Boston Medical Center to offer first US-FDA approved gene therapies for sickle cell disease

Boston Medical Center (BMC), a national leader in the treatment of sickle cell disease for more than 50 years, announced that it will offer the first-ever gene therapies for sickle cell disease, including one that uses a type of novel genome editing technology. The new therapies leverage the latest advances in medical science to alleviate the severe painful vaso-occlusive crises (VOCs) associated with sickle cell disease, in a long-awaited step toward equity for a disproportionally impacted Black patient population.

The announcement comes as the FDA today granted approval of a new gene editing therapy, jointly developed by Boston-based Vertex Pharmaceuticals and CRISPR Therapeutics of Switzerland, along with approval of a separate gene therapy developed by bluebird bio of Somerville, Mass., for sickle cell disease. BMC is the only hospital in New England to offer the therapies to eligible adults with sickle cell disease.

“Boston Medical Center has a longstanding commitment to providing advanced clinical care to those with sickle cell disease,” said Dr. Jean-Antoine Ribeil, MD, PhD, clinical director of the Center of Excellence in Sickle Cell Disease at Boston Medical Center and an internationally renowned haematologist who has dedicated his career to the development of gene therapies for patients with sickle cell disease and beta thalassemia. “The new gene therapies can be life changing for eligible patients who are impacted by sickle cell disease and the extreme pain that it causes, with previously limited treatment options.”

Sickle cell disease is a debilitating and chronic disease that afflicts more than 100,000 Americans and predominantly impacts people of African descent, representing 1 out of every 365 births. The disease is caused by a gene mutation that makes blood cells misshapen which can lead to strokes, organ damage and episodes of agonizing pain. Those with sickle cell disease often rely on regular blood transfusions to ease pain and reduce risk of additional complications.

The new Vertex Pharmaceuticals treatment is the first therapy to treat a genetic disease with the CRISPR gene-editing technique. Vertex began clinical trials on the therapy in the United States in 2018. The SCD clinical trial enrolled a patient population that was predominantly of African ancestry. The FDA reviewed the safety and effectiveness of the treatment in that trial before today’s approval.

Bluebird bio’s new therapy, also approved by the FDA following clinical trials, is a one-time gene therapy that has the potential to resolve vaso-occlusive events and is custom-designed to treat the underlying cause of sickle cell disease.

Both treatments will be offered to select, eligible adult sickle cell patients at BMC as part of its Center of Excellence in Sickle Cell Disease, which is the largest center of its kind in New England, serving approximately 600 adult and paediatric patients annually. The introduction of these therapies gives those with sickle cell disease more options to try to achieve a life without chronic pain.

The BMC Center of Excellence in Sickle Cell Disease has long taken an innovative and multidisciplinary approach to caring for patients suffering from this debilitating disease. The Center pairs hematologists with specialists, such as pulmonologists, nephrologists, and primary care doctors who understand the needs and many complications of patients with sickle cell disease. The addition of cutting-edge gene therapy to its suite of services reflects BMC’s ongoing commitment to this population and to being a leader in offering advanced, equitable care to its patients.

Boston Medical Center models a new kind of excellence in healthcare, where innovative and equitable care empowers all patients to thrive. Boston combine world-class clinicians and cutting-edge treatments with compassionate, quality care that extends beyond our walls.

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