Patent classifications
C12N2310/322
OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF
- Prashant Monian ,
- Chikdu Shakti Shivalila ,
- Subramanian Marappan ,
- Chandra Vargeese ,
- Pachamuthu Kandasamy ,
- Genliang Lu ,
- Hui Yu ,
- David Charles Donnell Butler ,
- Luciano Henrique Apponi ,
- Mamoru Shimizu ,
- Stephany Michelle Standley ,
- David John Boulay ,
- Andrew Guzior Hoss ,
- Jigar Desai ,
- Jack David Godfrey ,
- Hailin Yang ,
- Naoki Iwamoto
Among other things, the present disclosure provides oligonucleotides and compositions thereof. In some embodiments, provided oligonucleotides and compositions are useful for adenosine modification. In some embodiments, the present disclosure provides methods for treating various conditions, disorders or diseases that can benefit from adenosine modification.
Optimized anti-FLT1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
This disclosure relates to novel targets for angiogenic disorders. Novel oligonucleotides are also provided. Methods of using the novel oligonucleotides for the treatment of angiogenic disorders (e.g., preeclampsia) are also provided.
Optimized anti-FLT1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
This disclosure relates to novel targets for angiogenic disorders. Novel oligonucleotides are also provided. Methods of using the novel oligonucleotides for the treatment of angiogenic disorders (e.g., preeclampsia) are also provided.
TRANSMEMBRANE PROTEASE, SERINE 6 (TMPRSS6) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the Transmembrane protease, serine 6 (TMPRSS6) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a TMPRSS6 gene and to methods of preventing and treating a TMPRSS6-associated disorder, e.g., a disorder associated with iron overload and/or a disorder of ineffective erythropoiesis, e.g., hereditary hemochromatosis, β-thalassemia (e.g., β-thalassemia major and β-thalassemia intermiedia), polycythemia vera, myelodysplastic syndrome, congenital dyserythropoietic anemias, pyruvate kinase deficiency, erythropoietic porphyria, Parkinson's Disease, Alzheimer's Disease or Friedreich's Ataxia.
COMPOSITIONS AND METHODS FOR INHIBITING NUCLEAR RECEPTOR SUBFAMILY 1 GROUP H MEMBER 3 (NR1H3) EXPRESSION
Oligonucleotides are provided herein that inhibit NR1H3 expression. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with NR1H3 expression.
COMPOSITIONS AND METHODS FOR INHIBITING NUCLEAR RECEPTOR SUBFAMILY 1 GROUP H MEMBER 3 (NR1H3) EXPRESSION
Oligonucleotides are provided herein that inhibit NR1H3 expression. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with NR1H3 expression.
RNAI AGENTS, COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING TRANSTHYRETIN (TTR) ASSOCIATED DISEASES
The present invention provides RNAi agents, e.g., double stranded RNAi agents, that target the transthyretin (TTR) gene and methods of using such RNAi agents for treating or preventing TTR-associated diseases.
RNAI AGENTS, COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING TRANSTHYRETIN (TTR) ASSOCIATED DISEASES
The present invention provides RNAi agents, e.g., double stranded RNAi agents, that target the transthyretin (TTR) gene and methods of using such RNAi agents for treating or preventing TTR-associated diseases.
METHODS FOR CONTROLLING EXTRACORPOREAL MEMBRANE OXYGENATION (ECMO) COAGULATION
The present application provides for a method for controlling coagulation in a subject in need of extracorporeal membrane oxygenation. The method comprises administering an effective amount of an anticoagulant agent that directly inhibits one or more steps in a coagulation pathway to the subject, wherein the effective amount of the anticoagulant agent is capable of controlling coagulation during extra corporeal membrane oxygenation for a coagulation control time of at least 4 hours.
METHODS FOR CONTROLLING EXTRACORPOREAL MEMBRANE OXYGENATION (ECMO) COAGULATION
The present application provides for a method for controlling coagulation in a subject in need of extracorporeal membrane oxygenation. The method comprises administering an effective amount of an anticoagulant agent that directly inhibits one or more steps in a coagulation pathway to the subject, wherein the effective amount of the anticoagulant agent is capable of controlling coagulation during extra corporeal membrane oxygenation for a coagulation control time of at least 4 hours.