CRISPR Therapeutics
NEWS
Currently, there are no gene editing–based treatments on the market, but the technology continues its march toward potential FDA approval, with several products in mid- and late-stage trials.
In a potential leap forward for sickle cell disease, Vertex moved closer to launching its one-time treatment for sickle cell disease and transfusion-dependent beta-thalassemia.
Partners CRISPR and ViaCyte (Vertex), and Genprex are pioneers in the quest to develop a gene therapy for Type 1 diabetes.
2022 featured pivotal data that could change the landscape in non-alcoholic steatohepatitis, Alzheimer’s disease, sickle cell disease and many more indications.
The FDA lifted its partial clinical hold on bluebird’s experimental sickle cell disease gene therapy for patients younger than 18.
PACT Pharma shared results of a first-of-its-kind trial using CRISPR technology to swap a gene in a patient’s immune cell to treat solid tumors.
While worthy advances have recently been made in sickle cell disease, companies such as Graphite Bio, bluebird bio, Vertex Pharmaceuticals and Editas Medicine have loftier ambitions.
The FDA had a busy week, approving drugs, greenlighting clinical trials and other regulatory activities for Immuneering, Amylyx, Sarepta, Sanofi, and Regeneron and more.
The FDA has granted a rolling review to CRISPR Therapeutics and Vertex’s exa-cel, a potential one-time treatment for sickle cell disease and transfusion-dependent beta-thalassemia.
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