Remove Genetic Disease Remove Licensing Remove Protein Remove RNA
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MiNA and BioMarin partner to speed development of RNAa therapies

Pharmaceutical Technology

MiNA Therapeutics has entered into a research collaboration and option licensing agreement with BioMarin Pharmaceutical to speed up the development of therapeutic ribonucleic acid activation (RNAa) candidates to treat rare genetic diseases. The option licensing agreement is based on early-stage clinical results.

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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.

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Biopharma Money on the Move: December 2 – 8

The Pharma Data

Through its Shielded Living Therapeutics platform, the company is developing functional cures for chronic diseases. The impact of CNS diseases extends beyond patients—to their families and society as well.” Laying down a new track for RNA processing, Remix launched with $81 million in financing. Pear Therapeutics.

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Top 10 Fastest Growing Biotech Companies in 2023

XTalks

Related: Top 30 Pharma Companies in 2023: Statistics and Trends 1) Moderna Compound annual growth rate: 415 percent Moderna, headquartered in Massachusetts, is a prominent biotechnology company specializing in RNA therapeutics, particularly mRNA vaccines. Alpine Immune Sciences has recently announced an augmentation in its R&D investment.

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Eloxx Pharmaceuticals to Report Third Quarter 2020 Financial Results and Provide Business Update on November 5, 2020

The Pharma Data

is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are formulated to treat rare and ultra-rare premature stop codon diseases. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA.

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Advances in neuroscience drug discovery

Drug Discovery World

Indeed, we have shown that the causative mutations of some leukodystrophies affect the subunits of an important cellular enzyme, RNA polymerase III, preventing its normal assembly – it turned out that Riluzole can counteract this assembly defect,” says Maxime Pinard, the researcher responsible for the project in Coulombe’s lab. ‘’For

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