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 has the potential to stabilize or prevent progression of cerebral adrenoleukodystrophy (CALD), a fatal and rare genetic disorder that affects the nervous system of boys and men with one treatment. Our approach involves inserting a functional copy of the ABCD1 gene in the patient’s hematopoietic stem cells outside the body, then re-introducing the genetically modified cells into the patient.
Lenti-D is currently being evaluated in the Starbeam Study, a Phase 2/3 clinical study, for its effectiveness in treating CALD. 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.
The primary efficacy endpoint for the Starbeam Study is the proportion of patients with no major functional disabilities (MFDs) at 24 months post treatment. MFDs are six components of the neurological function score (NFS) that, if present, would have a profound negative impact on patients’ lives: loss of communication, cortical blindness, tube feeding, total incontinence, wheelchair dependence and complete loss of voluntary movement. Secondary endpoints include Loes score (a method for quantifying demyelination and atrophy on brain MRI in patients with ALD), NFS (a scoring system assessing clinical deficits of CALD across 15 functional domains), gadolinium enhancement on MRI (an indicator of active neuroinflammation) and safety.
We presented the first, interim data from 17 treated patients in the Starbeam Study at the American Academy of Neurology (AAN) Annual Meeting 2016. Highlights of those findings include:
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encouraging clinical data support our gene therapy’s potential
Our LentiGlobin® BB305 product candidate aims to treat transfusion-dependent β-thalassemia (also known as β-thalassemia major) and severe sickle cell disease (SCD). 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.
We currently have three ongoing studies evaluating our LentiGlobin therapy for the treatment of transfusion-dependent β-thalassemia and severe SCD – the Northstar Study in transfusion-dependent β-thalassemia, the HGB-205 study in transfusion-dependent β-thalassemia or severe SCD and the HGB-206 study in severe SCD. As we continue to gather a growing body of data for LentiGlobin, we are learning and better understanding the potential clinical benefit our therapy can have on patients suffering from these diseases.
transfusion-dependent β-thalassemia (β-thalassemia major)
At the 57th American Society of Hematology (ASH) 2015 Annual Meeting, we presented data on 17 patients with transfusion-dependent β-thalassemia from the Northstar and HGB-205 studies. Findings include:
The data presented at the 2015 ASH Annual Meeting build on the data previously presented at the 20th Annual Congress of the European Hematology Association (EHA) and the 2014 American Society of Hematology Annual Meeting.
severe sickle cell disease
Also at the 2015 ASH Annual Meeting, we presented updated data on the first ever patient with severe SCD treated with gene therapy from our HGB-205 study and the first data on patients with severe SCD from our HGB-206 study.
The safety profile in the infused patients is consistent with autologous transplantation and no drug product-related grade >3 adverse events have been reported.
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