Page 13 - Crispr News Today

Last year, Verve Therapeutics started the first human trial of a CRISPR treatment that could benefit most people—a signal that gene editing may be ready to go mainstream.

Gene therapy takes a giant leap forward as Exa-cel becomes the world’s first CRISPR/Cas9 gene-edited therapy approved to treat sickle cell disease and thalassemia.

Treatment offers relief and possibly a cure for patients with sickle cell disease and one other blood ailment

The companies changing our phones, our media and our meat.

Microbubbles were used to deliver CRISPR tools to the liver to inhibit a gene that increases cardiovascular risk.

The treatment, authorized for sickle cell disease and beta thalassemia, is being considered for approval by the FDA, with a decision expected in December.

The United Kingdom has become the first country to give regulatory approval to a medical treatment involving the revolutionary CRISPR gene editing tool.

The gene-editing therapy, called Casgevy, uses Crispr to prevent debilitating pain in patients with sickle cell disease. It also eliminates the need for regular blood transfusions in people with beta thalassemia.

The approval of a first CRISPR treatment, for sickle cell disease and beta thalassemia, is just the start for a technology still in its infancy

With a landmark U.K. approval in hand for Casgevy (exagamglogene autotemcel [exa-cel]) to treat sickle cell disease and transfusion-dependent beta thalassemia, Crispr Therapeutics AG and partner Vertex Therapeutics Inc. are turning their attention to the PDUFA dates set by the U.S. FDA for the treatment in both conditions.