
Filed: 30 Sep 20
Utility
Processes for Preparing Triazole Glycolate Oxidase Inhibitors
27 Apr 23
Elham AKBARIROMANI, Pooran CHAND, Sunil Kumar CHOUDHARY, Miguel Xavier FERNANDES, Yves LEBLANC, Hans MAAG, Omkar REVU, Pavan SHEKHAWAT
Filed: 7 Jan 21
Utility
Compounds and Methods of Use
9 Feb 23
This present disclosure relates to compounds with ferroptosis inducing activity, a method of treating a subject with cancer with the compounds, and combination treatments with a second therapeutic agent.
Chun Jiang, Ruihong Chen, Anjali Pandey, Biswajit Kalita, Athisayamani Jeyaraj Duraiswamy
Filed: 6 Jul 21
Utility
Methods of treating disorders associated with castor
10 Jan 23
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.
Suzanne Jackowski, Charles O. Rock, Richard E. Lee, Lalit Kumar Sharma, Mi Kyung Yun, Chitra Subramanian, Rajendra P. Tangallapally, Anne V. Edwards, Robert Zamboni, T. Jagadeeswar Reddy, Jiuyu Liu
Filed: 26 Dec 18
Utility
Detection of Dystroglycan
15 Sep 22
Provided are methods of determining an amount of alpha-dystroglycan (αDG) in a sample, determining an amount of the glycosylated form of αDG in the sample, and determining a ratio of the amount of the glycosylated form of αDG to the amount of αDG in the sample.
Hector M. RODRIGUEZ
Filed: 24 Feb 22
Utility
Triazole glycolate oxidase inhibitors
2 Aug 22
The present invention provides triazole carboxylic acids and related compounds, as well as pharmaceutically acceptable salts thereof, which are useful as glycolate oxidase inhibitors.
Hans Maag, Miguel Xavier Fernandes, Robert Zamboni, Elham Akbariromani, Marc-Andre Beaulieu, Yves Leblanc, Pallavi Thakur
Filed: 5 Jul 19
Utility
Methods of Treating Cholangiocarcinoma
28 Jul 22
Provided herein are methods of treating advanced or metastatic cholangiocarcinoma in a patient by administering to the patient infigratinib or a pharmaceutically acceptable salt thereof, wherein the patient has progression of the cholangiocarcinoma after previous administration of another therapy.
Riccardo Panicucci, Michael Monteith, Gang Li, Susan Arangio, Craig Berman, Michael Howland, Daniel Mulreany, Carl Dambkowski
Filed: 28 May 20
Utility
Methods of Treating Urinary System Cancers
28 Jul 22
Provided herein are methods of treating an upper tract urothelial carcinoma in a patient by administering to the patient infigratinib or a pharmaceutically acceptable salt thereof.
Riccardo Panicucci, Susan Arangio, Craig Berman, Michael Monteith, Harris Soifer, Gang Li, Carl Dambkowski, Daniel Mulreany
Filed: 29 May 20
Utility
Benzimidazole and Hydrogenated Carbazole Derivatives As GPX4 Inhibitors
21 Jul 22
This present disclosure relates to compounds with ferroptosis inducing activity, a method of treating a subject with cancer with the compounds, and combination treatments with a second therapeutic agent.
Anjali Pandey, Chun Jiang, Biswajit Kalita
Filed: 26 Aug 20
Utility
Pyrido-indole Analogues As GPX4 Inhibitors
21 Jul 22
This present disclosure relates to compounds with ferroptosis inducing activity, a method of treating a subject with cancer with the compounds, and combination treatments with a second therapeutic agent.
Anjali Pandey, Chun Jiang, Ruihong Chen, Biswajit Kalita, Athisayamani Jeyaraj Duraiswamy
Filed: 26 Aug 20
Utility
Systems and Methods for Producing Collagen 7 Compositions
9 Jun 22
The present disclosure provides production systems and host cells to produce collagen 7 compositions comprising recombinant collagen 7 and/or functional variants thereof.
Mark Desouza, Malini Viswanathan, Pierra-Alain Girod, Alexandre Regamey, Valerie Le Fourn, David Calabrese, John Gill, Megan Gottlieb, Amie Lundquist, Anna Tchoudakova, George Enriquez, Rahul Chelikani, Thomas Gagliardi, Tatiana Laivins, Hang Yuan
Filed: 27 Mar 20
Utility
Treatment Methods of Triphenyl Calcilytic Compounds
24 Mar 22
The present disclosure provides a method of treating an autosomal dominant hypocalcemia type 1 (ADH1) with a therapeutically effective amount of a compound of formula (I), in particular CLTX-305, wherein the therapeutically effective amount of the compound increases a blood calcium concentration (cCa) to a range of about 7.5 mg/dL to about 10.5 mg/dL, such as about 8.5 mg/dL to about 10.5 mg/dL.
Simon BRUCE, Jonathan FOX, Ramei SANI-GROSSO, Ali KOMEYLI, Ananth SRIDHAR, Mary Scott ROBERTS
Filed: 16 Sep 21
Utility
PTPN11 Inhibitors
10 Feb 22
The present invention relates to compounds which are useful as inhibitors of PTPN11 for the treatment or prevention of cancer and other PTP-mediated diseases.
Philip JONES, Barbara CZAKO, Christopher L. CARROLL, Pijus MANDAL, Jason CROSS
Filed: 20 Jul 21