Rare Diseases

Rare genetic diseases arise when our genes do not function properly. Gene therapy is proven to deliver dramatic disease-modifying effects and potentially life-long cures from a single infusion of genetically-modified cells. A few gene therapies have already received regulatory approval, and together with a growing number of gene therapy clinical trials, the patient benefits have been remarkable. They provide evidence for the long-sought promise of gene therapy to deliver cures, or at least long-term benefit, for many severely disabling diseases. With the maturing of the technology, the time has come to expand the range of gene therapies for rare diseases.

AVROBIO is working to meet this challenge by developing innovative gene therapies for a wide range of rare diseases, including lysosomal storage disorders.


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Dahl M et al. Lentiviral gene therapy using cellular promoters cures type 1 Gaucher disease in mice.
Mol. Ther. 23(5):835-44, 2015