The new gene therapies for sickle cell disease—including the gene-editing treatment Casgevy, based on research at Boston Children's Hospital—have been game-changing for the patients who have ...

At risk of defaulting on financial commitments, gene therapy firm bluebird bio has agreed to sell itself to private equity firms at a knockdown price. The FDA is scrutinising reports of ...

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Casgevy and Lyfgenia, the two cell-based gene therapies approved by the FDA, utilise the CRISPR/Cas9 genome editing technology. Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR ...

The biotech's Casgevy, a treatment for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT), which it developed with Vertex Pharmaceuticals, is now approved in the U.S ...

This latest gene therapy offering is Casgevy, which treats sickle cell disease and transfusion-dependent beta thalassemia, a blood disorder that reduces the production of hemoglobin.

Its earnings rose 12% in 2024, reaching $11.02 billion, driven by new treatments like ALYFTREK and CASGEVY. With its stock price up 17% year-to-date and 68 hedge funds holding stakes, analysts expect ...

The company is anticipating continued growth in fiscal 2025 with the approval of JOURNAVX, the launch of its fifth CF medicine, ALYFTREK, and the continuing global acceptance of CASGEVY ...

Casgevy, which carries a $2.2 million list price, won FDA approval in December 2023 to treat sickle cell disease, and a month later received agency authorization to treat transfusion-dependent ...

Vertex is still in the early stages of the commercial launch of Casgevy, a one-time treatment for sickle cell disease and transfusion-dependent beta-thalassemia. The company now has more than 50 ...

Bluebird Bio will sell itself to private equity firms Carlyle and SK Capital for about $30 million. Bluebird makes three gene therapies: Zynteglo for beta thalassemia, Lyfgenia for sickle cell ...