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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