Adverum Biotechnologies, a California-based gene therapy company focusing on the discovery and development of therapeutics for retinal and rare diseases, has delayed the initiation of its Phase 1/2 gene therapy trial for the treatment of alpha-1 antitrypsin (A1AT) deficiency by 12 months.
Adverum, formerly known as Avalanche Biotechnologies, has explained that this delay in the initiation of its ADVM-043 trial is due to its need to upgrade the manufacturing process and transfer it to a third-party contract manufacturer. Through this, the company aims to increase the production scale so that the same manufacturing process is used from clinical trials through to the commercial stage. Adverum expects to begin enrolment of patients in the fourth quarter of 2017.
The ADVM-043 trial is designed to deliver A1AT gene in patients with A1AT deficiency, a rare genetic disorder that results in serious respiratory and liver disease. The trial uses Adverum’s industrialized manufacturing process based on its baculovirus expression system and the company claims this next generation expression system as highly efficient and scalable, with production yielding up to one hundred times greater than those obtained using conventional adeno-associated virus based production systems.
The company’s other lead programs for wet age-related macular degeneration and hereditary angioedema are not affected and is in preparation to initiate toxicology studies in the first half of 2017.
Dr Amber Salzman, Adverum’s new CEO commented: “Adverum has industry-leading capabilities in process development and manufacturing, consisting of a baculovirus-based AAV production system and state-of the-art purification technology to manufacture AAV vectors of various serotypes. This allows us to deliver a turn-key large scale process to third-party cGMP manufacturers. We plan to leverage our leading manufacturing capabilities and upgrade our ADVM-043 manufacturing process now, to prepare for both our anticipated clinical and commercial product needs.”
Source: Adverum delays start of gene therapy PhI/II by one year, blames manufacturing. Reference