Mass Eye & Ear & MGH Select Novartis’ AveXis as Manufacturer for Gene Therapy-based COVID-19 Vaccine in Preclinical Stage

Mass Eye & Ear & MGH Select Novartis’ AveXis as Manufacturer for Gene Therapy-based COVID-19 Vaccine in Preclinical Stage

The AAVCOVID vaccine program at Massachusetts Eye and Ear and Massachusetts General Hospital, member of Mass General Bingham, has entered into a manufacturing agreement to produce its novel genetic vaccine with an industry leader in gene therapy, AveXis, a Novartis Company. Known as a pathbreaker for its novel therapy with a $2 million price tag, AveXis will commence manufacturing the vaccine this month while AAVCOVID undergoes further safety and efficacy testing in preclinical studies ongoing at academic medical institutions including Mass. Eye and Ear.

No Cost for Key Clinical Trials Resources

In this arrangement, AveXis has been recruited to 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.

Moreover, in what some might consider a surprising move, AveXis has committed to contribute its technology, expertise and supply chain at no cost to supply the AAV vaccine for COVID-19 clinical trials scheduled to begin in the second half of 2020.

AAVCOVID Vaccine Program Background

This 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. A gene-based vaccine approach, the investigational 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, to elicit an immune response.

Clinical Trials Leadership

The effort to develop clinical trials intended to establish safety and efficacy of the experimental vaccine is led by Mason Freeman, MD, director and founder of the MGH Translational Research Center—he is also a Professor of Medicine at Harvard Medical School. One of the early studies are completed, including the first clinical trials, reports Mass Eye and Ear in the recent press release. Now the researchers aim to advance the study into larger phases.

Options for AveXis

By entering into this arrangement, AveXis negotiated the option to manufacture the AAV vaccine for additional clinical development, registration and/or commercial activities.

What key elements does Novartis’ AveXis bring to the table?

Dr. Vandenberghe commented on the fact that there are many promising COVID-19 focused vaccine candidates but the question around scalability is a concern and hence this Boston-based group considered the AveXis deal: “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 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 of 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.

About Mass Eye and  Ear

Mass Eye and Ear is an international center for treatment and home to the world’s largest vision and hearing research centers. It is also a teaching hospital of Harvard Medical School.  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. With a main campus in Boston, they also have 20 satellite locations.

Lead Research/Investigator

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