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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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#news #biotech Lilly invests in MiNA to fund trials of protein-upregulating RNA

BioTech 365

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Lilly invests in MiNA to fund trials of protein-upregulating RNA.Lilly invests in MiNA to fund trials of protein-upregulating RNA ntaylor Tue, 07/06/2021 – 07:58 from FierceBiotech: Biotech … Continue reading → (..)

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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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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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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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Roivant joins RNA splicing push with Eisai deal

pharmaphorum

Roivant is the latest pharma group to take a position in the emerging field of therapies targeted at RNA splicing, licensing exclusive rights to a drug developed by a unit of Japanese drugmaker Eisai. . The post Roivant joins RNA splicing push with Eisai deal appeared first on.

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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.