According to a recent article featured in Biospace, researchers found substantial evidence of a biochemical correction after treating an eleven-year girl for Gaucher Disease. This is the first pediatric case involving Avrobio’s AVR-RD-02. The gene therapy is being developed by AVROBIO as a treatment for advanced Gaucher Disease Type 3 (GD3).
AVROBIO’s CMO Essra Ridha, M.D. noted that the girl’s response to the treatment was very encouraging as GD3 patients generally do not respond (refractory) to therapy. In fact, at the fifteen-month mark, there was a thirty-three percent increase in albumin levels. AVROBIO reports this increase as an improvement in lymphadenopathy (swelling of lymph nodes). An improvement was also noted in enteropathy (intestinal disease). The child had never experienced these improvements with her current regime according to Dr. Ridha.
There was no change in her neurological status nor were there any such signs after treatment. During the same fifteen-month period, AVROBIO officials confirmed that there were no safety concerns relating to the AVR-RD-02 infusion.
Based on such encouraging data, Avrobio plans to conduct a Phase II/III clinical trial investigating the AVR-RD-02 gene therapy.
Dr. Ridha further stated that a Phase II/III trial will compare enzyme replacement therapy, the current standard of care, with that of AVR-RD-02.
Forty patients are expected to participate and randomly receive AVR-RD-02 or standard of care.
Also, regulatory U.S. and European agencies have greenlighted a late-stage trial that has the potential for wide approval.
About Gaucher Disease
Adults and children with Gaucher disease may resort to surgery and medicine to prevent organ damage and have a better quality of life.
Gaucher Disease Type 1 is one of almost fifty rare diseases that affect the lysosome, a structure in cells that when working properly breaks down proteins and other substances for recycling. It is an inherited disease and one of the most common forms. If working improperly it causes fatty substances to accumulate in the liver, spleen, and bone marrow.
Promising Data
CEO Geoff MacKay spoke of promising data from the Phase I/II GUARD 1 trial of Gaucho Disease Type 1. The gene therapy brought about a meaningful reduction of organ size as well as reduced toxic metabolites.
A drug is transformed into metabolites when it is broken down, processed, converted to chemicals, and eventually eliminated from the body. Four patient cases were discussed during the company’s presentation.
Dr. Ridha noted that three patients had reduced spleen and liver volume but the fourth patient had not been participating long enough to qualify for scanning spleen or liver volume.
One option had been the high-risk hematopoietic stem cell transplantation until the introduction of enzyme replacement therapy currently the most commonly used treatment.
In conclusion, AVR-RD-02 gene therapy demonstrated significant safety and efficacy data.
