354 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
Complement component C5 iRNA compositions and methods of use thereof
16 Jan 24
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.
Kevin Fitzgerald, James Butler, Brian Bettencourt, Anna Borodovsky, Satyanarayana Kuchimanchi, Klaus Charisse, Muthiah Manoharan, Martin A. Maier, Kallanthottathil G. Rajeev, Donald Foster
Filed: 12 Jul 21
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
Complement component C3 iRNA compositions and methods of use thereof
9 Jan 24
The invention relates to iRNA, e.g., double stranded ribonucleic acid (dsRNA), compositions targeting the complement factor C3 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of a C3 gene and to treat subjects having a complement component C3-associated disease, e.g., paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), atypical hemolytic uremic syndrome (aHUS), neuromyelitis optica (NMO), multifocal motor neuropathy (MMN), myasthenia gravis (MG), and C3 glomerulonephritis.
Gregory Hinkle, Anna Borodovsky
Filed: 1 Nov 18
Utility
Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
9 Jan 24
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: 22 Nov 21
Utility
Transmembrane protease, serine 6 (TMPRSS6) iRNA compositions and methods of use thereof
9 Jan 24
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
Glucokinase (GCK) iRNA compositions and methods of use thereof
2 Jan 24
The invention relates to double stranded ribonucleic acid (dsRNA) compositions targeting a glucokinase (GCK) gene, as well as methods of inhibiting expression of a glucokinase (GCK) gene, and methods of treating subjects having a glycogen storage disease (GSD), e.g., type Ia GSD.
Kevin Fitzgerald, David Erbe, Gregory Hinkle
Filed: 14 Oct 20
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
Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
12 Dec 23
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: 22 Nov 21
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
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
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
Angiotensinogen (AGT) iRNA compositions and methods of use thereof
5 Dec 23
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: 7 Apr 21
Utility
Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
5 Dec 23
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: 22 Nov 21
Utility
Dosages and methods for delivering lipid formulated nucleic acid molecules
28 Nov 23
Methods, kits and devices for dosing a subject to reduce a hypersensitivy response to a lipid-formulated nucleic acid (e.g., RNA) molecule are disclosed.
Pia Kasperkovitz, Jared Gollob
Filed: 8 Apr 21
Utility
Compositions and methods for treating transthyretin (TTR) mediated amyloidosis
7 Nov 23
Disclosed herein are methods for treating hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in a human patient in need thereof by administering an effective amount of a transthyretin (TTR)-inhibiting composition.
Jared Gollob
Filed: 19 Sep 18