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FDA grants approval for bluebird’s Zynteglo to treat beta-thalassemia

Pharmaceutical Technology

The US Food and Drug Administration (FDA) has granted approval for bluebird bio ’s Zynteglo (betibeglogene autotemcel, beti-cel) for the treatment of the underlying genetic cause of beta?thalassemia 0 genotypes. thalassemia in adult and paediatric patients.

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Can genetic data be a magic bullet for drug R&D?

pharmaphorum

Drug development has long been an issue for the pharma industry, due to the expense and the high failure rate of potential treatments. Ben Hargreaves finds that the vast amount of genetic data that exists today could help provide a faster, more targeted way of developing new drug candidates.

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The Role of Oncology Biomarkers in Personalizing Hematology Treatment Plans

Worldwide Clinical Trials

Historically, these indications have challenged the one-size-fits-all treatment approach due to patient variability, such as genetic differences in drug metabolism and underlying health conditions. This resistance may occur due to genetic mutations, epigenetic changes, or a tumor’s microenvironment.

Genetics 195
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HFpEF vs. HFrEF: How To Improve Heart Failure Drug Development

XTalks

Experts from Servier and Genuity Science recently spoke on a webinar about using genomics data to drive drug development in heart failure and identify new targets for novel therapeutics. However, these drugs have shown limited effectiveness in treating HFpEF. Case Study: Genomic Approaches in Coronary Artery Disease Drug Development.

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How Pharmacogenomics may finally realise its promise

pharmaphorum

More than 60 percent of all American adults take prescription drugs , amounting to approximately 131 million individuals. These drugs have been rigorously tested by regulatory bodies around the world before they’re made available to ensure they work as labelled, but despite that, adverse events crop up.

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Top 30 New Medical Devices of 2024

XTalks

Agent drug-coated balloon. The balloons outer surface is coated with the drug paclitaxel, a safe and effective measure to prevent the arteries from narrowing again. There, plasma is separated, and tumor DNA is identified by detecting genetic mutations, methylation patterns and fragmentation signals.

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Osimertinib Resistance and EGFR Mutations in NSCLC Treatment

Bioengineer

Another genetic change at the C797 residue in the ATP binding region of EGFR’s exon 20 has emerged as a key mechanism of resistance to osimertinib. G796R and L792 are rare mutations reported in NSCLC treated with osimertinib and may interfere with the drug’s binding, thereby reducing its efficacy.

Genomics 119