Novartis to make US researchers' COVID-19 gene therapy vaccine hopeful

Novartis re-entered vaccine making on Thursday, inking a manufacturing deal with a US team whose COVID-19 candidate relies on technology similar to that of the Swiss drugmaker's $2.1 million-per-patient gene therapy, Zolgensma.

Human trials are planned this year, said Massachusetts General Hospital and Massachusetts Eye and Ear, which are behind the early stage project, adding vaccine manufacturing at Novartis's AveXis gene therapy unit will start before Monday.

The U.S. researchers aim to insert genetic material that makes up the new coronavirus's "spikes" into a so-called adeno-associated virus (AAV), before injecting it into humans to provoke an immune response against COVID-19.

Zolgensma, developed by AveXis for deadly spinal muscular atrophy, works similarly, delivering a copy of a missing gene packed inside AAV to babies whose muscles would otherwise not develop.

While Novartis divested its vaccines business to GlaxoSmithKline in 2015, AveXis President David Lennon said his unit's gene therapy expertise made it a good fit to join the Massachusetts' effort, a major departure from traditional vaccine making.

"When we evaluated the science of it, we were really enthusiastic," Lennon told Reuters. "And we believe we're one of the few companies that actually could provide the product in the time frame...given the urgency of the COVID-19 pandemic."

AveXis will donate technology and supply chain resources to making vaccine for early trials, while retaining future production options, if all goes well, Lennon said.

"We are at the scale of being able to do millions of doses," he said. "Obviously, we can tap into production in other parts of the industry."

The effort will not sap any production capacity from Zolgensma, he added.

Harvard Medical School professor Luk Vandenberghe, who initiated this vaccine candidate in his lab at Massachusetts Eye and Ear, said AveXis's commercial gene therapy experience made it the logical partner.

“We are more on track than ever to reaching our goal of developing a vaccine capable for wide distribution," Vandenberghe said in a statement announcing the deal.