Our Product Pipeline

Childhood Cerebral Adrenoleukodystrophy
Product Development

Our Lenti-D product candidate has the potential to be a one-time treatment to stabilize the disease and prevent progression of childhood cerebral adrenoleukodystrophy. Our approach involves the ex vivo insertion of a functional copy of the ABCD1 gene into a patient's own hematopoietic stem cells. We are involved with an ongoing Phase 1/2 clinical program in France. Early promising clinical proof-of-concept results for two patients in a French study sponsored by INSERM and licensed by bluebird bio were reported in the November 2009 issue of Science. The safety and therapeutic benefits have been maintained for more than five years, and two additional patients have subsequently been treated. We are developing a next generation product, called Lenti-D. We were granted orphan drug designation for Lenti-D by both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) in 2012.

We have initiated a larger Phase 2/3 clinical study, The Starbeam Study (ALD-102), to treat childhood cerebral adrenoleukodystrophy patients at sites in the United States and Europe in 2013. Information about the U.S. study is available on ClinicalTrials.gov or Starbeamstudy.com.