354 patents
Page 4 of 18
Utility
Modified Double-stranded Rna Agents
13 Apr 23
One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene.
Martin MAIER, Don Foster, Stuart Milstein, Satya Kuchimanchi, Vasant Jadhav, Kallanthottathil Rajeev, Muthiah Manoharan, Rubina Parmar
Filed: 9 Sep 22
Utility
Methods and Compositions for Treating a Proprotein Convertase Subtilisin Kexin (PCSK9) Gene-associated Disorder
6 Apr 23
The invention relates to methods of inhibiting the expression of a PCSK9 gene in a subject, as well as therapeutic and prophylactic methods for treating subjects having a lipid disorder, such as a hyperlipidemia using RNAi agents, e.g., double-stranded RNAi agents, targeting the PCSK9 gene.
Kevin Fitzgerald
Filed: 31 Mar 22
Utility
STEROL REGULATORY ELEMENT BINDING PROTEIN (SREBP) CHAPERONE (SCAP) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
30 Mar 23
The invention relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting the SCAP gene, as well as methods of inhibiting expression of a SCAP gene and methods of treating subjects having a SCAP-associated disorder, such as nonalcoholic fatty liver disease (NAFLD) or nonalcoholic steatohepatitis (NASH), using such dsRNAi agents and compositions.
Kevin Fitzgerald, Huilei Xu, Gregory Hinkle
Filed: 8 Jul 22
Utility
Kisspeptin 1 (KISS1) Irna Compositions and Methods of Use Thereof
30 Mar 23
The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the KISS1 gene.
Mangala Meenakshi Soundarapandian, Gregory Hinkle
Filed: 10 Jun 22
Utility
Biodegradable lipids for the delivery of active agents
28 Mar 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
Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
28 Mar 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: 8 Mar 22
Utility
Chirally-enriched double-stranded RNA agents
7 Mar 23
The present invention relates to a chirally-modified dsRNA agent capable of inhibiting the expression of a target gene.
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: 21 Dec 18
Utility
APOLIPOPROTEIN C3 (APOC3) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
2 Mar 23
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: 11 May 22
Utility
Biodegradable lipids for the delivery of active agents
28 Feb 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
ATAXIN3 (ATXN3) Rnai Agent Compositions and Methods of Use Thereof
23 Feb 23
The disclosure relates to double-stranded ribonucleic acid (dsRNA) agents and compositions targeting the ATXN3 gene, as well as methods of inhibiting expression of an APP gene and methods of treating subjects having an ATXN3-associated disease or disorder, such as SCA3, using such dsRNA agents and compositions.
ADAM CASTORENO, ELANE FISHILEVICH, JASON A. GILBERT, CHARALAMBOS KAITTANIS, JAMES D. MCININCH, STUART MILSTEIN, MARK K. SCHLEGEL
Filed: 20 Nov 20
Utility
Compositions and Methods for Inhibiting Expression of Mutant Egfr Gene
23 Feb 23
The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a mutant Epidermal Growth Factor Receptor (EGFR), and methods of using the dsRNA to inhibit expression of mutant EGFR.
DINAH SAH, PAMELA TAN, WEBSTER CAVENEE, FRANK FURNARI, MARIA DEL MAR INDA PEREZ, RUDY BONAVIA
Filed: 29 Apr 22
Utility
Modified Double Stranded Oligonucleotide
16 Feb 23
The disclosure is directed to dual variable domain immunoglobulin double-stranded RNA conjugates that are advantageous for inhibition of target gene expression, as well as compositions suitable for therapeutic use.
Alexander V. KEL'IN, Alex R. NANNA, Christopher RADER, Christopher THEILE, Justin PIERSON, Kevin FITZGERALD, Zhi Xiang VOO
Filed: 18 Jun 20
Utility
Compositions and Methods for Silencing DNAJB1-PRKACA Fusion Gene Expression
9 Feb 23
The disclosure relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the DNAJB 1-PRKAC A fusion gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of DNAJB 1-PRKAC A fusion.
LEILA NOETZLI, HO-CHOU TU, PATRICK HASLETT, JAMES MCININCH
Filed: 30 Oct 20
Utility
SMALL RIBOSOMAL PROTEIN SUBUNIT 25 (RPS25) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
2 Feb 23
The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting an RPS25 gene, as well as methods of inhibiting expression of an RPS25 gene and methods of treating subjects having an RPS25-associated disease or disorder, such as a nucleotide repeat expansion disorder, e.g., c9orf72 amyotrophic lateral sclerosis (ALS)/frontotemporal demential (FTD) and Huntington-Like Syndrome Due To C9orf72 Expansions, using such dsRNAi agents and compositions.
Bret Lee Bostwick, Mangala Meenakshi Soundarapandian, James D. McIninch, Gregory Hinkle
Filed: 10 Feb 22
Utility
Tunable Reversir TM Compounds
26 Jan 23
The present invention relates, in general to agents that modulate the pharmacological activity of siRNAs.
Ivan ZLATEV, Adam CASTORENO, Martin MAIER, Vasant JADHAV, Jae KIM, Pushkal GARG
Filed: 25 Jan 22
Utility
Synthesis of 3 -Rna Oligonucleotides
26 Jan 23
Provided is a method of treating cancer cells localized in the lung by administering to such patients a therapeutically effective amount of a liposomal annamycin formulation (L-Ann).
Jayaprakash K. NAIR, Juan C. SALINAS, John Frederick BRIONES, Mark K. SCHLEGEL, Shigeo MATSUDA, Alexander V. KEL'IN, Ligang ZHANG, Martin A. MAIER
Filed: 23 Nov 20
Utility
TRANSTHYRETIN (TTR) iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING TTR-ASSOCIATED DISEASES
26 Jan 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 diseases.
Tracy Zimmermann, Amy Chan, Vasant R. Jadhav, Martin A. Maier, Kallanthottathil G. Rajeev
Filed: 26 Jan 22
Utility
Neurofilament Light Chain (NFL) As a Biomarker for Transthyretin Amyloidosis Polyneuropathy
26 Jan 23
The disclosure provides biomarkers for diagnosis and monitoring of transthyretin (TTR) amyloidosis.
Simina Ticau, Paul Nioi, Gautham Vivek Sridharan, Shira Tsour
Filed: 16 Feb 22
Utility
Compositions and Methods for Inhibiting Expression of the ALAS1 Gene
26 Jan 23
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: 4 May 21
Utility
Extrahepatic Delivery
19 Jan 23
One aspect of the present invention relates to a compound 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 monomers, containing one or more lipophilic moieties, conjugated to one or more positions on at least one strand, optionally via a linker or carrier.
Jayaprakash K. NAIR, Martin A. MAIER, Juan C. SALINAS, Shigeo MATSUDA, Alexander V. KEL'IN, Scott P. LENTINI, Guo HE, Michelle H. JUNG, Justin M. PIERSON, Muthiah MANOHARAN, Dale C. GUENTHER, Ivan ZLATEV, Christopher S. THEILE, Vasant R. JADHAV, Stuart MILSTEIN, Maja JANAS, Dhrubajyoti DATTA
Filed: 6 Nov 20