Gene Therapy Platform Shows Positive Results in Fabry Disease Trial

AVROBIO has released data from the ongoing Phase 2 FAB-GT trial, which is investigating AVR-RD-01 as a treatment for Fabry disease. This ex vivo lentiviral gene therapy was shown to lower toxic substrate in they kidneys by 100%, completely clearing it. This data and information on additional trials will be presented at the 17th annual WORLDSymposium, which you can find here.

About Fabry Disease

Fabry disease is a lysosomal storage disorder that occurs when globotriaosylceramide, a type of fat, builds up in the cells. It typically affects males. This accumulation can affect multiple organs throughout the body, but typically affects the kidneys and heart. As the buildup usually begins in childhood, symptoms will worsen as one ages. These symptoms include pain in the hands and feet, dull and cloudy vision, the inability to sweat, clusters of red spots on the skin, tinnitus, loss of hearing, and gastrointestinal issues. Heart attacks, strokes, and acute kidney damage may be complications. This disease is usually diagnosed after birth, as it is picked up in newborn screening exams. Treatments are symptomatic.

Data from the Trial

In Fabry disease patients, a fatty substrate called Gb3 accumulates in the cells and causes damage to a number of organs. The heart, kidneys, and central nervous system are all impacted by this buildup.

In the Phase 2 trial, doctors observed a 100% reduction of Gb3 per peritubular capillary after one year of treatment in one patient’s kidney biopsy. This patient is the first to be treated with AVROBIO’s plato® gene therapy platform. The platform is composed of a personalized conditioning regiment, lentiviral vector, and a closed, automated manufacturing process that can smoothly and precisely deliver drugs to sites across the world.

Further results include:

  • Sustained activity of the leukocyte alpha-galactosidase A enzyme
  • Reduced levels of toxic plasma lyso-Gb3
  • Steady cardiac function and structure one year post treatment
  • There have not been any unexpected safety events
  • There were eight serious adverse events, all were resolved without clinical sequelae

Researchers are very excited by these results, especially as they have been sustained for 2.5 years. They believe that AVR-RD-01 will prove to be a better treatment option than the current standard of care.

Find the source article here.

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