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After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based genetherapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw.
Pharma has used this to begin creating targeted treatments, such as genetherapies , 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.
“However, around the time of the Human GenomeProject, 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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