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Exa-Cel CRISPR Gene Therapy for Sickle Cell Disease Deemed Safe by FDA Advisory Panel

XTalks

After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene therapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw. CRISPR works as genetic scissors to edit parts of the genome.

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As the Smithsonian wraps a landmark genome exhibit, leaders in the field reflect on what’s changed

STAT News

When the Smithsonian National Museum of Natural History opened its genomics exhibit in 2013, the field was just celebrating the 10th anniversary of the completed Human Genome Project. Sequencing that first genome cost over $500 million. The genomes since cost $10,000.

Genome 98
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Reference genome comparison finds exome variant discrepancies in 206 genes

Scienmag

HOUSTON – (June 14, 2021) – In the two decades since the Human Genome Project mapped the entire human genome, improvements in technology have helped in developing updated reference genomes used for sequencing.

Genome 40
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Q&A: Gene therapy opportunities from long-read sequencing 

Drug Discovery World

Breakthroughs in gene therapy are only possible with an exact understanding of the genetic underpinnings of disease. To develop safe and effective gene therapies, researchers need confidence that genomic data is both complete and accurate. Recent years have seen a great deal of progress in both short- and long-read sequencing.

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Realising the promise of genomic testing across oncology

pharmaphorum

Unlocking the secrets of the human genome has long been an ambitious pursuit for researchers around the world. Today, the landscape of genomic testing and research is rapidly progressing, with significant scientific and technological advances driving a paradigm shift in the understanding of oncology at a molecular level.

Genome 52
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Genomic projects exploit scale as clinical applications play catch-up

Pharmaceutical Technology

The group analysed 12,222 samples collected through whole genome sequencing efforts of the UK National Health Service as part of the 100,000 Genomes Project and added further data on 6,418 cancers from the International Cancer Genome Consortium and the Hartwig Medical Foundation. Both teams had the same underlying goal.

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

pharmaphorum

Pharma has used this to begin creating targeted treatments, such as gene therapies , which are able to hone in on gene mutations that cause health issues and eradicate or replace the faulty gene. The information gathered from the project is still providing insights today, a decade later.