AAVCOVID Vaccine Program from Mass. Eye and Ear and Mass General Enters Manufacturing Agreement with Gene Therapy Leader AveXis, a Novartis Company

AveXis, a Novartis Company, agrees to contribute the use of its gene therapy manufacturing expertise to help produce and scale novel vaccine that uses adeno-associated virus (AAV) vector for clinical trials

Boston, Mass. — The AAVCOVID vaccine program Massachusetts Eye and Ear and Massachusetts General Hospital, members of Mass General Brigham, has entered into a manufacturing agreement to produce its novel genetic vaccine with an industry leader in gene therapy, AveXis, a Novartis Company. AveXis will begin manufacturing the vaccine this month while AAVCOVID undergoes further safety and efficacy testing in preclinical studies taking place at academic medical institutions including Mass. Eye and Ear.

AveXis, will lead the manufacturing efforts of the new vaccine, utilizing its cutting-edge AAV technology for treatment of rare and life-threatening neurological genetic diseases. AveXis is one of the first companies in the world to have successfully scaled up gene therapy manufacturing with over 1 million square feet of manufacturing capacity.

Through this strategic partnership, AveXis is contributing its technology, expertise and supply chain at no cost to supply the AAV vaccine for COVID clinical trials scheduled to begin in the second half of 2020.

“The COVID-19 pandemic is the most urgent public health crisis of our time and we recognize the significance of evaluating the potential role of a gene-based vaccine,” said Dave Lennon, president of AveXis. “As one of the world’s leading gene therapy companies, we are pleased to lend our unparalleled manufacturing expertise, technology and supply chain to produce a COVID vaccine for use in clinical trials. Having developed and produced one of just two, FDA-approved AAV gene therapies, we are uniquely poised to help the team move quickly toward this accelerated effort.”

The AAVCOVID vaccine program was developed in the laboratory of Luk H. Vandenberghe, PhD, director of the Grousbeck Gene Therapy Center at Massachusetts Eye and Ear and Associate Professor of Ophthalmology at Harvard Medical School. The experimental vaccine uses an AAV vector to deliver the genetic code to produce protein fragments of the SARS-CoV-2 virus, the cause of COVID-19 disease, to elicit an immune response. Mason Freeman, MD, director and founder of the MGH Translational Research Center and a Professor of Medicine at Harvard Medical School, is leading the efforts to develop the clinical studies intended to establish safety and efficacy of the experimental vaccine. Once these early studies are completed, including the first clinical trials, the researchers aim to advance the study into later phases. AveXis has the option to manufacture the AAV vaccine for additional clinical development, registration and/or commercial activities.

Mass. Eye and Ear and Mass General are member hospitals of Mass General Brigham.

“There are many vaccine approaches to COVID-19 with great promise, but often the first question that gets asked is, ‘how can this vaccine be scaled to the millions of doses needed to prevent a worldwide pandemic?’” said Dr. Vandenberghe. “By partnering with an industry leader in AveXis that already produces AAV gene therapy products at large scales, we are more on track than ever to reaching our goal of developing a vaccine capable for wide distribution to prevent infection at population levels.”

About AAVCOVID Vaccine Program

The AAVCOVID vaccine program is a gene-based vaccine strategy that seeks to deliver genetic sequences of the SARS-CoV-2 using an AAV vector. Vaccination delivers genetic DNA fragments SARS-CoV-2 which generates an antigen protein, which is designed to elicit an immune response to prevent infection. This approach is supported by extensive experience with the safety of the AAV technology platform in other diseases, including the use of AAVs in two FDA-approved medications.

AAVCOVID is an academia-industry collaboration led by Dr. Vandenberghe, the Grousbeck Family Chair in Gene Therapy at Mass. Eye and Ear, who is a world-renowned leader and pioneer of viral gene transfer and therapeutic gene transfer. Dr. Vandenberghe is working in conjunction with a clinical partner in Dr. Freeman, who serves as Director of the Translational Medicine Group of the MGH Center for Computational and Integrative Biology and is Professor of Medicine at Harvard Medical School.

Dr. Vandenberghe and his laboratory began work on the vaccine in mid-January following the Wuhan outbreak and the first publication of genetic sequences of the new coronavirus. Using a specific AAV with desirable vaccine properties, the program seeks to induce immunity to prevent infection and or disease in healthy populations, leveraging the existing manufacturing capabilities of the AAV industry.

AAV is also a rapidly adaptable technology. If a new strain of the SARS-CoV-2 virus emerges, the genetic code inside the AAVCOVID vaccine can be exchanged for an updated genetic code and processed into an updated vaccine in weeks, according to the researchers.

About Massachusetts Eye and Ear

Massachusetts Eye and Ear, founded in 1824, is an international center for treatment and research and a teaching hospital of Harvard Medical School. A member of Mass General Brigham, Mass. Eye and Ear specializes in ophthalmology (eye care) and otolaryngology–head and neck surgery (ear, nose and throat care). Mass. Eye and Ear clinicians provide care ranging from the routine to the very complex. Also home to the world's largest community of hearing and vision researchers, Mass. Eye and Ear scientists are driven by a mission to discover the basic biology underlying conditions affecting the eyes, ears, nose, throat, head and neck and to develop new treatments and cures. In the 2019–2020 “Best Hospitals Survey,” U.S. News & World Report ranked Mass. Eye and Ear #4 in the nation for eye care and #2 for ear, nose and throat care. For more information about life-changing care and research at Mass. Eye and Ear, visit our blog, Focus, and follow us on Instagram, Twitter and Facebook.

About Massachusetts General Hospital

Massachusetts General Hospital, founded in 1811, is the original and largest teaching hospital of Harvard Medical School. The MGH Research Institute conducts the largest hospital-based research program in the nation, with an annual research budget of more than $1 billion and comprises more than 8,500 researchers working across more than 30 institutes, centers and departments. In August 2019 the MGH was once again named #2 in the nation by U.S. News & World Report in its list of "America’s Best Hospitals."

About Harvard Medical School Department of Ophthalmology

The Harvard Medical School Department of Ophthalmology is one of the leading and largest academic departments of ophthalmology in the nation. Composed of nine affiliates (Massachusetts Eye and Ear, which is home to Schepens Eye Research Institute; Massachusetts General Hospital; Brigham and Women’s Hospital; Boston Children’s Hospital; Beth Israel Deaconess Medical Center; Joslin Diabetes Center/Beetham Eye Institute; Veterans Affairs Boston Healthcare System; Veterans Affairs Maine Healthcare System; and Cambridge Health Alliance) and several international partners, the department draws upon the resources of a global team to pursue a singular goal—eradicate blinding diseases so that all children born today will see throughout their lifetimes. Formally established in 1871, the department is committed to its three-fold mission of providing premier clinical care, conducting transformational research, and providing world-class training for tomorrow’s leaders in ophthalmology.

About AveXis

AveXis, a Novartis company, is the world’s leading gene therapy company, redefining the possibilities for patients and families affected by life-threatening genetic diseases through our innovative gene therapy platform. Founded in 2013 and headquartered in Bannockburn, IL, the goal of AveXis’ cutting-edge science is to address the underlying, genetic root cause of diseases. AveXis pioneered foundational research, establishing AAV9 as an ideal vector for gene transfer in diseases affecting the central nervous system, laying the groundwork to build a best-in-class, transformational gene therapy pipeline. The company developed the only gene therapy for spinal muscular atrophy (SMA) which has been approved for use in the U.S., Japan and Europe. AveXis is also developing therapies for other genetic diseases, including Rett syndrome, a genetic form of amyotrophic lateral sclerosis (ALS) SOD1 and Friedreich’s ataxia. For additional information, please visit avexis.com.