News

Gene Therapy 2.0

Levi Looks, diagnosed through newborn screening with “bubble boy” disease, is a healthy toddler thanks to gene therapy, resurgent now after early setbacks, MIT Technology Review reports.

One boy’s cure raises hopes and questions about gene therapy for sickle cell disease

Dana-Farber/Boston Children’s researchers, who are developing a gene therapy trial for sickle cell disease, commented in Stat and other outlets on a case report from a different trial.

Gene therapy trial gives Coopersville toddler second chance at life

A West Michigan toddler born with “bubble boy” disease is active and healthy, thanks to newborn screening and gene therapy, Fox 17 TV in Grand Rapids reports.

Pioneering doctor reflects on gene therapy’s trajectory

Alessandra Biffi, MD, director of gene therapy at Dana-Farber/Boston Children’s answers New Scientist’s questions about the resurgence of gene therapy after early setbacks.

Fighting genetic disease with help of HIV virus

A gene therapy trial in Italy led by Alessandra Biffi, MD, who now directs the Gene Therapy Program at Dana-Farber/Boston Children’s, has had promising results treating a devastating, lethal inherited neurological disorder, CBS Sunday Morning reports.

Gene therapy offers hope for treatment of sickle cell anemia

Scientists are finalizing plans to use gene therapy to treat one of the world’s most widespread inherited diseases – sickle cell anemia, the Guardian reports. The technique could begin trials next year, say researchers.

Finally, hope for a young patient with CGD

At 16, Brenden was so sick that he started planning his funeral, Harvard Gazette reports. Today, Brenden, who has a rare immune disorder called chronic granulomatous disease, is 23 and preparing, with cautious optimism, to return to college. What changed? He participated in a gene therapy clinical trial to correct a disease that severely weakens the infection-fighting abilities.

BCL11A-based gene therapy for sickle cell disease passes key preclinical test

A precision-engineered gene therapy virus, inserted into blood stem cells that are then transplanted, markedly reduced sickle-induced red-cell damage in mice with sickle cell disease, according to a study in the Journal of Clinical Investigation. The work sets the stage for bringing a decades-old discovery about sickle cell disease to the bedside. A clinical gene therapy trial, using a virus rendered harmless in the laboratory, is expected to launch in 2017.

Gene therapy trial wrenches families as one child’s death saves another

MIT Technology Review reports on a gene therapy trial in Italy that has reported strong early results for an inherited neurodegenerative disease. The trial, led by Alessandra Biffi, MD – now director of gene therapy at Dana-Farber/Boston Children’s – helped a number of babies identified through newborn screening after an older sibling died of metachromatic leukodystrophy (MLD).

Science commentary explores ways to pay for success in gene therapy

Noting the potential of gene therapy to be a one-time treatment for rare and serious diseases that otherwise cost hundreds of thousands, if not millions, of dollars in chronic care over a lifetime, the authors suggest several models for valuing, pricing and developing gene therapy.