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By John Miller
ZURICH, Jan 29 (Reuters) - Scientists who normally focus on
fixing defective genes said on Friday that up to $2.1 million
from the Bill & Melinda Gates Foundation will help them move
their COVID-19 vaccine candidate toward 2021 human trials.
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.
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)
