Drugs, Gene Editing and Genome Project - 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. CRISPR works as genetic scissors to edit parts of the genome.

Gene Therapy

Gene Therapy Gene Gene Editing In-Vivo

CRISPR therapies targeting the next breakthrough in oncology

pharmaphorum

APRIL 20, 2022

The potential to develop treatments with CRISPR gene editing is hinged on its ability to add, delete or replace elements within a target DNA sequence. The potential for CRISPR gene editing to offer a therapeutic option to patients is clear, based on its mechanism, but there are already gene editing therapeutics on the market.

Gene Editing

Gene Editing DNA Gene Genetics

Clinical Research Informer

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

CRISPR therapies targeting the next breakthrough in oncology

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