Remove Genetic Disease Remove Genome Remove Genomics Remove Protein
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CAMP4 raises $100m to take lead RNA drugs into clinic

pharmaphorum

The Cambridge, Massachusetts biotech has discovered that areas of the non-coding parts of the human genome – referred to by its chief executive Josh Mandel-Brehm (pictured above) as the “dark side” of the genome – actually produce regRNAs that control the expression of the 2% that codes for proteins.

RNA 52
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Searching for answers in rare epilepsy

pharmaphorum

Geneticist Dr Charles Steward has spent his career studying the human genome – but his work became much more personal when his children were diagnosed with severe neurological diseases. Charlie told pharmaphorum how his search for a genetic cause has led him to straddle the divide between scientist and patient advocate.

Genome 102
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Searching for answers in rare epilepsy

pharmaphorum

Geneticist Dr Charles Steward has spent his career studying the human genome – but his work became much more personal when his children were diagnosed with severe neurological diseases. Charlie told pharmaphorum how his search for a genetic cause has led him to straddle the divide between scientist and patient advocate.

Genome 80
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Sanofi wagers $400m on miRecule muscular dystrophy therapy

pharmaphorum

The new drug candidate for FSHD will combine an RNA molecule from miRecule targeting double homeobox 4 (DUX4) – a protein that is mutated in FSHD – with a nanobody developed by Sanofi that targets muscle cells. billion deal in 2018.

RNA 105
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Molecular Diagnostic Tests: The Unsung Hero of Precise Clinical Decision Making

Roots Analysis

Molecular diagnostic tests are advanced techniques and tools used to analyze biological markers in the genome and proteome. These diagnostic solutions are essential for detecting and monitoring diseases, identifying genetic abnormalities, and guiding personalized treatment plans. What are Molecular Diagnostic Tests?

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Epigenetic Editing with CRISPR Might Be Easier Than We Thought

XTalks

The tool could also prove to be safer than conventional CRISPR-based gene therapies as it does not involve DNA editing, and thus would not cause potentially harmful off-target genomic changes. Epigenetic Editing with CRISPR. DNA methylation occurs endogenously in all mammalian cells in response to various stimuli.

DNA 96
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Top 10 Fastest Growing Biotech Companies in 2023

XTalks

The company’s clinical diagnostic division provides a wide array of services, including molecular diagnostic testing, comprehensive genetic testing and high-quality anatomic pathology laboratory services. is a late-stage oncology company dedicated to developing targeted therapeutics for the treatment of metastatic disease.

Genetics 105