The Food and Drug Administration (FDA) has granted Orphan Drug designation to ADVM-053 (Adverum Biotechnologies), a novel gene therapy candidate for the treatment of hereditary angioedema (HAE).

HAE is caused by a genetic mutation that results in low levels of C1 esterase inhibitor. ADVM-053 is being developed as a single‑administration treatment that could potentially provide sustained levels of the C1 esterase inhibitor protein, which may prevent breakthrough angioedema attacks. In preclinical studies, a single dose of ADVM-053 increased C1 esterase inhibitor protein expression above therapeutic levels and decreased vascular permeability in a mouse model of HAE.

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“We are committed to developing effective treatments for patients living with HAE and the support from the FDA will be invaluable towards this goal,” said Leone Patterson, CEO of Adverum. “We look forward to submitting our IND [investigational new drug] application in the fourth quarter.” 

Orphan Drug designation is granted to treatments for rare diseases/disorders that affect fewer than 200,000 people in the US. 

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