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Bluebird Bio Shares Soar as FDA Approves Gene Therapy for Rare Genetic Disorder

March 25, 2023 - Bluebird Bio, a biotechnology company, saw its shares surge on Wednesday after the U.S. Food and Drug Administration (FDA) approved its gene therapy, Zynteglo, for the treatment of beta-thalassemia, a rare genetic disorder.

Zynteglo is a one-time gene therapy that uses a lentiviral vector to deliver a functional copy of the beta-globin gene to the bone marrow of patients with beta-thalassemia. The therapy has been shown to significantly reduce the need for blood transfusions and improve the overall health of patients with the disorder.

The FDA approval comes after a priority review of the therapy, which was based on data from a clinical trial that showed Zynteglo to be safe and effective in patients with beta-thalassemia. The trial data demonstrated that 93% of patients who received the therapy had a reduction in their need for blood transfusions, with 76% of patients achieving a complete elimination of transfusions.

"We are thrilled to bring this life-changing therapy to patients with beta-thalassemia," said Andrew Obenshain, Bluebird Bio's chief medical officer. "Zynteglo has the potential to revolutionize the treatment of this devastating disorder and we look forward to working with healthcare providers to make it available to patients as quickly as possible."

Bluebird Bio's shares jumped more than 20% in morning trading following the FDA approval, with investors cheering the news. The company's stock has been on a tear in recent months, with shares up more than 50% in the past year.

The approval of Zynteglo marks a major milestone for Bluebird Bio, which has been working on the therapy for several years. The company's pipeline also includes other gene therapies for rare genetic disorders, including sickle cell disease and severe combined immunodeficiency.

The FDA approval of Zynteglo is a significant development for patients with beta-thalassemia, who have limited treatment options and often require frequent blood transfusions to manage their condition. The therapy has the potential to transform the treatment of this disorder and improve the quality of life for patients with the condition.