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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).
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.
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.
When developing a clinical development program for these increasingly popular therapies, it is important that sponsors use modeling and pharmacokinetic (PK) analysis to evaluate parameters that can be measured while dosing with gene therapy drugs, to characterize exposure-response data and inform rational dosing.
Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Researchers identify key RNA “gatekeeper” in geneexpression, pointing to possible new drug targets.An
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 geneexpression paving the way for the design of new antiviral drugs. All cellular […].
RNA therapeutics are a novel class of biopharmaceuticals that harness the power of RNA molecules for the treatment and prevention of a wide range of disorders, including oncological, and genetic disorders as well as infectious diseases. Non-coding RNAs include antisense oligonucleotides (ASOs) and RNA aptamers.
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.
The 2024 Nobel Prize in Physiology or Medicine has been awarded to American scientists Victor Ambros and Gary Ruvkun for their groundbreaking discovery of microRNA (or miRNA) and its role in post-transcriptional gene regulation. A gene contains instructions within our DNA.
million ($22 million) in second-round financing as it prepares to start clinical trials of its lead drug candidate INT-1B3 for solid tumours. The start of dosing in the phase 1/1b trial of the drug has been delayed by the COVID-19 pandemic, but recruitment of the target 80 participants with advanced solid tumours is reported to be ongoing.
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?
Use of the drug verapamil to treat Type 1 diabetes continues to show benefits lasting at least two years, researchers report in the journal Nature Communications. The suggestion that verapamil might serve as a potential Type 1 diabetes drug was the serendipitous discovery of study leader Anath Shalev, M.D.,
For instance, GTX-102 , an antisense therapy silencing the geneexpression by binding to the RNA molecule behind the regulation of its expression, has also secured USFDA Fast Track Designation. Thus, restoring the presence of this particular gene can help to restore several of the functions of the brain.
Mechanism of Gene Switch During the transcription process, the promoter region, which is located near the upstream end of each gene, binds to transcription factor, which is a specific type of protein. An additional level of genetic control is provided by gene switches that are located upstream of the promoter region.
a biopharmaceutical company engaged in the discovery and development of RNAi therapeutics against cancer and fibrotic diseases, today announced dose administration for the first patient in a Phase 2a clinical study of the company’s lead drug candidate, STP705, for the treatment of cutaneous basal cell carcinoma. GAITHERSBURG, Md.
The drug, Yeliva (opaganib) is a first-in-class, oral spingosine kinase-2 (SK2) selective inhibitor with triple-action effect on the pathophysiological processes linked to COVID-19. Galidesivir is a broad-spectrum antiviral, an adenosine nucleoside analog that blocks viral RNA polymerase. Non-COVID-19-Related.
CTCs enable the complete picture of a cancer to be seen, as, being an intact cell, they allow DNA, RNA and protein analysis and thus provide comparable analysis to a tissue biopsy. In addition, the live CTCs harvested can be cultured, which offers the potential for testing response to drugs outside the patient.
BOSTON – An important player in the healthy development of female embryos turns out also to play a key role in regulating the behavior of chromosome loops and geneexpression in both sexes, according to a new study by researchers at Massachusetts General Hospital (MGH).
From rare disease drug approvals to treatments involving immunotherapies and gene therapies and awarding of a Nobel Prize to the inventors of the gene-editing tool CRISPR, 2020 was a year of great activity and productivity despite the backdrop of the pandemic. Drugs, Testing and Vaccines. Drug Approvals.
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