Our Focus

rare diseases

Each person inherits features in the form of genes, which are made up of a molecule called DNA. Genetic diseases involve problems with the functioning genes. Our gene therapy technology intends to make up for genetic defects by creating a functioning copy of the genes that express functional proteins and correct or address the underlying cause of the disease. We believe gene therapy has the potential to provide transformative, disease-modifying effects – potentially with life-long clinical benefits, based on a single administration.

leading the revolution –

how our gene therapy works

Our gene therapy process works by genetically modifying a patient’s own cells by adding a functional copy of the gene of interest. We have extensive expertise in viral vector design and manufacturing and transduction, which we have developed into a potent gene therapy platform with potentially broad applications in a wide variety of indications with significant medical needs.


Our Lenti-DTM product candidate is currently being evaluated in the Starbeam Study, a Phase 2/3 clinical study, for its effectiveness in treating childhood cerebral adrenoleukodystrophy (CCALD), a fatal and rare genetic disorder that affects the nervous system of young boys. Promising, but early 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.

Our Lenti-D product candidate has the potential to stabilize or prevent progression of CCALD with one treatment. Our approach involves inserting a functional copy of the ABCD1 gene in the patient’s hematopoietic stem cells outside the body (ex vivo), then re-introducing the genetically modified cells into the patient.

learn how gene therapy works to potentially treat CCALD

learn more about adrenoleukodystrophy

our ongoing clinical trial in CCALD

The Starbeam Study is a Phase 2/3 clinical trial that is evaluating our Lenti-D product candidate for the treatment of CCALD.
learn more about our Starbeam Study


encouraging clinical data support our gene therapy’s potential

Our LentiGlobin® BB305 product candidate aims to treat beta-thalassemia major and severe sickle cell disease. Recent data presented at the 20th Annual Congress of the European Hematology Association (EHA) demonstrate continued transfusion independence in the first two patients treated with beta-thalassemia major at 16 and 14 months, respectively. The third patient in the study, the first patient with severe sickle cell disease to be treated with gene therapy, has experienced increasing levels of HbAT87Q, which represented 40 percent of total hemoglobin at their six-month visit post-drug product infusion and has been free of transfusions for more than three months. This level is above the 30 percent threshold expected to demonstrate a potential clinical effect. Notably, the proportion of anti-sickling hemoglobin (HbAT87Q + HbF) accounted for 45 percent of all hemoglobin production [40% HbAT87Q + 5% HbF).

These results build on data presented at the 19th Annual Congress of the EHA in June 2014 and the 2014 American Society of Hematology Annual Meeting.

The data presented at ASH also included the first five subjects treated in the Northstar Study, including the first subject with the most severe form of beta-thalassemia (beta-0/beta-0 genotype of beta-thalassemia) to be treated with LentiGlobin. These subjects demonstrated they could produce sufficient hemoglobin to reduce or eliminate the need for transfusions to replace the hemoglobin they lack due to the disease.

LentiGlobin works by inserting a functional human beta-globin gene into a patient’s own hematopoietic stem cells outside the body (ex vivo) and then transplanting those modified cells into the patient’s blood stream through infusion, also known as autologous stem cell transplantation.

A unique amino acid substitution in the beta-globin gene in LentiGlobin confers important anti-sickling properties to help potentially address the most severe forms of sickle cell disease. Promising results from a preclinical proof-of-concept study using gene therapy to treat sickle cell disease were published in Science.

learn how gene therapy works to potentially treat beta-thalassemia

learn more about beta-thalassemia learn more about Sickle cell disease

our ongoing clinical trials in beta-thalassemia and sickle cell disease

Our LentiGlobin product candidate is being studied in three ongoing clinical trials in patients with beta-thalassemia major and severe sickle cell disease.
learn more about our ongoing clinical trials