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CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Pharmaceutical Technology

Last week, CAMP4 Therapeutics announced the close of a $100 million Series B round , which will be used to advance their regulatory RNA (regRNA)-focused programs. The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder.

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Merck and Orna partner for RNA technology-based vaccines and therapies

Pharmaceutical Technology

Merck (MSD outside North America) has entered a partnership agreement with Orna Therapeutics for discovering, developing and marketing various programmes based on next-generation RNA technology. These programmes will include therapies and vaccines in infectious disease and oncology areas.

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Jupiter Neurosciences Makes Way in CNS Treatments with $11M IPO

XTalks

Jupiter Neurosciences, a clinical-stage pharma company specializing in neuroinflammation, made its public market debut with an initial public offering (IPO) on the Nasdaq Capital Market under the symbol “JUNS.” ” The company priced 2,750,000 shares of common stock at $4.00

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Donidalorsen Shows Sustained HAE Attack Reduction According to New Trial Data

XTalks

Carlsbad, California-based Ionis Pharmaceuticals announced it will be sharing promising new trial data for its RNA-targeted prophylactic treatment donidalorsen for hereditary angioedema (HAE) at the 2025 American Academy of Allergy, Asthma & Immunology (AAAAI)/World Allergy Organization (WAO) Joint Congress in San Diego, California.

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Sanofi’s Qfitlia Gets FDA Approval as First siRNA Therapy for Hemophilia

XTalks

Through RNA silencing, it targets the expression of antithrombin, a protein that inhibits blood clotting. Qfitlia is the second RNA interference therapy to receive a highly anticipated FDA approval recently. Qfitlia will be entering a small but competitive market. Sanofi is not new to the hemophilia market.

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Solid Biosciences partners with Phlox for cardiac disease therapies

Pharmaceutical Technology

The collaboration will use the vector biology, manufacturing capabilities and the drug development experience of Solid Biosciences and Phlox Therapeutics’ knowledge in genetic cardiomyopathies and RNA therapies. The post Solid Biosciences partners with Phlox for cardiac disease therapies appeared first on Pharmaceutical Technology.

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Beyond the pandemic: the potential of mRNA technology

pharmaphorum

Previously, messenger RNA (mRNA) therapies were a niche part of the global R&D pipeline, now a wide section of the public is at least familiar with the name of this type of therapy. The company has also stated, at the same time as the study was announced, that it will look at developing mRNA technology in oncology and genetic diseases.

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