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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. CAMP4’s CSO David Bumcrot PhD tells Pharmaceutical Technology that the company plans to see clinical trials go forward for their urea cycle disorder programs late next year.

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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. “We

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Therapy for rare skeletal disorder granted orphan status

Drug Discovery World

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to SiSaf’s small interfering RNA (siRNA) therapeutic for skeletal disorder Autosomal Dominant Osteopetrosis Type 2 (ADO2). There are currently no approved treatments for Osteopetrosis ADO2 and no other treatments currently in clinical trials.

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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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Colorectal Cancer Awareness Month: Drug discovery innovation

Drug Discovery World

In July 2022, DDW reported that molecular genetics company Mainz Biomed had enrolled its first patient in an international clinical study assessing the potential combination of gene expression (mRNA) biomarkers with a diagnostic test for colorectal cancer.

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How transcriptomics is driving drug discovery

Drug Discovery World

SB: Transcriptomics is the study of genomic RNA transcripts in a biological system. RNA transcript expression levels can provide insight into underlying biology – eg. which genes are upregulated, and which ones are downregulated – which is very useful when studying different disease states. SB: Certainly.

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Gene Therapy and Pharmacokinetics

Camargo

Additionally, gene editing allows us either to remove or to modify harmful genes. While most gene therapy clinical studies are ongoing, a number of products are in advanced clinical development, and several are approved by FDA. Gene Expression Considerations. Route of Administration Considerations.