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

pharmaphorum

Though instances of this kind of therapy are rare, it is a growing area. The cost of testing per human genome in 2006 was approximately $14 million , and in less than two decades, an average consumer-purchased genetic test costs $100. The information gathered from the project is still providing insights today, a decade later.

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The Biotech Effect

Pharmaceutical Technology

“However, around the time of the Human Genome Project, there was a ‘land grab’ for the new technologies as big pharmaceuticals tried to catch up paying high prices to access technology platforms in areas such as genomics and high throughput screening.”

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