Remove Drugs Remove Gene Expression Remove RNA
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CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Pharmaceutical Technology

Last week, CAMP4 Therapeutics announced the close of a $100 million Series B round , which will be used to advance their regulatory RNA (regRNA)-focused programs. In preparation for this, drug manufacturing will begin later this year and will be outsourced to external contract manufacturing organisations (CMOs).

RNA 245
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US FDA grants Fast Track status for Avidity’s AOC 1020 to treat FSHD

Pharmaceutical Technology

The US Food and Drug Administration (FDA) has granted Fast Track designation for Avidity Biosciences’ AOC 1020 to treat facioscapulohumeral muscular dystrophy (FSHD). AOC 1020 has been designed for the treatment of the underlying cause of FSHD, which is caused by the abnormal expression of a gene known as double homeobox 4 or DUX4.

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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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#news #biotech Researchers identify key RNA “gatekeeper” in gene expression, pointing to possible new drug targets

BioTech 365

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Researchers identify key RNA “gatekeeper” in gene expression, pointing to possible new drug targets.An

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Solving ancient problem of nucleic acid synthesis helps to design new antiviral drugs

Scienmag

An international team of scientists from the University of Turku, Finland and PennState University, USA have solved a long-standing mystery of how living organisms distinguish RNA and DNA building blocks during gene expression paving the way for the design of new antiviral drugs. All cellular […].

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Angelman syndrome Pipeline: Unmet needs call for a robust pipeline

Delveinsight

Ovid Therapeutics is running Phase III clinical trials for its drug, Gaboxadol ( OV101 ). The drug is a small molecule, which selectively activates GABAA receptors located on the surface of nerve cells and away from the synapse. OV101 is considered as the first compound that targets disrupted tonic inhibition.

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Molecular Diagnostic Tests: The Unsung Hero of Precise Clinical Decision Making

Roots Analysis

Molecular diagnostic tests refer to tests intended to detect specific sequences in human genomic samples, such as DNA or RNA, in order to diagnose a particular disease. This technology can identify genetic variations, mutations, and expression patterns associated with various health conditions. What are Molecular Diagnostic Tests?