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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.

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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.

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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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