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US FDA grants Fast Track status for Avidity’s AOC 1020 to treat FSHD

Pharmaceutical Technology

This DUX4 protein abnormal expression leads to modifications in gene expression in muscle cells which are associated with progressive muscle function loss in FSHD patients. Avidity Biosciences’ AOC 1020 is intended to reduce the DUX4 mRNA and DUX4 protein expression in muscles in these patients.

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Cash round sets up trial of InteRNA’s microRNA for cancer

pharmaphorum

million ($22 million) in second-round financing as it prepares to start clinical trials of its lead drug candidate INT-1B3 for solid tumours. InteRNA is focusing on the development of gene-silencing drugs based on microRNA, naturally occurring, non-coding strands of RNA that are thought to regulate gene expression in cells.

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How big data analytics can make personalised care a reality

Drug Discovery World

Typically, the data obtained from such studies is large and high dimensional with thousands of molecules being measured for each individual sample requiring special statistical, data science, and bioinformatics skills to integrate the information and correlate it with the clinical outcome of interest.

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What spatial biology can tell us about disease and drug discovery

Drug Discovery World

RA: With spatial biology being cross-disciplinary – in that it uses techniques in both gene expression analysis and immunofluorescence – is expertise in the field limited? There is a lack of data and case studies beyond pilot projects related to patient recruitment and clinical trials.

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CRISPR breakthroughs: New solutions for common diseases

Drug Discovery World

These methods offer safer CAR T-based therapies, laying the foundation for clinical trials and the rapid development of a new generation of CAR T cells for both autologous and allogeneic use. CRISPR diagnostics have successfully detected various pathogens, including RNA viruses (eg.

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Clinical Catch-Up: December 21-25 | BioSpace

The Pharma Data

Even with the holidays among us, there were a number of clinical trial announcements. Vir Biotechnology and GlaxoSmithKline dosed the first patient in a new sub-trial of a Phase III study of monoclonal antibody VIR-7831 for hospitalized adults with COVID-19. Here’s a look. COVID-19-Related. Non-COVID-19-Related.

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Sirnaomics Announces First Patient Dosed In Phase 2a Study of STP705 for Treatment of Cutaneous Basal Cell Carcinoma

The Pharma Data

” The Company expects to report initial clinical data from the trial in 2021. Additional information about this clinical trial is available at clinicaltrials.gov using the identifier: NCT04669808. 1 and COX-2 gene expression. About Sirnaomics, Inc. Sirnaomics, Inc.,