Cambridge, Mass.-based Avrobio said today that it expanded its pipeline to include a preclinical program focused on a gene therapy for Gaucher disease.
The investigational gene therapy makes use of a patient’s blood stem cells, genetically modifying them with a fully functional copy of the faulty gene responsible for the glucosylceramide accumulation that is characteristic of Gaucher disease. Avrobio licensed the technology from Lund University’s Dr. Stefan Karlsson, but did not disclose the terms of the agreement.
“The addition of a gene therapy targeting a [lysosomal storage disorders] with compelling preclinical proof-of-concept is an excellent strategic fit for Avrobio,” president & CEO Geoff MacKay said in prepared remarks. “Our talented team of industry veterans enables us to accelerate the development of this program with the ultimate goal of benefiting Gaucher disease patients worldwide. IND-enabling activities are currently underway in preparation for a Phase 1/2 clinical study. We are excited to add this program to our portfolio as we continue to focus on building our pipeline of gene therapies to treat rare diseases.”
Gaucher disease is caused by a hereditary deficiency of an enzyme, glucocerebrosidase, and occurs in up to 1 in 40,000 live births, according to Avrobio. Traditional therapies include lifelong, expensive intravenous infusions of enzyme replacement therapy.
Avrobio said that it aims to eliminate the need for these infusions with its gene therapy.
I have a three years and 7 months old daughter with Gaucher Disease type 1, Her health is deteriorating and she is not on ERT because we can not afford . I am requesting to volunteer for your gene therapy trials .
Please contact me to this email