FDA Approves First Gene Therapies to Treat Patients with ...
Casgevy, a cell-based gene therapy, is approved for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises. Casgevy is the first...
The world's 1st CRISPR therapy has been approved. Here's ...
Jan 18, 2024 · Exa-cel, also known by its brand name Casgevy, received its first regulatory approval on Nov. 16, 2023 from the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to treat two...
Casgevy: UK approves gene-editing drug for sickle cell - BBC News
Nov 16, 2023 · In a world first, medical regulators in the UK have approved a gene therapy that aims to cure two blood disorders. The treatment for sickle cell disease and beta thalassemia is the first to be ...
FDA approves world's first CRISPR-based medicine for sickle cell
Dec 8, 2023 · The new medicine, called Casgevy, is made by Vertex Pharmaceuticals and CRISPR Therapeutics. Its authorization is a scientific triumph for the technology that can efficiently and precisely repair...
FDA approves cure for sickle cell disease, the first ...
Dec 8, 2023 · Casgevy, which was approved for people ages 12 and older, removes the need for a donor. Using CRISPR, it edits the DNA found in a patient’s stem cells to remove the gene that causes the disease.
FDA clears first CRISPR treatment for a second disease, beta ...
Jan 17, 2024 · CNN — The US Food and Drug Administration has approved a second use for the first CRISPR-based medicine, Casgevy, which was approved in December to treat sickle cell disease. FDA approves two...
Positive Results from Pivotal Trials of CASGEVY ...
Dec 11, 2023 · BOSTON -- (BUSINESS WIRE)--Dec. 11, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced two oral presentations at the American Society of Hematology (ASH) Annual Meeting and Exposition from the global pivotal trials of CASGEVY™ (exagamglogene autotemcel [exa-cel]).
Vertex Presents Positive Long-Term Data On CASGEVY ...
Jun 14, 2024 · CASGEVY is the first and only approved CRISPR-based gene-editing therapy. The data being presented are from more than 100 patients (46 SCD; 56 TDT) treated with exa-cel in clinical trials, with the longest follow-up now extending more than 5 years.
The world’s first CRISPR therapy is approved: who will ...
Nov 21, 2023 · Casgevy (exagamglogene autotemcel) is a first-of-its-kind treatment made by Vertex Pharmaceuticals and CRISPR Therapeutics in Zug, Switzerland. It comes just 11 years after Jennifer Doudna and...
Casgevy: First CRISPR gene-editing treatment approved in U.S.
Dec 8, 2023 · The U.S. Food and Drug Administration has approved Casgevy, the first gene-editing treatment to be marketed in the country. Casgevy uses Nobel Prize-winning technology CRISPR to treat sickle...