Adverum Bio Initiates Advance Phase 1/2 Trial of ADVM-043

Adverum Biotechnologies, a clinical-stage gene therapy company targeting unmet medical needs in serious rare and ocular diseases, announced the dosing of the first patient in the ADVANCE phase 1/2 clinical trial of ADVM -043 for alpha-1 antitrypsin (A1AT) deficiency. The ADVANCE clinical trial is designed to evaluate the safety and protein expression following a single administration of ADVM-043, Adverum's novel gene therapy candidate.

"We are excited to dose the first patient in the ADVANCE trial, which is an important achievement for Adverum as we are now in the clinic developing a potential new treatment option for individuals with A1AT deficiency," said Amber Salzman, Ph.D., president and chief executive officer of Adverum Biotechnologies. "We are working diligently to develop ADVM-043 as a single -administration gene therapy for individuals living with this disease."

"Patients with A1AT deficiency need new therapeutics to prevent the progression of this disease," said Charlie Strange, M.D., professor of pulmonary and critical care medicine at the Medical University of South Carolina (MUSC) and principal investigator for the Alpha-1 Foundation Research Registry, the largest registry of individuals with A1AT deficiency. "We are excited to be part of this phase 1/2 study evaluating ADVM-043, a gene therapy approach that has demonstrated promising potential in preclinical studies."

The ADVANCE phase 1/2 clinical trial is a multi-center, open-label, dose -escalation study of ADVM-043 in patients with A1AT deficiency. The study will include up to 20 patients across up to four dosing cohorts of up to 5 patients each. The first cohort will receive an intravenous (IV) low dose of ADVM-043 of 8E13 total vg (equivalent to approximately 1E12 vg/kg based on an 80-kg patient). The next two cohorts will receive an intermediate IV dose or high IV dose, with the fourth cohort potentially evaluating intrapleural (IP) delivery of ADVM-043.