Gene Therapy, Genome Project and Scientist - Clinical Research Informer

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

XTalks

NOVEMBER 3, 2023

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.

Gene Therapy

Gene Therapy Gene Gene Editing In-Vivo

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

pharmaphorum

JANUARY 24, 2023

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.

Genetics

Genetics Drugs Genome Project Biobanking

Clinical Research Informer

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

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

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