354 patents
Page 2 of 18
Utility
ANAPLASTIC LYMPHOMA KINASE (ALK) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
26 Oct 23
The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting an anaplastic lymphoma kinase (ALK) gene, as well as methods of inhibiting expression of an ALK gene and methods of treating subjects having an ALK-associated disease or disorder, e.g., type 2 diabetes, obesity, or an obesity-associated disorder, using such dsRNAi agents and compositions.
BRET L. BOSTWICK, JAMES D. MCININCH
Filed: 15 Jun 21
Utility
Sirna Therapy for Transthyretin (TTR) Related Ocular Amyloidosis
26 Oct 23
The invention relates to a method of treating ocular amyloidosis by reducing TTR expression in a subject by administering a double-stranded ribonucleic acid (dsRNA) that targets a TTR gene to the retinal pigment epithelium of the subject.
Yukio Ando, Hirofumi Jono, Rene Alvarez, Dinah Wen-yee Sah
Filed: 1 Nov 22
Utility
Irna Compositions and Methods for Silencing Growth Factor Receptor Bound Protein 10 (GRB10) or Growth Factor Receptor Bound Protein 14 (GRB14) In the Liver
19 Oct 23
The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the GRB 10 or GRB 14 gene, as well as methods of inhibiting expression of GRB 10 or GRB 14, and methods of treating subjects that would benefit from reduction in expression of GRB 10 or GRB 14, such as subjects having a GRB 10- or GRB 14-associated disease, disorder, or condition, such as diabetes, using such dsRNA compositions.
JAMES D. MCININCH, AIMEE DEATON, LUCAS BONDURANT, MARK K. SCHLEGEL, ADAM CASTORENO
Filed: 9 Sep 21
Utility
TUMOR NECROSIS FACTOR RECEPTOR ASSOCIATED FACTOR 6 (TRAF6) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
12 Oct 23
The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the TRAF6 gene, as well as methods of inhibiting expression of TRAF6, and methods of treating subjects that would benefit from reduction in expression of TRAF6, such as subjects having a TRAF6-associated disease, disorder, or condition, using such dsRNA compositions.
ARLIN ROGERS, MELISSA MOBLEY, JAMES D. MCININCH
Filed: 8 Jun 21
Utility
KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
12 Oct 23
The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the ketohexokinase (KHK) gene.
Leila Noetzli, James D. McIninch, Frederic Tremblay, Mark K. Schlegel, Adam Castoreno
Filed: 2 Dec 22
Utility
Polynucleotide Agents Targeting Angiotensinogen (Agt) and Methods of Use Thereof
5 Oct 23
The invention relates to polynucleotide agents, e.g., antisense polynucleotide agents, targeting an angiotensinogen (AGT) gene, and methods of using such polynucleotide agents to inhibit expression of AGT and to treat subjects having an AGT-associated disease, e.g., hypertension.
Gregory Hinkle
Filed: 30 Aug 22
Utility
TRANSTHYRETIN (TTR) iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING TTR-ASSOCIATED OCULAR DISEASES
28 Sep 23
The present invention provides iRNA agents, e.g., double stranded iRNA agents, that target the transthyretin (TTR) gene and methods of using such iRNA agents for treating or preventing TTR-associated ocular diseases.
Jayaprakash K. Nair, Martin A. Maier, Vasant R. Jadhav, Mark Keating, Kevin Fitzgerald, Stuart Milstein, John R. Petrulis
Filed: 4 May 22
Utility
Compositions and Methods for Silencing Myoc Expression
21 Sep 23
The disclosure relates to double-stranded ribonucleic acid (dsRNA) compositions targeting MYOC, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of MYOC.
MARK KEATING, JAMES D. MCININCH
Filed: 6 Apr 21
Utility
RNAi COMPOSITIONS AND METHODS OF USE THEREOF FOR DELIVERY BY INHALATION
14 Sep 23
The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents for administration by inhalation preferably targeting genes expressed in the respiratory system, as well as methods of inhibiting expression of a target gene after administration by inhalation, preferably a gene expressed in the respiratory system.
Christopher Brown, Donald Foster, Arlin Rogers, Vasant R. Jadhav, Akin Akinc, Amy R. Simon
Filed: 8 Dec 22
Utility
G Protein-coupled Receptor 146 (GPR146) Irna Compositions and Methods of Use Thereof
14 Sep 23
The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the G protein-coupled receptor 146 (GPR146).
KEVIN FITZGERALD, FREDERIC TREMBLAY, JAMES D. MCININCH
Filed: 26 Feb 21
Utility
COMPLEMENT COMPONENT C3 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING COMPLEMENT COMPONENT C3-ASSOCIATED DISEASES
31 Aug 23
The present invention provides iRNA agents, e.g., double stranded iRNA agents, that target the complement component C3 gene and methods of using such iRNA agents for treating or preventing C3-associated ocular diseases or C3-associated neurodegenerative diseases.
Mark Keating, James D. McIninch
Filed: 1 Sep 22
Utility
Modified Rna Agents with Reduced Off-target Effect
17 Aug 23
One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene.
Mark K. SCHLEGEL, Maja JANAS, Vasant R. JADHAV, Muthiah MANOHARAN, Kallanthottathil G. RAJEEV, Muthusamy JAYARAMAN, Alexander V. KEL'IN, Shigeo MATSUDA, Klaus CHARISSE, Jayaprakash K. NAIR, Martin A. MAIER, Alfica SEHGAL, Christopher BROWN, Christopher THEILE, Stuart MILSTEIN
Filed: 30 Sep 22
Utility
Biodegradable Lipids for the Delivery of Active Agents
17 Aug 23
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: 20 Apr 23
Utility
Compositions and Methods for Inhibiting Expression of Transthyretin
17 Aug 23
The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a transthyretin (TTR) gene, and methods of using the dsRNA to inhibit expression of TTR.
Dinah Wen-yee Sah, Gregory Hinkle, Rene Alvarez, Stuart Milstein, Qingmin Chen
Filed: 15 Apr 22
Utility
Circular siRNAs
17 Aug 23
One aspect of the present invention relates to a small circular interfering RNA (sciRNA) comprising a sense strand and an antisense strand, each of said sense and antisense strands comprising at least one nucleic acid modification, optionally wherein the sense strand has a circular or substantially circular structure.
Hartmut JAHNS, Ivan ZLATEV, Muthiah MANOHARAN, Christopher S. THEILE
Filed: 9 Jul 21
Utility
Complement Factor B (CFB) Irna Compositions and Methods of Use Thereof
17 Aug 23
The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the complement factor B (CFB) gene.
James D. McIninch, Mark K. Schlegel, Adam Castoreno, Elane Fishilevich, Kristina Yucius
Filed: 22 Mar 23
Utility
Serpina1 iRNA compositions and methods of use thereof
15 Aug 23
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: 8 Mar 21
Utility
TMPRSS6 iRNA compositions and methods of use thereof
15 Aug 23
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, Martin A. Maier, Kevin Fitzgerald, Shannon Fishman, Donald Foster, Vasant R. Jadhav, Stuart Milstein
Filed: 5 Oct 20
Utility
TRANSMEMBRANE PROTEASE, SERINE 6 (TMPRSS6) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
13 Jul 23
The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the Transmembrane protease, serine 6 (TMPRSS6) gene.
Aimee M. Deaton, John Michael Gansner, James D. McIninch, Mark K. Schlegel, Benjamin P. Garfinkel
Filed: 6 Jan 23
Utility
MICROTUBULE ASSOCIATED PROTEIN TAU (MAPT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
29 Jun 23
The disclosure relates to double stranded ribonucleic acid interference (dsRNAi) agents and compositions targeting a microtubule-associated protein tau (MAPT) gene, as well as methods of inhibiting expression of a MAPT gene and methods of treating subjects having a MAPT-associated disease or disorder, e.g., Alzheimer's disease, frontotemporal dementia, progressive supranuclear palsy, or other tauopathies, using such dsRNAi agents and compositions.
MANGALA MEENAKSHI SOUNDARAPANDIAN, JAMES D. MCININCH, ELANE FISHILEVICH, ADAM CASTORENO, CHARALAMBOS KAITTANIS, MARK K. SCHLEGEL, JONATHAN EDWARD FARLEY, JEFFREY ZUBER
Filed: 30 Mar 21