ASGCT Press Release

ASGT 12th Annual Meeting: "Bubble Boy" disese the first of many expected gene therapy successes

SAN DIEGO-In the past 20 years, gene therapy has advanced from concept to effective clinical application, according to a presentation at the American Society of Gene Therapy’s 12th Annual Meeting, May 27.

XAdenosine deficiency disorder, a condition known commonly as bubble boy disease, has been the focus of several recent international, long-term trials of gene therapy and is the treatment’s most significant success story to date. By correcting the defective genes that cause ADA deficiency, and grafting those repaired cells to patients, researchers have engineered a reliable cure.

The advancements in gene therapy that made treatment of ADA deficiency possible may soon translate to other conditions.

“The technologies and understanding gained through gene therapy for ADA deficiency will have benefits for other inherited diseases of the hematopoietic and immune systems,” said Bobby Gaspar, MD, PhD, Professor of Pediatrics and Immunology at the Institute of Child Health in London. “We are already seeing successes in other immunodeficiencies, such as Wiskott-Aldrich syndrome.”

ADA deficiency is rare, but its impact is extreme. Patients, usually diagnosed in the first six months of life, suffer a defective immune system, their bodies unable to defend against even minor infections and viruses. They are susceptible to serious and recurrent illness.

ADA deficiency was the first condition treated by gene therapy, beginning in the 1990s. Initial trials showed only modest success but, today, more than 20 patients worldwide have been cured.

The American Society of Gene Therapy (ASGT) 12th Annual Meeting is the world’s largest scientific meeting surrounding the latest developments in gene and cell therapy, attended by nearly 2,000 researchers from around the world and featuring 60 scientific presentations.

The American Society of Gene & Cell Therapy (ASGCT) is a professional non-profit medical and scientific organization dedicated to the understanding, development and application of genetic and cellular therapies and the promotion of professional and public education in the field. For more information on ASGCT, visit its website, www.asgct.org.

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