Avrobio has received no objection from Health Canada for their clinical trial application for their Phase 1/2 study of an investigational gene therapy for Gaucher disease. Following this news, Avrobio plans to go ahead with the study in 2019. For more detailed information you can view the source press release at Globe Newswire by clicking here.
About Gaucher Disease
Gaucher disease is a lysosomal storage disorder that can affect many areas of the body and can cause a wide range of symptoms that often vary between patients. According to the NIH, Gaucher disease is caused by mutations to a gene that prevents an enzyme, called beta-glucocerebrosidase, from functioning properly. This enzyme is involved in breaking down a fatty substance called glucocerebroside into a sugar. However, people with Gaucher disease often do not have enough copies of the functioning enzyme to carry out this process, and so the fatty substance can build up to toxic levels and cause damage.
About the Investigational Gene Therapy
Avrobio is developing a gene therapy called AVR-RD-02 as a potential treatment for people with Gaucher disease. AVR-RD-02 is designed to carry a functional copy of the gene that codes for the enzyme that patients with Gaucher disease are lacking. By doing this, researchers hope that they can help patients’ bodies to produce enough functional copies of the enzyme.
Research into AVR-RD-02
AVR-RD-02 will be assessed in a Phase 1/2 clinical trial, which is expected to enrol between eight and sixteen patients diagnosed with Type 1 Gaucher disease. All the patients that take part will be given the gene therapy, and then followed up for 52 weeks. Researchers are hoping to assess the safety and effectiveness of the therapy and will use measures including enzyme activity, bone mineral density, platelet counts, and others.