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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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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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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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Gene Therapy and Pharmacokinetics

Camargo

When developing a clinical development program for these increasingly popular therapies, it is important that sponsors use modeling and pharmacokinetic (PK) analysis to evaluate parameters that can be measured while dosing with gene therapy drugs, to characterize exposure-response data and inform rational dosing.

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3rd RNA-Targeted Drug Discovery Summit

pharmaphorum

The world of drug discovery and chemical probes is still protein-centric and developing highly selective small molecules targeting RNA is often considered to be an insurmountable challenge. Why You Should Attend the RNA- Targeted Drug Discovery Summit. The post 3rd RNA-Targeted Drug Discovery Summit appeared first on.

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Risk adjusted net present value: What is the current valuation of Moderna’s MRNA-0184?

Pharmaceutical Technology

The rNPV model is a more conservative valuation measure that accounts for the risk of a drug in clinical development failing to progress. MRNA-0184 Overview mRNA-0184 is under development for the treatment decompensated heart failure. The therapeutic candidate consists of messenger RNA (mRNA) encoding for relaxin.

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Sanofi wagers $400m on miRecule muscular dystrophy therapy

pharmaphorum

Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRecule. billion deal in 2018. The aim will be to identify promising candidates in areas of “unmet patient need.”

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