12 Sep 2016
Lonza to Offer Novel Anc-AAV Gene Therapy Technology Through Exclusive Licensing Agreement with Massachusetts Eye and Ear ®
  • The strategic licensing agreement between Lonza and Massachusetts Eye and Ear® underscores Lonza's position as a leading AAV manufacturing service provider and offers customers unprecedented technology and services to commercialize next-generation gene therapies.
  • The Anc-AAV vector platform has the potential to overcome pre-existing immunity in order to treat more patients compared with other viral vectors currently in development.
  • Anc80, the lead novel Anc-AAV available for sublicensing, is a potent gene therapy vector capable of superior gene expression levels in retina, liver, muscle and other tissue targets.

Basel (CH) and Boston, MA (USA), 12 September 2016 – Lonza Houston, Inc., a global leader in viral gene and cell therapy manufacturing, and Massachusetts Eye and Ear®, the world's largest vision and hearing research center, have entered into a strategic agreement that provides customers the ability to in-license Anc80 and other Anc-AAVs for the clinical development and commercialization of novel gene therapies. The agreement is designed to accelerate gene therapy drug development across the field in order to address diseases in need of treatments and thereby ultimately reach more patients.

Anc-AAVs are in silico designed synthetic adeno-associated viral vectors (AAVs), developed first in the laboratory of Dr. Luk H. Vandenberghe, Assistant Professor at Harvard Medical School, and Director of the Grousbeck Gene Therapy Center at Massachusetts Eye and Ear®. As part of the agreement, an innovative platform development effort will be initiated with a focus on discovering additional next generation Anc-AAVs.

“In this era of personalized medicine, the partnership with Lonza is unique, and potentially very effective,” Dr. Vandenberghe said, “as it brings a highly potent vector technology under one roof with a leading manufacturer of biologics. We believe this concept will bring innovative gene therapies to patients in a more efficient and expedient way, and that it will increase access to enabling gene therapy technology to unlock treatment for diseases of unmet need, including those affecting vision and hearing.”

By applying its AAV manufacturing technology innovation, Lonza agrees to work towards the establishment of modern best-in-class large-scale manufacturing platforms for Anc80 and any future vectors generated out of Dr. Vandenberghe's lab. The agreement comes only months after Lonza broke ground in April 2016 to construct a state-of-the-art viral gene and cell therapy manufacturing facility in Pearland, TX (USA). The new facility, expected to come online at the end of 2017, is anticipated to be one of the largest facilities in the world for the supply of clinical and commercial grade viral-based gene therapies.

Under the agreement Lonza will fund research at the Grousbeck Gene Therapy Center at Massachusetts Eye and Ear® to discover, characterize and develop next-generation gene transfer reagents in order to improve upon important limitations of current AAVs, including pre-existing immunity, manufacturing yields, immunogenicity, tissue tropism, and specificity.

“This strategic licensing deal with Massachusetts Eye and Ear® emphasizes Lonza's strong commitment to the field of gene therapy and our continuous quest for improving patients' lives,” stated Marc Funk, COO, Lonza's Pharma&Biotech segment. “Drawing on our licensing expertise, we will be able to leverage our experience as the leading global AAV manufacturer in our broad range of service offerings.”

As part of the agreement, Massachusetts Eye and Ear® grants Lonza the exclusive position to commercially license Anc-AAV while it retains certain commercial and academic rights, including the ability to commercialize self-directed gene therapy programs and all rights in the challenging space of ultra-rare diseases.