354 patents
Page 3 of 18
Utility
Sirna Compositions and Methods for Silencing Gpam (GLYCEROL-3-PHOSPHATE Acyltransferase 1, Mitochondrial) Expression
29 Jun 23
The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the GPAM gene, as well as methods of inhibiting expression of GPAM, and methods of treating subjects that would benefit from reduction in expression of GPAM, such as subjects having a GPAM-associated disease, disorder, or condition, using such dsRNA compositions.
JAMES D. MCININCH, LUCAS BONDURANT, AIMEE DEATON
Filed: 9 Jun 21
Utility
Programmed cell death 1 ligand 1 (PD-L1) iRNA compositions and methods of use thereof
27 Jun 23
The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the programmed cell death 1 ligand 1 (PD-L1) gene, and methods of using such RNAi agents to inhibit expression of a PD-L1 gene and methods of treating subjects having a PD-L1-associated disorder.
Gregory Hinkle
Filed: 6 Nov 20
Utility
Compositions and Methods for Silencing SCN9A Expression
25 May 23
The disclosure relates to double-stranded ribonucleic acid (dsRNA) compositions targeting SCN9A, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of SCN9A.
WILLIAM CANTLEY, JAMES D. MCININCH, ADAM CASTORENO, CHARALAMBOS KAITTANIS, MARK K. SCHLEGEL
Filed: 6 Apr 21
Utility
Superoxide dismutase 1 (SOD1) iRNA compositions and methods of use thereof for treating or preventing superoxide dismutase 1-(SOD1-) associated neurodegenerative diseases
16 May 23
The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a SOD1 gene, as well as methods of inhibiting expression of a SOD1 gene and methods of treating subjects having a SOD1-associated neurodegenerative disease or disorder, e.g., Amyotrophic Lateral Sclerosis (ALS), Alzheimer's disease (AD), Parkinson's disease (PD), and Down's syndrome (DS), using such dsRNAi agents and compositions.
Adam Castoreno, Jason Gilbert, Charalambos Kaittanis, James D. McIninch, Stuart Milstein, Mark K. Schlegel
Filed: 29 Jun 22
Utility
COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS FOR USE IN THE TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
4 May 23
The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the complement component C5 gene for methods of using such iRNA to inhibit expression of C5 and to treat subjects having ALS.
Anna Borodovsky, Bret Lee Bostwick
Filed: 26 Jul 22
Utility
Compositions and Methods for Silencing Vegf-a Expression
4 May 23
The disclosure relates to double-stranded ribonucleic acid (dsRNA) compositions targeting VEGF-A, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of VEGF-A.
JAMES D. MCININCH, MARK KEATING, MARK K. SCHLEGEL, ADAM CASTORENO, VASANT R. JADHAV, CHARALAMBOS KAITTANIS, ELENA CASTELLANOS-RIZALDOS, BHAUMIK A. PANDYA
Filed: 9 Feb 21
Utility
ALPHA-2A ADRENERGIC RECEPTOR (ADRA2A) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
4 May 23
The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting an alpha-2A adrenergic receptor (ADRA2A) gene, as well as methods of inhibiting expression of an ADRA2A gene and methods of treating subjects having an ADRA2A-associated disease or disorder, e.g., a primary tauopathy or Alzheimer's disease, using such dsRNAi agents and compositions.
BRET L. BOSTWICK, JAMES D. MCININCH, ADAM CASTORENO
Filed: 1 Apr 21
Utility
ANGIOTENSIN-CONVERTING ENZYME 2 (ACE2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
4 May 23
The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the angiotensin converting enzyme 2 (ACE2) gene.
Akin Akinc, James D. McIninch, Yesseinia Anglero-Rodriguez, Mark K. Schlegel
Filed: 30 Sep 22
Utility
Angiopoietin-like 3 (ANGPTL3) Irna Compositions and Methods of Use Thereof
4 May 23
The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the Angiopoietin-like 3 (ANGPTL3) gene.
Lucas D. BonDurant, Mark K. Schlegel, Jeffrey Zuber, Lauren Blair Woods, Tyler Chickering
Filed: 1 Sep 22
Utility
Mannan Binding Lectin Serine Peptidase 2 (MASP2) Irna Compositions and Methods of Use Thereof
27 Apr 23
The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the mannan binding lectin serine peptidase 2 gene (MASP2).
JAMES D. MCININCH, ANNA BORODOVSKY
Filed: 18 Feb 21
Utility
HEPATITIS B VIRUS (HBV) dsRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
27 Apr 23
The present disclosure relates to double stranded RNA agents targeting the hepatitis B virus (HBV) genome, and methods of using such agents to inhibit expression of one or more HBV genes and methods of treating subjects having an HBV infection or HBV-associated disorder, e.g., chronic hepatitis B infection.
Vasant R. JADHAV, Martin A. MAIER, Stuart MILSTEIN, Mark K. SCHLEGEL
Filed: 28 Sep 22
Utility
LEUCINE-RICH REPEAT KINASE 2 (LRRK2) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
27 Apr 23
The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a leucine-rich repeat kinase 2 (LRRK2) gene, as well as methods of inhibiting expression of a LRRK2 gene and methods of treating subjects having a LRRK2-associated disease or disorder, e.g., Parkinson's disease, using such dsRNAi agents and compositions.
JAMES D. MCININCH, JASON A. GILBERT, ADAM CASTORENO, LAN THI HOANG DANG, SARAH LEBLANC, HAIYAN PENG, CHARALAMBOS KAITTANIS, MANGALA MEENAKSHI SOUNDARAPANDIAN
Filed: 22 Jan 21
Utility
Fc FRAGMENT OF IgG RECEPTOR AND TRANSPORTER (FCGRT) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
27 Apr 23
The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting a Fc fragment of IgG receptor and transporter (FCGRT) gene encoding neonatal Fc receptor (FcRn).
AIMEE DEATON, MARGARET PARKER, LEILA NOETZLI, JAMES D. MCININCH
Filed: 26 Mar 21
Utility
Biodegradable lipids for the delivery of active agents
25 Apr 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: 14 Feb 22
Utility
Biodegradable lipids for the delivery of active agents
25 Apr 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: 14 Feb 22
Utility
Compositions and Methods for Inhibition of HAO1 (Hydroxyacid Oxidase 1 (Glycolate Oxidase)) Gene Expression
20 Apr 23
The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the HAO1 gene, and methods of using such RNAi agents to inhibit expression of HAO1 and methods of treating subjects having, e.g., PH1.
William Querbes, Kevin Fitzgerald, Brian Bettencourt, Abigail Liebow, David V. Erbe
Filed: 15 Aug 22
Utility
Chirally-enriched Double-stranded Rna Agents
20 Apr 23
Muthiah MANOHARAN, Nate TANEJA, Hartmut Ingo JAHNS, Shigeo MATSUDA, Klaus CHARISSE, Guo HE, Jayaprakash K. NAIR, Christopher BROWN, Mark K. SCHLEGEL, Vasant JADHAV, Martin MAIER
Filed: 8 Nov 22
Utility
ANGIOTENSINOGEN (AGT) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
20 Apr 23
The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the angiotensinogen (AGT) gene, and methods of using such RNAi agents to inhibit expression of AGT and methods of treating subjects having an AGT-associated disorder, e.g., hypertension.
Donald Foster, Brian Bettencourt, Klaus Charisse, Gregory Hinkle, Satyanarayana Kuchimanchi, Martin A. Maier, Stuart Milstein
Filed: 1 Jul 22
Utility
HUMAN CHROMOSOME 9 OPEN READING FRAME 72 (C9ORF72) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
13 Apr 23
The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a human chromosome 9 open reading frame 72 (C9orf72) gene, as well as methods of inhibiting expression of a C9orf72 gene and methods of treating subjects having a C9orf72-associated disease or disorder, e.g., C9orf72 amyotrophic lateral sclerosis/frontotemporal dementia or Huntington-Like Syndrome Due To C9orf72 Expansions, using such dsRNAi agents and compositions.
Elane Fishilevich, Stuart Milstein, Kirk Brown, Tracy Zimmermann, James D. McIninch, David Frendewey, Eric Chiao, Aarti Sharma-Kanning, Anthony Gagliardi, Gustavo Droguett, Brittany Dubose, Brian Zambrowicz
Filed: 9 Jun 22
Utility
TRANSTHYRETIN (TTR) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
13 Apr 23
The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the transthyretin (TTR) gene.
Mark K. Schlegel, Adam Castoreno, James D. McIninch
Filed: 28 Sep 22