- Novartis' AveXis division will manufacture a coronavirus vaccine being developed by Massachusetts Eye and Ear Hospital, allowing the academic institution to take advantage of the technology that has delivered the gene therapy Zolgensma to spinal muscular atrophy patients.
- AveXis will begin manufacturing the vaccine this month as researchers conduct preclinical testing. The hospital expects to begin a Phase 1 trial in humans in the second half of 2020.
- Massachusetts Eye and Ear's project joins more than 100 others in a race to launch a vaccine to prevent serious disease resulting from infections of the SARS-CoV-2 virus. Manufacturing is a key question for the biopharma sector, as hundreds of millions of doses will be necessary to keep infection rates under control.
The hospital's project, called AAVCOVID, stems from work at its Grousbeck Gene Therapy Center. The center's co-director, Luk Vandenberghe, helped found French gene therapy company GenSight Biologics and advise Nightstar Therapeutics before its buyout by Biogen.
Both companies favor a common gene therapy delivery tool, a type of adeno-associated virus (AAV), to shuttle genetic instructions into the body. Likewise, the new AAVCOVID project uses an AAV to deliver a gene that will stimulate production of an antibody to SARS-CoV-2's signature "spike" protein. Partnering with AveXis, Novartis's gene therapy unit, appears to be a natural fit, as Zolgensma relies on AAV technology as well.
The Translational Research Center at partner Massachusetts General Hospital is preparing the clinical trials that will be necessary to test the vaccine in humans. However, the deal with AveXis means that the institutions are already thinking ahead to an eventual launch, as AveXis will have an option to manufacture the vaccine for advanced clinical trials and commercial distribution.
"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," Vandenberghe said in a statement.
The hospital did not say how many doses AveXis has the capacity to manufacture. Zolgensma treats a patient population that numbers in the hundreds per year in the U.S., but patients must receive massive doses: Infants and toddlers with SMA receiving a systemic infusion must receive about 100 trillion vector genomes per kilogram of body weight.
AAVCOVID joins at least 30 other viral vector vaccines now in development, according to the Milken Institute coronavirus vaccine and treatment tracker. The leading one has already generated early clinical data: CanSino published results from a trial of its AAV5-based vaccine in the Lancet last week.