Bluebird bio Tapped for Humanitarian Award for Efforts to Develop Gene Therapy for Thalassemia

Published: May 04, 2018 By

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Living with a genetic blood disorder like thalassemia can be difficult. Many patients who have forms of this genetic disease must undergo regular blood transfusions – a procedure that can hinder the quality of life.

Companies like Cambridge, Mass.-based bluebird bio are on the cutting edge of developing treatments for different forms of the disease. Earlier this year the company released stellar interim data reports from two clinical trials for transfusion-dependent beta-thalassemia (TDT) patients. The Northstar trial, which was completed in February, and the ongoing HGB-205, show a one-time treatment of bluebird’s gene therapy treatment LentiGlobin have allowed the majority of 22 dosed patients to remain free from transfusions for two years. The company is closer than ever to achieving an approved treatment for the blood disease and helping patients live a fuller life. Transfusion-dependent thalassemia is a severe genetic disease noted by reduced or absent hemoglobin production. TDT causes severe anemia and ineffective red blood cell production.

It’s for that reason, among others, that bluebird has been chosen to receive the Cooley Anemia Foundation’s Humanitarian of the Year Award at its annual gala in June. In its announcement, the foundation noted bluebird’s its lentiviral-based gene therapies and said the company has “built an integrated product platform with broad potential application to severe genetic diseases and cancer.” The foundation went on to note that the company’s one-time gene therapy has the “potential to eliminate or reduce chronic blood transfusions” in patients with transfusion-dependent β-thalassemia.

“For years, the Cooley’s Anemia Foundation has been a champion of gene therapy as a potential curative approach to thalassemia. We are thrilled to have this opportunity to recognize the exceptional work and dedication of bluebird bio in this exciting area. bluebird bio’s commitment gives hope to thalassemia patients here in the United States and around the world,” Peter Chieco, incoming National President of the Cooley’s Anemia Foundation Board of Directors, said in a statement.

Nick Leschly, bluebird’s chief executive officer, or chief bluebird as he’s known around the company, said the company was honored to receive the award. Despite being close to a potential regulatory approval for LentiGlobin, Leschly said the company’s work “is just beginning.”

“We are dedicated to proving our belief that a one-time gene therapy holds the potential to transform the treatment of thalassemia and improve the lives of patients. We could not have made the progress we’ve made, or look to the road ahead, without the tremendous partnership of the thalassemia patients and their families who have participated in our clinical studies, the study investigators, and patient advocacy organizations who come together with a common purpose to advance treatment,” Leschly added.

Bluebird will first seek regulatory approval of its TBT treatment in Europe. Following the release of the company’s first quarterly report for 2018 on Thursday, Leschly said the company is on-track to seek Marketing Authorization from the European Medicines Agency in the second half of the year. The company is awaiting additional data from two other clinical trials before filing for approval in the United States.

In addition to its plan MA filing in Europe, Leschly also pointed to advances the company has made with bb2121 chimeric antigen receptor (CAR-T) cell therapy for the potential treatment of patients with relapsed/refractory multiple myeloma. In March bluebird teamed up with Celgene for the development of bb2121.

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