Adverum Biotechnologies Doses First Patient in the ADVANCE Phase 1/2 Clinical Trial of ADVM-043 for A1AT Deficiency

Clinical Trials

Adverum Biotechnologies, Inc , 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.”

About the ADVANCE Phase 1/2 Clinical Trial of ADVM-043 for A1AT deficiency
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.

The study will be conducted at 5 leading centers in the United States. The primary endpoint is safety and tolerability and secondary endpoints include changes in plasma concentrations of both total and M-specific A1AT levels. Adverum expects to report preliminary data from this trial in the second half of 2018.

About ADVM-043
ADVM-043 (AAVrh.10-A1AT) is a gene therapy candidate that has the potential to induce stable, long-term A1AT protein following a single administration. In a preclinical proof-of-concept study, ADVM-043 demonstrated robust protein expression above therapeutic levels in mice following either IV or IP administration. In another study in non-human primates, evidence of stable long-term expression of hA1AT mRNA was observed out to one year following IP administration of ADVM-043.

About Alpha-1 Antitrypsin (A1AT) Deficiency
A1AT deficiency is a fairly common orphan disease impacting approximately 100,000 individuals in the United States. It is estimated that 20 million people carry the gene for the disease, which unfortunately can be passed to their children. A1AT deficiency is associated with premature emphysema. The disease is caused by mutations in the SERPINA1 gene, resulting in very low levels of A1AT. The current standard-of-care treatment for patients with A1AT deficiency with lung disease can be challenging, with weekly IV infusions of an alpha-1 proteinase inhibitor. The current treatment regimen can result in underdosing and lead to worsening lung function.

About Adverum Biotechnologies, Inc.
Adverum is a clinical-stage gene therapy company targeting unmet medical needs in serious rare and ocular diseases. Adverum has a robust pipeline that includes product candidates designed to treat rare diseases alpha-1 antitrypsin (A1AT) deficiency and hereditary angioedema (HAE) as well as wet age-related macular degeneration (wAMD). Leveraging a next-generation adeno-associated virus (AAV)-based directed evolution platform, Adverum generates product candidates designed to provide durable efficacy by inducing sustained expression of a therapeutic protein. Adverum has collaboration agreements with Regeneron Pharmaceuticals to research, develop, and commercialize gene therapy products for ophthalmic diseases and Editas Medicine to explore the delivery of genome editing medicines for the treatment of inherited retinal diseases. Adverum’s core capabilities include clinical development and in-house manufacturing expertise, specifically in process development and assay development. For more information please visit

Contact for Adverum:
Leone Patterson
Chief Financial Officer
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