Adverum Biotechnologies Announces Completion of Dosing of First Cohort of Patients in the ADVANCE Phase 1/2 Clinical Trial of ADVM-043 Gene Therapy for A1AT Deficiency
-- Data Monitoring Committee Recommends Proceeding to the Second Cohort --
-- Company Expects to Report Preliminary Data in the Second Half of 2018 --
“We are excited to advance to the second cohort of patients with A1AT deficiency in this clinical trial,” said
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 (n=2) received an intravenous (IV) low dose of ADVM-043 of ~1E12 vg/kg (8E13 total vg). The second cohort will receive an IV intermediate dose of ~5E12 vg/kg (4E14 total vg) and the third cohort will receive an IV high dose of ~1.5E13 vg/kg (1.2E15 total vg). A potential fourth cohort may be opened to evaluate an intrapleural (IP) delivery of ADVM-043.
The study will be conducted at 5 leading centers in
Additional information about this clinical trial can be found at ClinicalTrials.gov under trial identifier number NCT02168686.
ADVM-043 (AAVrh.10-A1AT) is a gene therapy candidate designed as a potential single-administration treatment to induce stable, long-term A1AT protein expression. In a preclinical proof-of-concept study, ADVM-043 demonstrated robust protein expression above therapeutic levels in mice following either intravenous (IV) or intrapleural (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 an orphan disease impacting approximately 100,000 individuals in
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
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Source: Adverum Biotechnologies, Inc.