AVROBIO Announces Expansion of Phase 2 trials of AVR-RD-01 Gene Therapy for Fabry Disease

A lysosome is a membrane-bound cell organelle that contains digestive enzymes. Lysosomes are involved with various cell processes. They break down excess or worn-out cell parts. They may be used to destroy invading viruses and bacteria. If the cell is damaged beyond repair, lysosomes can help it to self-destruct in a process called programmed cell death, or apoptosis.

AVROBIO, Inc has announced that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application for AVR-RD-01, its gene therapy candidate for the treatment of Fabry disease.

The company is now able to expand the phase 2 trial to patients at clinical sites in the United States. Currently, clinical trials are underway on Australia, and only 7 patients have been dosed with the treatment so far. The expansion of the clinical trial to the United States will allow AVROBIO to find more patients, which is critical, as Fabry disease is estimated to occur in only 1 in ~80,000 people, thus finding patients for clinical trials is difficult.

AVR-RD-01 is a lentiviral gene therapy that is designed to provide a permanent cure for Fabry disease by inserting functional copies of the GLA gene that encodes active a-AGA ( the enzyme that is deficient in Fabry disease ) into the patient’s stem cells.

You can find out more from AVROBIO’s press release: http://investors.avrobio.com/news-releases/news-release-details/avrobio-inc-announces-fda-clearance-investigational-new-drug


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