bluebird bio’s Gene Therapy Treatment for Blood Disorder Advances

bluebird bio, a gene therapy company with operations in Durham, has passed a regulatory milestone for its potential treatment of an inherited blood disorder.

The European Medicines Agency has accepted bluebird’s marketing authorization application for LentiGlobin, a gene therapy for transfusion-dependent β-thalassemia (TDT). LentiGlobin was previously granted an accelerated assessment by the regulatory agency in July, potentially reducing the review time of the application from 210 days to 150 days.

The acceptance “advances us toward our goal of providing to patients the first one-time gene therapy that addresses the underlying genetic cause of TDT,” said David Davidson, M.D., chief medical officer of bluebird bio. “We share this important milestone with the patients, families and healthcare providers who made it possible through their participation in our pioneering clinical studies of LentiGlobin.”

TDT is an inherited blood disorder caused by a mutation in the β-globin gene. It interferes with red blood cell production, leading to severe anemia. 

Supportive care for people with TDT consists of a lifelong regimen of blood transfusions to enable survival and suppress symptoms of the disease, and iron chelation therapy to manage iron overload that results from the transfusions.

Despite the supportive care, many TDT patients have serious complications and organ damage. By eliminating or reducing the need for blood transfusions, the long-term complications of TDT may be reduced.

A one-time therapy

LentiGlobin is a one-time gene therapy being studied as a potential treatment to address the underlying genetic cause of TDT, potentially eliminating or reducing the need for blood transfusions.

bluebird’s clinical development program for LentiGlobin includes ongoing studies around the world with sites in Australia, Germany, Greece, France, Italy, Thailand, the United Kingdom and the United States.
In addition, bluebird is conducting a long-term safety and efficacy follow-up study for people who have participated in bluebird-sponsored clinical studies of LentiGlobin for TDT and sickle cell disease.

The European Medicines Agency previously granted Priority Medicines (PRIME) eligibility and Orphan Medicinal Product designation to LentiGlobin for the treatment of TDT. LentiGlobin is also part of the agency’s Adaptive Pathways pilot program, intended to improve timely access for patients to new medicines.

LentiGlobin has also been granted Orphan Drug status and Breakthrough Therapy designation for the treatment of TDT by the U.S. Food and Drug Administration.

Currently the only other option for potentially correcting the genetic deficiency underlying TDT is allogeneic hematopoietic stem cell transplantation. However, complications of that procedure include a risk of treatment-related mortality, graft failure, graft-versus-host disease and opportunistic infections, particularly in patients who undergo non-sibling matched transplants.

Other treatments in pipeline

With its lentiviral-based gene therapies, T cell immunotherapy expertise and gene editing capabilities, bluebird has built a pipeline with broad potential application in severe genetic diseases and cancer.
Its gene therapy clinical programs include investigational treatments for TDT, cerebral adrenoleukodystrophy and severe sickle cell disease. The company is also developing cancer therapies based on its lentiviral gene delivery and T cell engineering, with a focus on developing novel T cell-based immunotherapies.

bluebird is a publicly held company headquartered in Cambridge, Mass., with facilities in Durham, Seattle, and Zug, Switzerland.

The Durham facility is a 125,000-square-foot manufacturing plant that bluebird purchased last year. The site’s initial build-out will allow for production of clinical and commercial supply of lentiviral vector, a critical component of the company’s gene and cell therapies. 

“Over time, it is our intent to have the (Durham) facility support all of our programs,” Stephanie Fagan, bluebird’s senior vice president of corporate communications, said in May. 

bluebird had over 500 employees then and expects to have more than 700 by the end of this year, Fagan said. 

“These headcount are to support continued commercialization efforts in the United States and European Union, along with ramping up internal manufacturing capabilities at our North Carolina facility,” she said.