dm+d

Unassigned

New Medicines

Males >16 years with a confirmed diagnosis of classic Fabry's disease based on deficient AGA enzyme activity who have not previously received treatment with enzyme replacement therapy (ERT) and/or chaperone therapy within 3 years of the time of screening

Information

New molecular entity
AVROBIO
AVROBIO

Development and Regulatory status

None
None
Phase II Clinical Trials
Yes
Yes

Category

Gene therapy to be given as a single, one-off dose. Autologous haematopoietic stem cells are collected, genetically modified ex vivo using a lentiviral vector containing the functional GLA gene. Cells are infused back into the patient. [2]
Anderson-Fabry disease is an X-linked recessive inborn error of glycosphingolipid metabolism caused by deficiency of alpha-galactosidase A. It is estimated to occur in 1 in 55,000 males in the classical form but the atypical variant may be more common [1].
Males >16 years with a confirmed diagnosis of classic Fabry's disease based on deficient AGA enzyme activity who have not previously received treatment with enzyme replacement therapy (ERT) and/or chaperone therapy within 3 years of the time of screening
Intravenous infusion