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– First-ever approval of a CRISPR-based gene-editing therapy in the U.S. –– Approximately 16,000 patients 12 years of age and older with severe sickle cell disease may now be eligible for this one-time treatment –– Multiple authorized treatment centers activated –BOSTON & ZUG, Switzerland--(BUSINESS WIRE)--Vertex Ph...
The FDA's approval of Casgevy, which uses the Nobel Prize-winning gene-editing therapy to treat patients with sicke cell disease, opens the door to many other therapies.
The Foundation for the Accreditation of Cellular Therapy (FACT) has also initiated investigations into the circumstances raised by MOH over Cordlife's storage tanks being exposed to suboptimal temperatures.
FDA soon rules if gene-editing CRISPR drug Exa-cel can be used commercially to treat sickle cell disease, opening door to many other therapies.
Imetelstat is currently under regulatory review by the FDA and EMA for the treatment of transfusion-dependent anemia in adult patients with lower risk MDS ...