BRIEF—Adverum wins orphan designation for possible HAE therapy

24 August 2018

California-based Adverum Biotechnologies (Nasdaq: ADVM) has received an orphan designation for its ADVM-053, a preclinical gene therapy targeted at treating patients suffering from hereditary angioedema (HAE).

This conditions affects around one in 50,000 people worldwide and its side-effects — unpredictable and intense bouts of bodily swelling — can be highly disruptive to patients, occasionally even lethal.

Announced on Friday, the designation comes alongside news that Shire (LSE: SHP)  receieved US approval for its own HAE treatment.

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