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 Harvard University scientist Luk Vandenberghe and University of
Pennsylvania gene therapy head James Wilson said primate safety and
efficacy tests have proven promising for their single-dose
candidate, targeted for room-temperature storage.
"We believe there is real potential," Vandenberghe said in an
interview. "That being said, we're not naive. There's 300 vaccines
racing towards the door and biology is complex."
Their candidate relies on an adeno-associated virus (AAV), deemed
harmless to humans, to deliver DNA fragments from the new
coronavirus, telling human cells to make a protein that provokes an
immune response.
AAVs are already used in gene therapies sold by Novartis, including
its $2.1 million-per-patient Zolgensma for spinal muscular atrophy.
The Swiss company's gene therapy unit is backing the project with
technical assistance and supply.
Other COVID-19 vaccines use viral vectors, too, though AstraZeneca
and Oxford University's shot, Russia's Sputnik V and Johnson &
Johnson's one-dose candidate rely on so-called adenoviruses to
transport coronavirus DNA.
Gates Foundation money will support more pre-clinical tests ahead of
a overseas safety trial in a still-undisclosed location where
vaccines are not broadly available, Vandenberghe, who runs Mass
General Brigham hospital's Grousbeck Gene Therapy Center in Boston,
said.
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 Though previously unused in
approved vaccines, he is optimistic AAVs will be
safe, since only tiny amounts are needed
compared to what is required for gene therapies
that have been given to many patients.
While he predicts every approved COVID-19
vaccine dose available through 2022 will see
use, Vandenberghe acknowledged his group's
success also hinges on finding a deep-pocketed
partner with vaccine experience to bankroll
large-scale production.
"We've already invested more than $10 million,"
he said. "If indeed we want to meet the time
lines we were just talking about, this needs a
commercial larger vaccine player to be
accelerated, like an AstraZeneca for Oxford's."
(Reporting by John Miller; Editing by Angus
MacSwan)
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