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Rare Disease

Azafaros raises $28 million for lysosomal storage diseases

by Ryan Cross
February 15, 2020 | APPEARED IN VOLUME 98, ISSUE 7

The Dutch start-up Azafaros has raised about $28 million in series A financing to develop drugs for lysosomal storage diseases (LSDs), a group of rare metabolic diseases. Some LSDs are treated with enzyme replacement therapies, and many gene therapy firms are developing gene replacement therapies for LSDs. Azafaros is using small molecules discovered by scientists at Leiden University and Amsterdam University Medical Center. Called azasugars, the molecules block the harmful accumulation of glycolipids in LSDs.

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