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.

Publications

Pacienza N, Yoshimitsu M, Mizue N, Au BC, Wang JC, Fan X, Takenaka T, Medin JA. Lentivector transduction improves outcomes over transplantation of human HSCs alone in NOD/SCID/Fabry mice.
Molecular Therapy: J. American Society Gene Therapy 20(7):1454-61 (2012).

Higuchi K, Yoshimitsu M, Fan X, Guo X, Rasaiah VI, Yen J, Tei C, Takenaka T, Medin JA. Alpha-galactosidase A-Tat fusion enhances storage reduction in hearts and kidneys of Fabry mice.
Molecular Medicine 16(5-6):216-21 (2010).

Yoshimitsu M, Higuchi K, Ramsubir S, Nonaka T, Rasaiah VI, Siatskas C, Liang SB, Murray GJ, Brady RO, Medin JA. Efficient correction of Fabry mice and patient cells mediated by lentiviral transduction of hematopoietic stem/progenitor cells.
Gene Therapy 14(3):256-65 (2007).

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