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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. Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression.

RNA 245
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Researchers identify RNA editing events that impact gene expression and phenotype

Scienmag

CHOP researchers find small changes in RNA due to RNA editing could have a large impact on gene expression and phenotype Philadelphia, March 9, 2021–Combining computational mining of big data with experimental testing in the lab, researchers at Children’s Hospital of Philadelphia (CHOP) have identified RNA editing events that influence (..)

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

Pharmaceutical Technology

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. Avidity Biosciences’ AOC 1020 is intended to reduce the DUX4 mRNA and DUX4 protein expression in muscles in these patients. .

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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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Gene Therapy and Pharmacokinetics

Camargo

Administration routes for gene therapies can differ depending on the indication target’s delivery needs. For example, it is beneficial to administer RNA treatments for some lung diseases directly to the disease site through inhalation. Gene Expression Considerations. Route of Administration Considerations.

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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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Global Gene Expression Market Size, Share & Trends Analysis 2021-2028 by Product (DNA Chip/Microarray, Kits & Reagents), Technique (Promoter Analysis, RNA Exp.) – ResearchAndMarkets.com

BioTech 365

Global Gene Expression Market Size, Share & Trends Analysis 2021-2028 by Product (DNA Chip/Microarray, Kits & Reagents), Technique (Promoter Analysis, RNA Exp.) – ResearchAndMarkets.com Global Gene Expression Market Size, Share & Trends Analysis 2021-2028 by Product (DNA Chip/Microarray, Kits & … Continue reading → (..)