236 patents
Utility
Complement Factor B (CFB) Irna Compositions and Methods of Use Thereof
18 Jan 24
The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the complement factor B (CFB) gene.
James D. McIninch, Adam Castoreno, Mark K. Schlegel, Elane Fishilevich, Kristina Yucius, Charalambos Kaittanis
Filed: 27 Oct 22
Utility
Extrahepatic Delivery
11 Jan 24
One aspect of the present invention relates to a double stranded iRNA agent comprising an antisense strand which is complementary to a target gene; a sense strand which is complementary to said antisense strand; and one or more lipophilic moieties conjugated to one or more internal positions on at least one strand, optionally via a linker or carrier.
Jayaprakash K. NAIR, Martin MAIER, Vasant JADHAV, Stuart MILSTEIN, Kirk BROWN, Rubina G. PARMAR, Kallanthottathil G. RAJEEV, Muthiah MANOHARAN, Alexander V. KEL'IN, Muthusamy JAYARAMAN, Klaus CHARISSE, Adam CASTORENO, Christopher THEILE, Kevin FITZGERALD
Filed: 3 May 23
Utility
Snca Irna Compositions and Methods of Use Thereof for Treating or Preventing Snca-associated Neurodegenerative Diseases
11 Jan 24
The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a SNCA gene, as well as methods of inhibiting expression of a SNCA gene and methods of treating subjects having a SNCA-associated neurodegenerative disease or disorder, e.g., Parkinson's Disease (PD), multiple system atrophy, Lewy body dementia (LBD), among other synucleinopathies, using such dsRNAi agents and compositions.
Mangala Meenakshi Soundarapandian, Lan Thi Hoang Dang, James D. Mclninch, Mark K. Schlegel, Adam Castoreno, Charalambos Kaittanis
Filed: 29 Sep 21
Utility
Mucin 5B (MUC5B) Irna Compositions and Methods of Use Thereof
28 Dec 23
The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the mucin 5B (MUC5B) gene.
Leila Noetzli, Margaret Parker, James D. McIninch, Mark K. Schlegel, Adam Castoreno, Jeffrey Zuber
Filed: 19 Apr 23
Utility
KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
28 Dec 23
The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the ketohexokinase (KHK) gene.
Frederic Tremblay, James D. McIninch
Filed: 1 Sep 22
Utility
Methods and Compositions for Inhibition of HAO1 (Hydroxyacid Oxidase 1 (Glycolate Oxidase)) Gene Expression
21 Dec 23
The invention relates methods of using RNAi agents to inhibit expression of HAO1 and methods of treating subjects having primary hyperoxaluria, e.g., PH1.
Tracy L. McGregor, John Michael Gansner, Ishir Bhan
Filed: 26 May 23
Utility
G Protein-coupled Receptor 75 (GPR75) Irna Compositions and Methods of Use Thereof
7 Dec 23
The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the G-protein coupled receptor 75 (GPR75) gene.
James D. McIninch, Bret Lee Bostwick, Adam Castoreno
Filed: 3 Apr 23
Utility
COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
7 Dec 23
The invention relates to iRNA, e.g., double stranded ribonucleic acid (dsRNA), compositions targeting the complement component C5 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of C5 and to treat subjects having a complement component C5-associated disease, e.g., paroxysmal nocturnal hemoglobinuria.
Anna Borodovsky
Filed: 8 Mar 23
Utility
HUNTINGTIN (HTT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
7 Dec 23
The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a Huntingtin (HTT) gene, as well as methods of inhibiting expression of an HTT gene and methods of treating subjects having an HTT-associated disease or disorder, e.g., Huntington's disease, using such dsRNAi agents and compositions.
Mangala Meenakshi Soundarapandian, James D. McIninch, Mark K. Schlegel, Adam Castoreno
Filed: 1 Jun 23
Utility
Methods and Compositions for Treating Primary Hyperoxaluria
7 Dec 23
The present invention provides methods and compositions for treating a pediatric subject having primary hyperoxaluria and methods for preventing at least one symptom in a pediatric subject having primary hyperoxaluria.
Gabriel Robbie, Varun Goel
Filed: 19 Apr 23
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