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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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Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug

pharmaphorum

So far, human trial data for gene-editing drugs have been generated for ex vivo therapies, meaning the CRISPR technology is used to modify cells outside the body, which are then reinfused into the patient. — Eric Topol (@EricTopol) June 26, 2021. . — Eric Topol (@EricTopol) June 26, 2021.

In-Vivo 105
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The Vital Role of Biomarkers in Neurodegenerative Pathways Early Phase Unit, Worldwide Clinical Trials

Worldwide Clinical Trials

Despite their clinical heterogeneity, these diseases often share overlapping pathological mechanisms, including protein misfolding and aggregation, synaptic dysfunction, neuroinflammation, and axonal degeneration. In vivo imaging using positron emission tomography (PET) provides spatial resolution of amyloid and tau deposition.

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Combining the Powers of Single Cell Sequencing and AI in Understanding Disease Biology and Drug Development

XTalks

In recent webinars by Genuity Science, formerly known as WuXi NextCODE, experts from the biotech and pharma industries spoke about leveraging the power of single cell RNA sequencing platforms and solutions in conjunction with machine learning technologies such as AI in cell biology and disease research. The Power of Single Cell Technology.

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CRISPR Cas-9 System Doubles Life Expectancy in Mice with Aggressive Cancers

The Pharma Data

Back in September, Vertex Pharmaceuticals and CRISPR Therapeutics – the company behind the CRISPR Cas-9 platform – announced that the European Medicines Agency (EMA) had granted Priority Medicines (PRIME) designation to CTX001, an investigational ex vivo CRISPR Cas-9 gene-edited therapy for the treatment of severe sickle cell disease.

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Angelman Syndrome Market: Insights Into The Recent Late-Stage Drug Failures And Novel Approaches To Treating This Rare Neurogenetic Disorder

Delveinsight

However, DelveInsight’s Angelman syndrome market analysis report estimates that several approaches focused primarily on the Gene Therapies, Topoisomerase Inhibitors, Cannabidiol, Protein Phosphatase 2A inhibitor, among others are under development to find a cure for the Angelman syndrome. GeneTx and Ultragenyx are co-developing the therapy.

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Receptor Occupancy Assays by Flow Cytometry: Benefits for Clinical Trials

XTalks

Flow cytometry is an analytical technique that is used to detect the presence of cell surface markers as well as intracellular proteins using antibodies in individual cells suspended in a buffer solution, which pass through a laser beam one at a time ( Figure 1 ). What is Flow Cytometry?