33 patents
Utility
RNAi agents for inhibiting expression of DUX4, compositions thereof, and methods of use
19 Dec 23
Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a double homeobox 4 (DUX4) gene.
Zhi-Ming Ding, Jonathan Van Dyke, Xiaokai Li, Anthony Nicholas, Casi M. Schienebeck, Tao Pei, Zhao Xu, Teng Ai, Susan Phan, Susan Ramos-Hunter
Filed: 9 Mar 23
Utility
Compositions and methods for inhibiting gene expression of Hif2alpha
12 Dec 23
RNA interference (RNAi) triggers and RNAi trigger conjugates for inhibiting the expression of Hif2α (EPAS1) gene are described.
So Wong, David L. Lewis, David B. Rozema, Darren H. Wakefield, Steven B. Kanner, Weijun Cheng, Lauren J. Almeida, Andrei V. Blokhin, Jeffrey C. Carlson, Anthony L. Nicholas, Aaron Almeida, Jonathan D. Benson, Justin Woods
Filed: 19 Feb 21
Utility
RNAi agents for inhibiting expression of xanthine dehydrogenase (XDH), pharmaceutical compositions thereof, and methods of use
18 Apr 23
The present disclosure relates to RNAi agents, e.g., double stranded RNAi agents, able to inhibit xanthine dehydrogenase (XDH) gene expression.
Anthony Nicholas, Tao Pei, Zhao Xu, Daniel Braas, Zhi-Ming Ding
Filed: 19 May 22
Utility
Integrin ligands and uses thereof
7 Mar 23
Zhen Li, Xiaokai Li, Erik W. Bush, Rui Zhu, Dongxu Shu, Jonathan Benson, Patrick Shao, Matthew Fowler-Watters
Filed: 31 Oct 18
Utility
RNAi agents for hepatitis B virus infection
28 Feb 23
Described are compositions and methods for inhibition of Hepatitis B virus gene expression.
Zhen Li, Rui Zhu, Christine I. Wooddell, Bruce D. Given, Tao Pei, David L. Lewis, Lauren J. Almeida, David B. Rozema, Darren H. Wakefield
Filed: 11 Aug 20
Utility
RNAi agents for inhibiting expression of xanthine dehydrogenase (XDH), pharmaceutical compositions thereof, and methods of use
10 Jan 23
The present disclosure relates to RNAi agents, e.g., double stranded RNAi agents, able to inhibit xanthine dehydrogenase (XDH) gene expression.
Anthony Nicholas, Tao Pei, Zhao Xu, Daniel Braas, Zhi-Ming Ding
Filed: 19 May 22
Utility
RNAi therapy for hepatitis B virus infection
27 Dec 22
Described are compositions and methods for inhibition of Hepatitis B virus gene expression.
Christine I. Wooddell, David B. Rozema, David L. Lewis, Darren H. Wakefield, Lauren J. Almeida
Filed: 9 Sep 20
Utility
RNAi agents for Hepatitis B virus infection
6 Dec 22
Described are compositions and methods for inhibition of Hepatitis B virus gene expression.
Zhen Li, Rui Zhu, Christine I. Wooddell, Bruce D. Given, Tao Pei, David L. Lewis, Lauren J. Almeida, David B. Rozema, Darren H. Wakefield
Filed: 17 Nov 21
Utility
RNAi agents and compositions for inhibiting expression of Asialoglycoprotein receptor 1
8 Nov 22
Described herein are compositions and methods for inhibition of Asialoglycoprotein receptor 1 (ASGR1) gene expression.
Zhen Li, Tao Pei, Rui Zhu, Bruce D. Given, Stacey Melquist
Filed: 16 Oct 18
Utility
Compositions and methods for inhibiting gene expression of alpha-1 AntiTrypsin
12 Jul 22
The invention relates to a RNA interference triggers for inhibiting the expression of an AAT gene through the mechanism of RNA interference.
Christine I Wooddell, David L Lewis, Darren H Wakefield, Lauren Almeida, Steven B Kanner
Filed: 31 May 19
Utility
Organic compositions to treat EPAS1-related diseases
1 Mar 22
The present disclosure relates to methods of treating EPAS1-related diseases such as cancer, metastases, astrocytoma, bladder cancer, breast cancer, chondrosarcoma, colorectal carcinoma, gastric carcinoma, glioblastoma, head and neck squamous cell carcinoma, hepatocellular carcinoma, lung adenocarcinoma, neuroblastoma, non-small cell lung cancer, melanoma, multiple myeloma, ovarian cancer, rectal cancer, renal cancer, clear cell renal cell carcinoma (and metastases of this and other cancers), gingivitis, psoriasis, Kaposi's sarcoma-associated herpesvirus, preeclampsia, inflammation, chronic inflammation, neovascular diseases, and rheumatoid arthritis, using a therapeutically effective amount of a RNAi agent to EPAS1.
Brian Bettencourt, Shanthi Ganesh, Elizabeth George, Dieter Huesken, Stuart Milstein, Jonathan Solomon, Emily Thomas, Ivanka Toudjarska, Jennifer Tullai, Jan Weiler
Filed: 21 Jan 20
Utility
RNAi agents and compositions for inhibiting expression of apolipoprotein C-III (APOC3)
4 Jan 22
The present disclosure relates to RNAi agents, e.g., double stranded RNAi agents, capable of inhibiting Apolipoprotein C-III (also called APOC3, apoC-III, APOC-III, and APO C-III) gene expression, and compositions that include APOC3 RNAi agents.
Zhen Li, Rui Zhu, Tao Pei, Steven Kanner, So Wong
Filed: 31 Jan 20
Utility
RNAi agents for inhibiting expression of alpha-ENaC and methods of use
4 Jan 22
Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of an alpha-ENaC (SCNN1A) gene.
Zhen Li, Rui Zhu, Tao Pei, Anthony Nicholas, Erik W Bush
Filed: 31 Jan 20
Utility
RNAi inhibition of alpha-ENaC expression
28 Dec 21
The invention relates to compositions and methods for modulating the expression of alpha-ENaC, and more particularly to the downregulation of alpha-ENaC expression by chemically modified oligonucleotides.
Gino Van Heeke, Emma Hickman, Henry Luke Danahay, Pamela Tan, Anke Geick, Hans-Peter Vornlocher
Filed: 24 Jan 20
Utility
Alpha-1 antitrypsin (AAT) RNAi agents, compositions including AAT RNAi agents, and methods of use
21 Dec 21
RNAi agents for inhibiting the expression of the alpha-1 antitrypsin (AAT) gene, compositions including AAT RNAi agents, and methods of use are described.
Zhen Li, Rui Zhu, Christine I. Wooddell, Tao Pei
Filed: 12 Sep 19
Utility
Alpha-v beta-6 integrin ligands and uses thereof
23 Nov 21
Integrin ligands having serum stability and affinity for αvβ6 integrins are described.
Aaron Almeida, Zhen Li, Erik W. Bush, Tao Pei, Agnieszka Glebocka, Anthony Nicholas, Jeffrey Carlson, Matthew Fowler-Watters
Filed: 30 Apr 19
Utility
Targeting ligands
16 Nov 21
Described are novel targeting ligands that may be linked to compounds, such therapeutic compounds that are useful in directing the compounds to the in vivo target.
Zhen Li, Tao Pei, Agnieszka Glebocka, Michael Lawler, Fred Fleitz, Erich Altenhofer, Pankaj Kumar
Filed: 10 Apr 19
Utility
Organic compositions to treat HSF1-related diseases
17 Aug 21
The present disclosure relates to methods of treating heat shock factor 1 (HSF1)-related diseases such as cancer and viral diseases, using a therapeutically effective amount of a RNAi agent to HSF.
Gregory Hinkle, Satyanarayana Kuchimanchi, Stuart Milstein, Markus Warmuth, Wenlai Zhou, Ping Zhu, Tracy S. Zimmermann
Filed: 14 May 19
Utility
5′-cyclo-phosphonate modified nucleotides
3 Aug 21
Zhen Li, Tao Pei, Michael Lawler
Filed: 3 Dec 18
Utility
Organic compositions to treat APOC3-related diseases
20 Jul 21
The present disclosure relates to compositions and methods for treating APOC3-related diseases such as: hypertriglyceridemia (e.g., Type V Hypertriglyceridemia), abnormal lipid metabolism, abnormal cholesterol metabolism, atherosclerosis, hyperlipidemia, diabetes, including Type 2 diabetes, obesity, cardiovascular disease, and coronary artery disease, among other disorders relating to abnormal metabolism or otherwise, using a therapeutically effective amount of a RNAi agent to APOC3.
Jan Weiler, William Chutkow, Jeremy Lee Baryza, Andrew Krueger, Junping Zhao
Filed: 6 Feb 19