Adverum Biotechnologies, Inc. updates its next-generation ADVM-053 targeting HAE.
“We are excited to be able to share positive progress in our lead gene therapy program and to share our continued commitment to improving the quality of life for patients with unmet medical needs,” said Leone Patterson, interim president and CEO of Adverum Biotechnologies. “We are planning to submit our IND for ADVM-053 in HAE in the fourth quarter of this year.”
ADVM-053 (AAVrh.10-C1EI) is designed as a potential single-administration treatment to provide sustained release of the C1 esterase inhibitor (“C1EI”) protein to eliminate protein level variability and prevent breakthrough angioedema attacks. In preclinical studies, a single intravenous administration of ADVM-053 increased C1EI protein expression above therapeutic levels.