118 patents
Page 4 of 6
Utility
Carbohydrate conjugates as delivery agents for oligonucleotides
7 Sep 21
Muthiah Manoharan, Kallanthottathil G. Rajeev, Jayaprakash Nair, Martin Maier
Filed: 29 Apr 20
Utility
Methods of treating transthyretin (TTR) mediated amyloidosis
3 Aug 21
Disclosed herein are methods for reducing or arresting an increase in a Neuropathy Impairment Score (NIS) or a modified NIS (mNIS+7) in a human subject by administering an effective amount of a transthyretin (TTR)-inhibiting composition.
Brian Bettencourt
Filed: 3 Aug 18
Utility
Biodegradable lipids for the delivery of active agents
27 Jul 21
The present invention relates to a cationic lipid having one or more biodegradable groups located in a lipidic moiety (e.g., a hydrophobic chain) of the cationic lipid.
Martin Maier, Muthusamy Jayaraman, Akin Akinc, Shigeo Matsuda, Pachamuthu Kandasamy, Kallanthottathil G. Rajeev, Muthiah Manoharan
Filed: 23 Jul 19
Utility
Insulin-like growth factor binding protein, acid labile subunit (IGFALS) and insulin-like growth factor 1 (IGF-1) iRNA compositions and methods of use thereof
20 Jul 21
The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the insulin-like growth factor binding protein, acid labile subunit (IGFALS) gene or the insulin-like growth factor 1 (IGF-1) gene, methods of using such double stranded RNAi agents to inhibit expression of an IGFALS gene or an IGF-1 gene, and methods of treating subjects having an IGF system-associated disorder.
James Butler, Kevin Fitzgerald, Gregory Hinkle, Brian Bettencourt, Huilei Xu
Filed: 15 Oct 19
Utility
Hepatitis B virus (HBV) iRNA compositions and methods of use thereof
13 Jul 21
The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the hepatitis B virus (HBV) genome, and methods of using such RNAi agents to inhibit expression of one or more HBV genes and methods of treating subjects having an HBV infection and/or HBV-associated disorder, e.g., chronic hepatitis B infection.
Gregory Hinkle, Laura Sepp-Lorenzino, Vasant Jadhav, Martin Maier, Stuart Milstein, Muthiah Manoharan, Kallanthottathil G. Rajeev
Filed: 4 Nov 19
Utility
Patatin-like phospholipase domain containing 3 (PNPLA3) iRNA compositions and methods of use thereof
6 Jul 21
The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the Patatin-Like Phospholipase Domain Containing 3 (PNPLA3) gene, and methods of using such RNAi agents to inhibit expression of a PNPLA3 gene and methods of treating subjects having Nonalcoholic Fatty Liver Disease (NAFLD) and/or a PNPLA3-associated disorder.
Kevin Fitzgerald, Gregory Hinkle
Filed: 5 Feb 19
Utility
Multi-targeted single entity conjugates
6 Jul 21
The present invention relates, in general to, compounds, compositions and methods useful for modulating gene expression of multiple target nucleic acids by a single chemical entity.
Vasant Jadhav, Martin Maier, Ivan Zlatev, Akin Akinc, Kallanthottathil G. Rajeev, Jayaprakash K. Nair, Pachamuthu Kandasamy, Muthiah Manoharan
Filed: 15 Jul 16
Utility
Amyloid precursor protein (APP) RNAi agent compositions and methods of use thereof
15 Jun 21
The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting the APP gene, as well as methods of inhibiting expression of an APP gene and methods of treating subjects having an APP-associated disease or disorder, such as cerebral amyloid angiopathy (CAA) and early onset familial Alzheimer disease (EOFAD or eFAD), using such dsRNAi agents and compositions.
Stuart Milstein, Kirk Brown, Jayaprakash Nair, Martin Maier, Vasant Jadhav, Mark Keating, Adam Castoreno, Patrick Haslett, Mangala Meenakshi Soundarapandian, Kevin Fitzgerald
Filed: 9 Jul 20
Utility
Apolipoprotein C3 (APOC3) iRNA compositions and methods of use thereof
15 Jun 21
The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the apolipoprotein C3 (APOC3) gene, and methods of using such RNAi agents to inhibit expression of APOC3 and methods of treating subjects having an APOC3 associated disorder, e.g., hypertriglyceridemia.
Kevin Fitzgerald, William Querbes, James Butler, Stephanie Williams, Abigail Liebow, Gregory Hinkle, Martin A. Maier, Stuart Milstein, Satyanarayana Kuchimanchi, Muthiah Manoharan
Filed: 21 Dec 20
Utility
Compositions and methods for inhibiting expression of the ALAS1 gene
8 Jun 21
The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ALAS1 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of ALAS1.
Brian Bettencourt, Kevin Fitzgerald, William Querbes, Robert J. Desnick, Makiko Yasuda
Filed: 20 Sep 18
Utility
iRNA agents with biocleavable tethers
25 May 21
The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose that further includes a tether having one or more linking groups, in which at least one of the linking groups is a cleavable linking group.
Muthiah Manoharan, Kallanthottathil G. Rajeev
Filed: 23 Jul 18
Utility
Angiotensinogen (AGT) iRNA compositions and methods of use thereof
25 May 21
The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the AGT gene.
Donald Foster, Gregory Hinkle, Mark K. Schlegel
Filed: 10 Nov 20
Utility
Serpina1 iRNA compositions and methods of use thereof
4 May 21
The invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the Serpina1 gene, and methods of using such RNAi agents to inhibit expression of Serpina1 and methods of treating subjects having a Serpina1 associated disease, such as a liver disorder.
Mark K. Schlegel, Maja Janas, Vasant R. Jadhav, Donald Foster, Muthiah Manoharan, Kallanthottathil G. Rajeev, Alexander V. Kel'in, Klaus Charisse, Jayaprakash K. Nair, Martin A. Maier, Shigeo Matsuda, Muthusamy Jayaraman, Alfica Sehgal, Christopher Brown, Kevin Fitzgerald, Stuart Milstein
Filed: 23 May 19
Utility
TMPRSS6 iRNA compositions and methods of use thereof
27 Apr 21
The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the TMPRSS6 gene, and methods of using such RNAi agents to inhibit expression of TMPRSS6 and methods of treating subjects having a TMPRSS6 associated disorder, e.g., an iron overload associated disorder, such as β-thalassemia or hemochromatosis.
James Butler, Brian Bettencourt, Kallanthottathil G. Rajeev, Martin A. Maier, Klaus Charisse
Filed: 18 Aug 20
Utility
Polynucleotide agents targeting aminolevulinic acid synthase-1 (ALAS1) and uses thereof
6 Apr 21
The invention relates to polynucleotide agents, e.g., antisense polunucleotide agents, targeting the ALAS1 gene, and methods of using such agents to alter (e.g., inhibit) expression of ALAS1 and to treat ALAS1 associated diseases, e.g., porphyria.
Gregory Hinkle
Filed: 30 Mar 17
Utility
GNAQ targeted dsRNA compositions and methods for inhibiting expression
23 Mar 21
The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a G-alpha q subunit (GNAQ) of a heterotrimeric G gene, and methods of using the dsRNA to inhibit expression of GNAQ.
Jared Gollob, Gregory Hinkle, Ivanka Toudjarska, David Bumcrot
Filed: 3 Apr 18
Utility
Polynucleotide agents targeting hydroxyacid oxidase (glycolate oxidase, HAO1) and methods of use thereof
16 Mar 21
The invention relates to polynucleotide agents targeting an hydroxyacid oxidase (HAO1) gene, and methods of using such polynucleotide agents to inhibit expression of HAO1 and to treat subjects having an HAO1-associated disease, e.g., hyperoxaluria.
Gregory Hinkle
Filed: 14 Dec 17
Utility
Myostatin iRNA compositions and methods of use thereof
2 Mar 21
The present invention provides methods comprising the in vivo delivery of small nucleic acid molecules capable of mediating RNA interference and reducing the expression of myostatin, wherein the small nucleic acid molecules are introduced to a subject by systemic administration.
Vasant R. Jadhav, Rubina Parmar, Laura Sepp-Lorenzino, Muthiah Manoharan
Filed: 20 Sep 17
Utility
Factor XII (hageman factor) (F12), kallikrein B, plasma (fletcher factor) 1 (KLKB1), and kininogen 1 (KNG1) iRNA compositions and methods of use thereof
2 Mar 21
The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the Kallikrein B, Plasma (Fletcher Factor) 1 (KLKB1) gene, the Factor XII (Hageman Factor (F12) gene, or the Kininogen 1 (KNG1) gene, and methods of using such RNAi agents to inhibit expression of a KLKB1 gene, an F12 gene, and/or a KNG1 gene, and methods of treating subjects having an hereditary angioedema (HAE) and/or a contact activation pathway-associated disorder.
Akin Akinc, Gregory Hinkle, Martin A. Maier, James Butler, Jingxuan Liu
Filed: 24 May 19
Utility
Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
2 Mar 21
The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ANGPTL3 gene, as well as methods of inhibiting expression of ANGPTL3 and methods of treating subjects having a disorder of lipid metabolism, such as hyperlipidemia or hypertriglyceridemia, using such dsRNA compositions.
Brian Bettencourt, William Querbes, Kevin Fitzgerald, Maria Frank-Kamenetsky, Stuart Milstein, Svetlana Shulga Morskaya
Filed: 14 May 19