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Moderna reports ‘encouraging’ early data for one of its rare disease medicines

Bio Pharma Dive

The biotech, best known for its vaccine research, said its clinical trial is the first to report results of a messenger RNA therapeutic designed for intracellular protein replacement.

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mRNA universal influenza vaccine enters clinical trials

Drug Discovery World

A clinical trial of an experimental universal influenza vaccine developed by researchers at the National Institute of Allergy and Infectious Diseases’ (NIAID) Vaccine Research Center (VRC) has begun enrolling volunteers at Duke University in Durham, North Carolina. The trial will enrol up to 50 healthy volunteers aged 18 to 49.

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Merck makes circular RNA play with $3.5bn Orna alliance

pharmaphorum

Merck & Co has ramped up its involvement in the RNA category, partnering with US biotech Orna Therapeutics in a deal valued at up to $3.5 Now, Merck has made its own play, partnering with Orna on its proprietary ‘oRNA’ technology, which stands for circular RNA. billion, including $150 million upfront.

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The Utility of Liquid Biopsy in Oncology Clinical Trials

XTalks

Gleich, MD, FACS, Senior Vice President, Medical Department, and Dr. Christopher Huth, PhD, Senior Clinical Trial Manager, Clinical Trial Management. Liquid Biopsy Use in Oncology Clinical Trials. Also, exosomes are mostly being used for the detection of circulating RNA and microRNA. “An

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RNA interference method could treat muscular dystrophy

Drug Discovery World

Japanese researchers propose using RNA interference for correcting a mistake in the genetic code of patients with Fukuyama muscular dystrophy. . The genetic anomaly blocks the chemical glycosylation of a biologically important protein. Modifying RNA function. Image credit: Mariko Taniguchi-Ikeda from Fujita Health University

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US FDA grants Fast Track status for Avidity’s AOC 1020 to treat FSHD

Pharmaceutical Technology

This DUX4 protein abnormal expression leads to modifications in gene expression in muscle cells which are associated with progressive muscle function loss in FSHD patients. Avidity Biosciences’ AOC 1020 is intended to reduce the DUX4 mRNA and DUX4 protein expression in muscles in these patients.

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CAMP4 raises $100m to take lead RNA drugs into clinic

pharmaphorum

Armed with a $100 million second-round financing, CAMP4 Therapeutics is preparing to start the first clinical trial of a drug targeting regulatory RNA (regRNA) molecules that can be used to fine-tune the expression of genes. ” The post CAMP4 raises $100m to take lead RNA drugs into clinic appeared first on. .

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