Lonza to Offer Novel Anc-AAV Gene Therapy Technology Through Exclusive Licensing Agreement with Massachusetts Eye and Ear

September 12, 2016
Luk Vandenberghe
  • 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, Switzerland and Boston, Mass. —  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 laboratory. 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 global leading 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.

Information in this release was provided by Lonza Houston, Inc

About Lonza
Lonza is one of the world’s leading and most-trusted suppliers to the pharmaceutical, biotech and specialty ingredients markets. We harness science and technology to create products that support safer and healthier living and that enhance the overall quality of life.

Not only are we a custom manufacturer and developer, Lonza also offers services and products ranging from active pharmaceutical ingredients and stem-cell therapies to drinking water sanitizers, from the vitamin B3 compounds and organic personal care ingredients to agricultural products, and from industrial preservatives to microbial control solutions that combat dangerous viruses, bacteria and other pathogens.

Founded in 1897 in the Swiss Alps, Lonza today is a well-respected global company with more than 40 major manufacturing and R&D facilities and approximately 9,800 full-time employees worldwide. The company generated sales of about CHF 3.8 billion in 2015 and is organized into two market-focused segments: Pharma&Biotech and Specialty Ingredients. Further information can be found at .

Lonza Contacts
Dirk Oehlers, Head Investor Relations
+41 61-316-8540

Dominik Werner, Head Corporate Communications
+41 61-316-8798

Constance Ward, Head External Communications
+41 61-316-8840

About Massachusetts Eye and Ear
Mass. Eye and Ear clinicians and scientists are driven by a mission to find cures for blindness, deafness and diseases of the head and neck. Now united with Schepens Eye Research Institute, Mass. Eye and Ear is the world's largest vision and hearing research center, developing new treatments and cures through discovery and innovation. Mass. Eye and Ear is a Harvard Medical School teaching hospital and trains future medical leaders in ophthalmology and otolaryngology, through residency as well as clinical and research fellowships.  Internationally acclaimed since its founding in 1824, Mass. Eye and Ear employs full-time, board-certified physicians who offer high-quality and affordable specialty care that ranges from the routine to the very complex. In the 2016–2017 “Best Hospitals Survey,” U.S. News & World Report ranked Mass. Eye and Ear #1 in the nation for ear, nose and throat care and #1 in New England for eye care. For more information about life-changing care and research, or to learn how you can help, please visit .

About AAV and Anc-AAV technology
Adeno-associated virus (AAV) vectors have gained traction in gene therapy due to their lack of pathogenicity and the predictability of gene transfer. In the clinic, these vectors have shown the ability to offer gene transfer for treatments of diseases like spinal muscular atrophy, hemophilia, and inherited forms of blindness.

Anc-AAV technology, developed in the Vandenberghe laboratory, uses computational and evolutionary methods to predict novel conformations of the adeno-associated viral particle. Anc-AAVs share several of the features of naturally occurring AAVs and their variants. However, Anc-AAVs are fully man-made and do not occur in nature.

Evidenced by Anc80, this unique synthetic biology and computational design approach allows for immediate use in therapeutic programs. Furthermore, it provides unprecedented engineering opportunities to overcome deficiencies of current technologies to address diseases of unmet need for broader patient populations. Anc80, a putative ancestor of AAV1, 2, 3, 6, 7, 8, rh.10, and AAV9, was designed to retain key desirable aspects for clinical translation, while distinguishing itself antigenically from circulating AAVs. Anc80 was shown in mice and non-human primates to be a safe and potent therapeutic gene delivery vector in preclinical studies and previously licensed to Selecta Biosciences for specific indications, including methylmalonic acidemia.

About Grousbeck Gene Therapy Center and Ocular Genomics Institute
Researchers in the Grousbeck Gene Therapy Center at Massachusetts Eye and Ear pursue the vision of genetic medicine in ophthalmology and beyond. Founded by Massachusetts Eye and Ear® and the Harvard Medical School Department of Ophthalmology in 2014 through a generous donation by the Grousbeck Family, the Center is an integral part of the Ocular Genomics Institute, with a mission to improve our understanding of the genetic basis of eye diseases in order to unlock novel treatment modalities. Led by Luk H. Vandenberghe, Ph.D., research in the Center is directed toward studying the biology of somatic gene transfer, the development of enabling gene transfer technology, and translation of gene therapies to the clinic. Dr. Vandenberghe is a co-inventor on several AAV vector technologies and methods, including Anc80 which are licensed to biotech and pharmaceutical entities for which he receives royalties.

Media Contact:
Suzanne Day
Media Relations, Mass. Eye and Ear