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Home » Topics » Science » RNA

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Biomarkers

Nobel Prize in Physiology or Medicine for discovering microRNA

Oct. 7, 2024
By Mar de Miguel
Research into the regulation of gene expression experienced a significant breakthrough with the discovery of microRNA, small RNA molecules that do not code for proteins but control their translation. This finding has earned its authors Victor Ambros and Gary Ruvkun the 2024 Nobel Prize in Physiology or Medicine.
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Illustration of Victor Ambros and Gary Ruvkun
Biomarkers

Two win Nobel for microRNA work

Oct. 7, 2024
By Mar de Miguel
Research into the regulation of gene expression experienced a significant breakthrough with the discovery of microRNA, small RNA molecules that do not code for proteins but control their translation. This finding has earned its discoverers – Victor Ambros and Gary Ruvkun – the 2024 Nobel Prize in Physiology or Medicine “for the discovery of microRNA and its role in post-transcriptional gene regulation.”
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Feet on scale
Endocrine/metabolic

Arrowhead takes aim at obesity

Aug. 16, 2024
Arrowhead Pharmaceuticals Inc. announced its plans to advance its RNAi drug candidates ARO-INHBE and ARO-ALK7 into clinical trials for the treatment of obesity and metabolic diseases.
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Dollar sign droplet above test tube
Genetic/congenital

Airna’s series A to bring lead project into clinic, develop pipeline

Aug. 2, 2024
Airna Corp. Inc., of Cambridge, Mass., announced it has closed an oversubscribed $60 million financing round, bringing its total series A funding to $90 million.
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Liver and DNA
Genetic/congenital

IN-016 granted orphan status for progressive familial intrahepatic cholestasis therapy

Aug. 2, 2024
Innorna Co. Ltd.’s IN-016 has received orphan drug status from the U.S. FDA for the treatment of progressive familial intrahepatic cholestasis (PFIC). Earlier in July, IN-016 had been granted Rare Pediatric Disease Designation. PFIC is a group of rare genetic disorders associated with defects in bile acid secretion or transport, resulting in unwanted bile accumulation within the liver.
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RNA illustration
Drug design, drug delivery & technologies

Triastek, Biontech to collaborate on orally available RNA drugs

July 24, 2024
Triastek Inc. and Biontech SE have entered into a research collaboration and platform technology license agreement for the development of RNA therapeutics for oral delivery based on Triastek’s Melt Extrusion Deposition (MED) 3D printing technology.
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Liver illustration
Endocrine/metabolic

Liver-targeted siRNA improves body composition in mouse models

July 8, 2024
Large-scale human genetic studies have revealed that loss-of-function INHBE variants are associated with a lower abdominal to gluteofemoral fat ratio, improved metabolic profile, and reduced fasting glucose levels.
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Drug design, drug delivery & technologies

Molecure reports advances within mRNA discovery platform

June 13, 2024
Molecure SA has confirmed the in vitro activity of a molecule binding to a new mRNA target within its mRNA discovery platform. This represents the discovery of another class of molecules binding to a second mRNA biological target, which has been confirmed in in vitro assays.
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AI-generated image of person in hazmat suit in poultry farm with chickens out of focus in the background
Infection

Preclinical H5 mRNA vaccine immunizes and prevents mortality

June 7, 2024
A preclinical trial in mice and ferrets of an experimental mRNA vaccine against the H5 highly pathogenic avian influenza (HPAI) virus generated neutralizing antibodies and prevented severe illness and mortality of the animals.
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Vaccine vial and syringe
Immuno-oncology

Achilles and Arcturus collaborate on sa-mRNA personalized cancer vaccines

May 23, 2024
Achilles Therapeutics plc has established a research collaboration with Arcturus Therapeutics Holdings Inc. to evaluate best-in-class, self-amplifying mRNA (sa-mRNA) personalized cancer vaccines targeting clonal neoantigens.
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