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Lilly and ProQR to expand genetic medicine development agreement

Pharmaceutical Technology

Eli Lilly and Company has expanded a licencing and partnership agreement with ProQR Therapeutics to discover, develop and market new genetic medicines. This alliance is utilising the Axiomer ribonucleic acid (RNA) editing platform of ProQR to address ailments affecting the liver and nervous system.

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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. By self-circularisation, Orna’s oRNA technology makes circular ribonucleic acids (oRNAs) from linear RNAs.

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Rare genetic disease caused by mutations in protein that controls RNA metabolism

Scienmag

Credit: UPMC PITTSBURGH, May 7, 2021 – In a paper published today in Nature Communications, an international group of collaborators led by researchers at UPMC Children’s Hospital of Pittsburgh have identified a genetic cause of a rare neurological disorder marked by developmental delay and loss of coordination, or ataxia.

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Chinook and Ionis partner to develop ASO therapy for kidney disease

Pharmaceutical Technology

It will use the precision medicine approach, as well as the deep expertise of Chinook Therapeutics in nephrology and knowledge of Ionis Pharmaceuticals in RNA-targeted therapeutics. Antisense therapies are designed for destroying mRNA, reducing the amount of disease-causing protein.

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Potential drug target for difficult-to-treat breast cancer: RNA-binding proteins

Scienmag

Studies using human cell lines and tumors grown in mice provide early evidence that inhibiting RNA-binding proteins, a previously overlooked family of molecules, might provide a new approach for treating some cancers Credit: UC San Diego Health Sciences In cancer research, it’s a common goal to find something about cancer cells — some sort (..)

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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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How a virus packages its genetic material

Scienmag

— Each simple RNA virus has a genome, its “native RNA.” The genome also has the code for making a capsid, the protein shell of a virus that encapsulates the genome and protects it like a nanocontainer. RIVERSIDE, Calif. This genome dictates how the virus replicates in cells to eventually cause disease.