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RNA InterferenceRNA, Small InterferingRNA, Double-StrandedGene SilencingCaenorhabditis elegansRNA-Induced Silencing ComplexRibonuclease IIICaenorhabditis elegans ProteinsArgonaute ProteinsGene Knockdown TechniquesRNA, HelminthDrosophila ProteinsDrosophila melanogasterTriboliumGenes, HelminthDrosophilaAnimals, Genetically ModifiedPhenotypeMolecular Sequence DataGenetic TechniquesCell LineLentivirusTrypanosoma brucei bruceiGreen Fluorescent ProteinsHeLa CellsGenome, InsectPlanariansGenetic VectorsInsect ProteinsMicroRNAsMutationRNA, MessengerHeterochromatinRNA, AntisenseBase SequenceCell Line, TumorTransgenesHelminth ProteinsHigh-Throughput Screening AssaysGene Expression Regulation, DevelopmentalGene Expression RegulationRNA-Binding ProteinsGenes, InsectTransfectionDEAD-box RNA HelicasesGene TargetingSignal TransductionRNA, UntranslatedEmbryo, NonmammalianRNA HelicasesPlants, Genetically ModifiedLarvaAmino Acid SequenceGenomicsReverse Transcriptase Polymerase Chain ReactionGenome, HelminthEndoribonucleasesSchizosaccharomycesGene Expression ProfilingDown-RegulationFat BodyRNATranscription FactorsEukaryotic Initiation FactorsSchizosaccharomyces pombe ProteinsGenes, ReporterModels, BiologicalNuclear ProteinsModels, GeneticGenetic TherapyGerm CellsRNA ReplicaseTranscription, GeneticNematodaCells, CulturedRNA Processing, Post-TranscriptionalCentromereCell ProliferationGenomeProtein TransportCell Cycle ProteinsDNA PrimersGene LibraryBlotting, WesternHost-Pathogen InteractionsCytokinesisProtein BindingTenuivirusGene Expression Regulation, PlantApoptosisMicroscopy, FluorescenceMitosisChromosomal Proteins, Non-HistonePlasmidsHEK293 CellsProtein Structure, TertiaryMoltingGene ExpressionPhylogenyRNA, Plant
TherapeuticsPatisiranInterferenceSiRNADiscovered the RNAi mechanismInvestigationalONPATTROMiRNAAntibodiesAntisenseMammalian cellsMechanismTransthyretinPathwayHereditaryAntiviralTargetsDouble-strandedAlnylamPhaseInfectionOncologySilenceMoleculesLipidPipelineVaccineDevelopmentPlatformPotentialClinicalPatientsCompoundsMethodsResearchDiscoverySearchDrugsApproachTreat
Therapeutics14
  • Paris, France and Cambridge, MA - July 10, 2017 - Sanofi Genzyme , the specialty care global business unit of Sanofi , and Alnylam Pharmaceuticals, Inc , the leading RNAi therapeutics company, announced today new positive results from the ongoing phase 2 open-label extension (OLE) study with fitusiran in patients with hemophilia A and B, with or without inhibitors (N=33). (sanofi.com)
  • Several key challenges currently face succesful translation of RNAi into viable therapeutics. (rcsi.com)
  • The approval of Leqvio, a potentially transformational medicine for lowering LDL-C is a historic event for Alnylam and its RNAi therapeutics platform. (theeveningleader.com)
  • More broadly, we believe the approval of Leqvio validates the future potential of RNAi therapeutics in large population diseases, and facilitates Alnylam's advancement toward its bold Alnylam P 5 x25 strategy and goals. (theeveningleader.com)
  • The U.S. approval of Leqvio will bring RNAi therapeutics to even more patients with elevated or inadequately controlled LDL-C. Moreover, this approval highlights the potential for additional Alnylam RNAi therapeutic programs in prevalent disease indications. (theeveningleader.com)
  • CAMBRIDGE, Mass.--( BUSINESS WIRE )-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) has set a date of September 13, 2023 for the meeting of the Cardiovascular and Renal Drugs Advisory Committee to review the supplemental New Drug Application for patisiran, an investigational treatment for the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis. (businesswire.com)
  • The data published in Nature Medicine contributes to existing data demonstrating the potential for developing RNAi therapeutics that target diseases associated with the lung, such as respiratory syncytial virus (RSV) infection, influenza, and asthma. (genengnews.com)
  • Alnylam is the exclusive licensee of the therapeutic and diagnostic application of Max Planck Society´s groundbreaking patents, which are a central component of Alnylam's technology portfolio for the development, manufacture, and marketing of RNAi therapeutics. (mpg.de)
  • RNAimmune is a spin-off entity from Sirnaomics , a biopharmaceutical company engaged in the discovery and development of RNAi therapeutics against cancer and fibrotic diseases. (prnewswire.com)
  • The therapeutic areas of focus include oncology and anti-fibrotic therapeutics. (prnewswire.com)
  • Officials of Kylin Therapeutics Inc. has announced on Thursday (July 5) that it has obtained from Purdue University an exclusive license to a RNA interference (RNAi) delivery technology called "pRNA. (technologynetworks.com)
  • pRNA solves this problem for RNAi and other RNA-based therapeutics. (technologynetworks.com)
  • Kylin's Therapeutics lead product is a pRNA-based therapeutic to treat cancer, and the company's initial plans are to focus on cancer therapy, with follow-on treatments for viral diseases such as AIDS. (technologynetworks.com)
  • pRNA is the missing link necessary for RNAi to be delivered in a targeted, systemic fashion," said Eric Davis, CEO of Kylin Therapeutics, which is a Purdue Research Park company. (technologynetworks.com)
Patisiran7
  • Alnylam launched its first RNAi therapeutic in 2018 with the FDA approval of ONPATTRO ® (patisiran) for treatment of the polyneuropathy caused by hATTR amyloidosis, a progressive and life-threatening, rare, genetic disease. (theeveningleader.com)
  • ONPATTRO (patisiran) is an RNAi therapeutic that is approved in the United States and Canada for the treatment of the polyneuropathy of hATTR amyloidosis in adults. (businesswire.com)
  • The United States Food and Drug Administration (FDA) approved the first-of-its-kind RNA interference (RNAi) therapeutic patisiran (Onpattro) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. (mpg.de)
  • Adams D et al (2018) Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis. (springer.com)
  • The US Food and Drug Administration (FDA) has declined to approve the RNA interference (RNAi) therapeutic agent patisiran (Onpattro, Alnylam Pharmaceuticals) for treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy, the company has announced . (medscape.com)
  • Quality of life outcomes in APOLLO, the phase 3 trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis. (msdmanuals.com)
  • Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis. (msdmanuals.com)
Interference8
  • RNA interference (RNAi) is a powerful, naturally occurring gene-silencing phenomenon. (rcsi.com)
  • Small interfering RNA (siRNA), sometimes known as short interfering RNA or silencing RNA, is a class of double-stranded RNA at first non-coding RNA molecules, typically 20-24 (normally 21) base pairs in length, similar to miRNA, and operating within the RNA interference (RNAi) pathway. (wikipedia.org)
  • RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. (mpg.de)
  • Background/aims: RNA interference (RNAi), a sequence-specific gene silencing technology triggered by small interfering RNA (siRNA), represents promising new avenues for treatment of various liver diseases including hepatitis C virus (HCV) infection. (eur.nl)
  • RNA interference (RNAi) is a sequence-specific RNA degradation mechanism mediated by small interfering RNA (siRNA), which represents a possible therapeutic application for the treatment of viral infections. (scienceopen.com)
  • RNA interference (RNAi) is an ancient antiviral response that processes dsRNA and associates it into a nuclease complex that identifies RNA with sequence homology and specifically cleaves it. (scienceopen.com)
  • It is a double-stranded oligomer ARO-APOC3 RNA interference-based liver targeted therapeutic. (pharmaceutical-technology.com)
  • Isis Pharmaceuticals, Inc. has announced that it has been awarded a multi-year Phase 2 Small Business Innovation Research (SBIR) grant by the National Institutes of Health (NIH) for up to $1.5 million to design oligonucleotide drugs that can exploit the RNA interference (RNAi) antisense mechanism for disease treatment. (news-medical.net)
SiRNA10
  • Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, function upstream of today's medicines by potently silencing messenger RNA (mRNA) - the genetic precursors - that encode for disease-causing proteins, thus preventing them from being made. (mpg.de)
  • In plants and invertebrates, RNAi provides an important mechanism of cellular defence against viral pathogens and is dependent on the spread of siRNA to neighbouring cells. (eur.nl)
  • Methods: Transmission of RNAi was investigated in culture by assessing silencing of HCV replication and expression of viral entry receptor CD81 using a human hepatic cell line and primary B lymphocytes transduced with siRNA-expressing vectors. (eur.nl)
  • Evidence of RNAi transmission in vivo was observed in NOD/SCID mice engrafted with human hepatoma cells producing CD81 siRNA, causing suppression of CD81 expression in mouse hepatocytes. (eur.nl)
  • Transmission of siRNA potentially extends the therapeutic reach of RNAi-based therapies against HCV as well as other liver diseases. (eur.nl)
  • Known for almost 30 years as premier providers of synthetic oligonucleotides, Dharmacon Reagents were the first to offer algorithm designed synthetic siRNA for RNAi, and the first to offer guide RNA for CRISPR-Cas9 gene editing applications. (horizondiscovery.com)
  • The therapeutic candidate is a siRNA which acts by targeting apolipoprotein C III. (pharmaceutical-technology.com)
  • Evaluation of an RNAi therapeutic approach against RA using theranostic siRNA nanoparticles for the targeted delivery of RNAi effectors to RA-promoting monocytes. (kcl.ac.uk)
  • Through the use of small interfering RNA (siRNA), RNAi-based technologies can be used to silence disease-causing genes. (technologynetworks.com)
  • The therapeutic relevance of siRNA is in jeopardy unless it can be delivered in a targeted fashion. (technologynetworks.com)
Discovered the RNAi mechanism1
  • In 1998, Andrew Fire at Carnegie Institution for Science in Washington DC and Craig Mello at University of Massachusetts in Worcester discovered the RNAi mechanism while working on the gene expression in the nematode, Caenorhabditis elegans. (wikipedia.org)
Investigational4
  • Sanofi Genzyme and Alnylam Report Positive Results from Ongoing phase 2 Open-Label Extension Study with Investigational RNAi Therapeutic Fitusiran in Patients with Hemophilia A and B With or. (sanofi.com)
  • Fitusiran is an investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of patients with hemophilia A and B, that is designed to lower levels of AT with the goal of promoting sufficient thrombin generation upon activation of the clotting cascade to restore hemostasis and prevent bleeding. (sanofi.com)
  • This comprehensive Phase 3 program represents the largest clinical program conducted to date for an investigational RNAi therapeutic program. (theeveningleader.com)
  • The company also said it will continue to focus on the HELIOS-B phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of cardiomyopathy of ATTR amyloidosis. (medscape.com)
ONPATTRO1
  • ONPATTRO is an intravenously administered RNAi therapeutic targeting transthyretin (TTR). (businesswire.com)
MiRNA2
  • Moreover, the … invention concerns methods and compositions for generating miRNA profiles and employing such profiles for therapeutic, diagnostic, and prognostic applications. (genomeweb.com)
  • Further investigations are now required to understand the biological, diagnostic, and therapeutic implications of miRNA/RNAi biogenesis defects in HD and related neurodegenerative disorders. (lu.se)
Antibodies1
  • Therapeutic Candidate or Device The therapeutic candidate is COVID-19 convalescent plasma (CCP) Indication The target indication is treatment of severe COVID-19 infection Therapeutic Mechanism Neutralizing antibodies are part of the humoral response of the adaptive immune system against viruses, and can be detected in plasma of convalescent individuals. (ca.gov)
Antisense6
  • AROAPOC-3 is an antisense rnai oligonucleotide commercialized by Arrowhead Pharmaceuticals , with a leading Phase III program in Familial Chylomicronemia (Type I Hyperlipoproteinemia). (pharmaceutical-technology.com)
  • The Phase 2 grant builds upon a successfully completed Phase 1 program that demonstrated the feasibility of using single-stranded antisense drugs to target the RNAi pathway. (news-medical.net)
  • We are pleased with the continued support from the NIH in our ongoing efforts to apply our RNA technologies, oligonucleotide chemistries and expertise to exploit antisense mechanisms, such as RNAi, for therapeutic benefit. (news-medical.net)
  • RNAi is an antisense mechanism that involves using RNA to target a specific messenger RNA sequence (mRNA) for degradation. (news-medical.net)
  • Although most currently practiced methods of inducing the RNAi mechanism utilize double-stranded RNAs, RISC requires only the antisense strand to function. (news-medical.net)
  • This provides the opportunity to develop simplified single-stranded antisense drugs that can utilize the RNAi pathway to treat human disease. (news-medical.net)
Mammalian cells2
  • Thomas Tuschl and colleagues soon reported in Nature that synthetic siRNAs could induce RNAi in mammalian cells. (wikipedia.org)
  • The identification of RNAi activity against HIV-1 presents a new approach to study viral infections and a proof of concept of RNAi antiviral activity in mammalian cells. (scienceopen.com)
Mechanism3
  • Our understanding of the mechanism has evolved rapidly, resulting in significant interest in its therapeutic potential. (rcsi.com)
  • Given the potential of the mechanism, and the impressive interest in RNAi for so many applications, it appears likely that RNAi will make a name for itself as a whole new therapeutic modality. (rcsi.com)
  • Indication COVID-19 positive or negative ARDS patients Therapeutic Mechanism It will hopefully improve oxygenation in ARDS patients and potentially decrease mortality. (ca.gov)
Transthyretin2
  • Phenotypic and genotypic heterogeneity in transthyretin-related cardiac amyloidosis: towards tailoring of therapeutic strategies? (medscape.com)
  • RNAi Therapeutic for the treatment of the polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in adults. (mpg.de)
Pathway2
  • The RNAi pathway is being used as a research tool and has great therapeutic potential. (videolectures.net)
  • Much of the work will focus on optimizing the chemical properties of single-stranded oligonucleotides that trigger the RNAi pathway. (news-medical.net)
Hereditary1
  • demonstrate through studies on induced pluripotent stem cell-derived cortical neurons that miR-33a is a potential therapeutic target for the treatment of SPG4-related hereditary spastic paraplegia. (portlandpress.com)
Antiviral1
  • R.P. van Rij and R. Andino review the role of RNAi as a therapeutic antiviral agent and its use by the host and virus during viral infections. (cdc.gov)
Targets1
  • B-cell lymphomas (BCLs) constitute a diverse set of tially identify new functional, diagnostic, and therapeutic tumors, both morphologically and clinically, that are mainly targets. (lu.se)
Double-stranded1
Alnylam6
  • Leqvio is the fourth Alnylam-discovered medicine using its RNAi therapeutic platform to be approved to date. (theeveningleader.com)
  • Alnylam has licenses to Arbutus Biopharma LNP intellectual property for use in RNAi therapeutic products using LNP technology. (businesswire.com)
  • Scientists from Alnylam Pharmaceuticals and Yale University found that an RNAi therapeutic can silence angiopoietin 2 ( Ang2 ), a key mediator in acute lung injury. (genengnews.com)
  • It is very gratifying to see that Max Planck spin-off company Alnylam succeded in bringing the first-ever RNAi-based drug to the market. (mpg.de)
  • Having a strong therapeutic pipeline with several RNAi candidates in advanced stages we are looking forward to see Alnylam bring more RNAi-based drugs on the market soon," says Jörn Erselius, CEO of Max Planck Innovation, the technology transfer organization of the Max Planck Society. (mpg.de)
  • If successful, we believe vutrisiran will offer convenient, quarterly subcutaneous dosing with a therapeutic profile that may potentially include cardiovascular outcome benefits," Alnylam CEO Yvonne Greenstreet, MBChB, said in the statement. (medscape.com)
Phase1
  • The acquisition includes EGEN's Phase Ib DNA-based immunotherapy product candidate EGEN-001 and its therapeutic platform technologies, TheraPlas™for delivery of DNA and mRNA, TheraSilence™for delivery of RNA, and RAST™for Cell Enabled Expression and Secretion of RNA. (inknowvation.com)
Infection4
  • A study was undertaken to investigate whether vector-delivered RNAi can transfer between hepatic cells in vitro and in mice, and whether this exchange could extend the therapeutic effect of RNAi against HCV infection. (eur.nl)
  • The new clinical data for AB-729, our RNAi therapeutic, continues to support its development as a potential cornerstone agent for the treatment of chronic hepatitis B (cHBV) infection. (biospace.com)
  • Taken together, our data suggest that RNAi could be investigated as a potential treatment for PCV infection. (scienceopen.com)
  • We demonstrate that RNAi mediated by 21-bp dsRNA specifically inhibits HIV-1 infection of permanent cell lines and primary CD4(+) T cells. (scienceopen.com)
Oncology1
  • It operates through the following segments: Oncology, Diagnostics, and RNAi. (businessinsider.com)
Silence1
  • RNAi represents a paradigm shift in the pharmaceutical industry giving clinicians and scientists the ability to selectively silence disease-causing genes. (technologynetworks.com)
Molecules1
  • A potent method, called SELEX, enables the isolation and selection of RNA molecules with desired functions and opens the potential for design of therapeutic molecules. (videolectures.net)
Lipid1
  • Tekmira's LNP technology (formerly called stable nucleic acid-lipid particles, or SNALP) encapsulates RNAi triggers with high efficiency in uniform lipid nanoparticles that are effective in delivering therapeutic compounds to disease sites. (genengnews.com)
Pipeline1
  • By integrating multiple established platform technologies, RNAimmune is aiming to develop a comprehensive mRNA drug discovery and development platform, from which the Company will enrich its therapeutic and vaccine product pipeline addressing tremendous unmet needs in treatments of viral infections, cancer and rare diseases. (prnewswire.com)
Vaccine1
  • E. Turnbull and P. Borrow provide a detailed description of the ineffectual roles of the innate and immune responses in the control of HIV and the long road ahead for development of either a prophylactic or therapeutic vaccine. (cdc.gov)
Development1
  • These discoveries led to a surge in interest in harnessing RNAi for biomedical research and drug development. (wikipedia.org)
Platform1
Potential2
Clinical2
  • Thus, an RNAi therapeutic targeting Agt ameliorated the clinical sequelae and improved fetal outcomes in 2 rodent models of preeclampsia. (jci.org)
  • Defining the unique therapeutic vulnerabilities of these subtypes of SCLC should help to focus and accelerate therapeutic research, leading to rationally targeted approaches that may ultimately improve clinical outcomes for patients with this disease. (cancerindex.org)
Patients2
  • Given the strength of the APOLLO data, including data showing the possibility of halting or improving disease progression in many patients, the therapeutic holds tremendous promise for people living with this disease. (mpg.de)
  • Therapeutic Candidate or Device Human placental hematopoietic stem cell derived natural killer cells (CYNK-001) Indication SARS-CoV-2 positive patients requiring hospital admission and have any 2 out of 3 symptoms: fever, cough, or positive disease-related chest x-ray. (ca.gov)
Compounds1
  • In addition to demonstrating that compounds optimized with Isis' chemistries produce superior results in animal models when compared to unoptimized compounds, the grant funds the discovery of RNAi-based drugs. (news-medical.net)
Methods2
  • The invention, the patent's abstract states, comprises "compositions and methods for the delivery of therapeutic agents to cells. (genomeweb.com)
  • In addition, the compositions and methods of the … invention are less toxic and provide a greater therapeutic index compared to compositions and methods previously known in the art. (genomeweb.com)
Research4
  • They won the Nobel prize for their research with RNAi in 2006. (wikipedia.org)
  • Understanding functional genomics is critical to advance genetic research and therapeutic discovery. (horizondiscovery.com)
  • Dharmacon Reagents have you covered with a complete range of genomic solutions from single-gene experiments to whole genome screens facilitating advancements in genetic research and therapeutic discovery. (horizondiscovery.com)
  • The multi-year grant will fund research by Isis to improve the stability and tissue distribution of RNAi drugs. (news-medical.net)
Discovery1
  • RNAi was the subject of the 2006 Nobel Prize for Medicine and Physiology and is regarded as one of the most important therapeutic discovery in decades," Guo said. (technologynetworks.com)
Search1
  • Check out our recent blog articles , read about gene editing and RNAi technologies on our application webpages, or search our knowledge base for recent videos , posters , protocols , web tools , and more. (horizondiscovery.com)
Drugs2
  • Read on for the scientific highlights and therapeutic trends reflected in 2018's new drugs. (acs.org)
  • The RNAi segment develops drugs with therapeutic activity based on reducing or silencing gene expression. (businessinsider.com)
Approach1
  • In applying an RNAi approach," explains Jack Elias, M.D., chair of internal medicine at Yale University School of Medicine, "we were able to observe in mice that silencing of the Ang2 protein resulted in animals that lived longer and had evidence of decreased lung injury compared to animals with the protein intact. (genengnews.com)
Treat1
  • The Leqvio approval marks the first U.S. approval of an RNAi therapeutic indicated to treat a major risk factor for a highly prevalent disease. (theeveningleader.com)