NVS Novartis AG

By Denise Roland 

Novartis AG has agreed to manufacture a gene-based coronavirus vaccine being developed by scientists at Massachusetts Eye and Ear hospital, Massachusetts General and the University of Pennsylvania, paving the way for human testing to begin later this year.

The Swiss drugmaker's gene therapy unit AveXis is already making test batches of the vaccine and plans to start producing doses later in the summer that can be used for a clinical trial, said Dave Lennon, the unit's president.

If testing goes smoothly, the vaccine could be made available on an emergency basis by the end of the year, according to Mason Freeman, director and founder of the Massachusetts General Hospital Translational Research Center, and one of the lead researchers.

Novartis, one of the world's biggest pharmaceutical companies by sales, had been a notable holdout as other large rivals announced coronavirus-vaccine efforts. Most recently, Merck & Co. said it was pursuing two candidates.

No vaccine has been proven effective against Covid-19, the illness caused by the novel coronavirus. Ten are in human testing and more than 100 are in early studies, according to the World Health Organization.

The vaccine that AveXis has agreed to make would work like a gene therapy in that it uses an inactive virus to deliver DNA into the body, the vaccine's researchers said.

The DNA would teach cells how to make the "spike protein" found on the surface of the new coronavirus, the researchers said. The hope is that cells in the body making this protein will elicit an immune response to the virus that will protect against it.

There aren't any approved vaccines against any illness that work like a gene therapy, but the researchers said they hope that using a proven DNA delivery method will give their candidate a good shot. Most of the handful of approved gene therapies use viruses from the same family.

"We're playing basketball on a court where we don't know where the hoop is," said Luk Vandenberghe, director of the Grousbeck Gene Therapy Center at Massachusetts Eye and Ear and one of the lead researchers. "We have to shoot in as many directions as possible, and at least we have a different shot and angle."

The inactivated virus used by the vaccine isn't found in humans, meaning the immune system is unlikely to destroy it before it can deliver the genetic material to cells, the researchers said. That is a potential pitfall for vaccines based on inactivated viruses.

Viralgen, a gene therapy manufacturer based in Spain, also agreed to make a supply of the vaccine candidate for use in clinical trials.

AveXis makes Zolgensma, one of the few gene therapies currently available in the U.S.

The Novartis unit has so far committed to manufacture doses for animal studies and a phase 1 trial, free of charge, Dr. Lennon said. The phase 1 trial will likely involve 50 to 60 people, the researchers said. It has the option to manufacture larger quantities of vaccine for a larger human study and for eventual sale should the vaccine progress to those stages.

Larger-scale manufacture would likely require financing, Dr. Lennon said. Right now AveXis has the capacity to churn out only a few million doses of vaccine a year.

Many companies developing vaccines against Covid-19 are seeking support from governments and other funders to ramp up production of their candidates.

Other large drugmakers pursuing coronavirus vaccines include Sanofi SA., GlaxoSmithKline PLC, Pfizer Inc., Johnson & Johnson, Merck & Co. and AstraZeneca PLC.

Among the most advanced candidates are vaccines from Boston-area biotech Moderna Inc. and China's CanSino Biologics Inc.

Write to Denise Roland at Denise.Roland@wsj.com

(END) Dow Jones Newswires

May 28, 2020 18:10 ET (22:10 GMT)

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