Adverum Biotechnologies delays gene therapy trial
Adverum Biotechnologies has provided an update on its development program for ADVM-043 (formerly ANN-001), a novel gene therapy for Alpha-1 Antitrypsin (A1AT) Deficiency, a rare genetic disorder that may result in serious respiratory and liver disease.
Adverum has decided to upgrade the ADVM-043 manufacturing process by implementing its proprietary baculovirus-based production system and plans to transfer the third-party contract manufacturing for ADVM-043 to a large-scale contract manufacturer. This upgrade is designed to increase the production scale, and comply with industry standards so that the same production process is used from clinical trials through commercial stage. The company now expects to begin enrolment of patients in the phase I/II clinical trial for ADVM-043 in the fourth quarter of 2017.
“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,” said Amber Salzman, Ph.D., chief executive officer of Adverum Biotechnologies. “Our goal is to meet with the FDA to review our modified plans in the first quarter of 2017, to allow us to move ahead with patient enrolment in our planned phase I/II clinical trial for ADVM-043 in the fourth quarter of 2017. We are committed to advancing this novel gene therapy for patients with A1AT deficiency, and are planning to be ready to deliver this potential product through a robust commercial-ready manufacturing process.”
The company’s other lead programs for wet age-related macular degeneration (wAMD) and hereditary angioedema (HAE) remain on target to initiate toxicology studies in the first half of 2017.