http://www.raredr.com/news/first-sickle-cell-patient-gene-therapy
First Sickle Cell Patient Undergoes Gene Therapy

RDR Staff


bluebird bio announced that the first subject with severe sickle cell disease has undergone infusion with LentiGlobin BB305 drug product in an autologous hematopoietic stem cell transplantation.

The patient is enrolled in the HGB-205 Study being conducted in Paris, France. A separate US-based trial (HGB-206) in the United States is also enrolling up to 8 severe sickle cell disease patients to also test LentiGlobin BB305.  The company plans to present preliminary data from these studies in 2015.

Marina Cavazzana, MD, PhD, Professor of Medicine at Paris Descartes University and Research Director at the Centre for Clinical Research in Biotherapy, Necker Hospital, and at the Institute of Genetic Diseases, Imagine, Paris France said:

“We are treating a sickle cell patient for the first time with gene therapy”

“Sickle cell disease is a devastating disease that affects hundreds of thousands of people in the US and Europe and millions around the world. The therapeutic options for patients with sickle cell disease are currently limited, so the opportunity to bring a one-time, potentially curative treatment to these patients by modification of autologous hematopoietic stem cells would represent a great advance for patients with sickle cell disease and for the field.”

David Davidson, MD, bluebird bio’s Chief Medical Officer added:

“Sickle cell disease shortens life expectancy by decades even in developed countries, so it is exciting to contemplate that LentiGlobin may offer the curative potential of allogeneic stem cell transplantation by using a patient’s own cells.”

“In June 2014, we reported preliminary results from the HGB-205 Study demonstrating that treatment with LentiGlobin drug product led to high-level production of beta-T87Q-globin and rapid transfusion independence in two beta-thalassemia major patients. Given the anti-sickling property of the amino acid substitution engineered into beta-T87Q-globin, we are optimistic about the potential for LentiGlobin to mitigate the signs and symptoms of sickle cell disease. We anticipate providing initial clinical data on LentiGlobin in sickle cell disease patients in 2015.”

HGB-205 Study (Paris, France)

This phase 1/2 study will evaluate the safety and efficacy of LentiGlobin BB305 drug product in the treatment of subjects with beta-thalassemia major and severe sickle cell disease. The study is designed to enroll up to 7 subjects.
For more information on the HGB-205 Study, please visit www.clinicaltrials.gov using identifier NCT02151526.

HGB-206 Study (Charleston, South Carolina)

This phase 1 study will evaluate the safety and efficacy of LentiGlobin BB305 drug product in the treatment of subjects with severe sickle cell disease. The study is designed to enroll up to 8 subjects.
For more information on the HGB-206 Study, please visit www.clinicaltrials.gov using identifier NCT02140554.

About sickle cell disease

Sickle cell disease  is a rare genetic blood disorder resulting from a mutation in the beta globin gene that causes polymerization of hemoglobin proteins and abnormal red blood cell function. Sickle cell disease affects 90,000 to 100,000 Americans, mostly African Americans.  A person with sickle cell disease has red blood cells that are hard, sticky, and C-shaped (like the farm tool the "sickle"). These cells clog smaller blood vessels resulting in pain as well as increased risk for infection, acute chest syndrome and stroke.
 
Image courtesy wikimedia commons
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