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An experimental gene therapy developed by the small biotech Avrobio (AVRO) reduced a type of fat that builds up to toxic levels in the cells and organs of patients with Fabry disease, the company reported Wednesday.

The newly presented clinical trial data were culled from just three patients born with the rare and sometimes deadly disease. Still, Avrobio believes the new data could start to alleviate concerns about the durability of the one-time gene therapy that surfaced last fall and ravaged the biotech’s stock price.


“We’re starting to collect substantial and consistent evidence that we’re controlling the disease,” said CEO Geoff Mackay, in an interview ahead of the presentation of the new Fabry gene therapy data at the World Symposium, a rare disease conference underway in Orlando, Fla.

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