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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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Symvivo’s Oral COVID-19 Vaccine Enters Clinical Trials

XTalks

Canadian clinical-stage biotech company Symvivo Corporation has developed an oral COVID-19 vaccine that entered clinical trials this week. The first healthy volunteer was dosed with the vaccine in Australia as part of the bacTRL-Spike COVID-19 Phase I clinical trial. COVID-19 Clinical Trials.

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bluebird seeks gene therapy trial restart after cancer scare

pharmaphorum

Known as Zynteglo in Europe where it is approved to treat the rare blood disorder beta-thalassemia, the gene therapy has been given the all-clear by bluebird’s scientists after reviewing the case of AML that emerged in a trial of the drug in sickle cell disease. Shares in bluebird ticked up following the announcement.

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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.

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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.

Drugs 52
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Genetic biomarkers could personalise therapy for TNBC

Drug Discovery World

.” The findings suggest it might be possible to predict who will benefit from carboplatin and docetaxel using a range of different biomarkers and suggest further investigation of these biomarkers in clinical trials is warranted.

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FogPharma clears another big-ticket financing for its ‘miniprotein’ pipeline

pharmaphorum

Ut comes just as FogPharma is preparing to start clinical trials of alpha-helical polypeptide candidate FOG-001. The Series D comes a little over 18 months after the six-year-old biotech’s third-round financing netted it around $107 million, taking its total raised to date north of $360 million.