Scientists who normally focus on repairing defective genes said Friday that up to $ 2.1 million from the Bill & Melinda Gates Foundation will help them get their COVID-19 vaccine candidate up to 2021 human trials .
Harvard University scientist Luk Vandenberghe and the head of gene therapy at the University of Pennsylvania James Wilson said the primate safety and efficacy tests have shown promise for their single-dose candidate, the one for storage at room temperature is provided.
“We believe there is real potential,” Vandenberghe said in an interview. “That being said, we’re not naive. 300 vaccines are racing toward the door and the biology is complex.”
Her candidate relies on an adeno-associated virus (AAV), believed to be harmless to humans, to deliver fragments of DNA from the new coronavirus and asks human cells to make a protein that triggers an immune response.
AAVs are already used in gene therapies sold by Novartis, including the Zolgensma for $ 2.1 million per patient for spinal muscular atrophy. The gene therapy unit of the Swiss company supports the project with technical support and supplies.
Other COVID-19 vaccines also use viral vectors, although the AstraZeneca and Oxford University shot, Russian Sputnik V, and Johnson & Johnson’s one-dose candidate rely on what is known as adenoviruses to carry coronavirus DNA.
The Gates Foundation money will support further pre-clinical testing ahead of an overseas safety study in an as-yet-unknown location where vaccines are not widely available, said Vandenberghe, who heads the Grousbeck gene therapy center at Mass General Brigham Hospital in Boston.
Although not previously used in approved vaccines, he is optimistic that AAVs will be safe as they require small amounts compared to gene therapies that have been given to many patients.
While predicting that every approved dose of COVID-19 vaccine available by 2022 will be used, Vandenberghe acknowledged that his group’s success also depends on finding a partner with vaccine experience to help scale production Finance scale.
“We have already invested more than $ 10 million,” he said. “If we really want to meet the schedules we just talked about, a commercial bigger vaccine player like an AstraZeneca for Oxford has to be accelerated.”
(Except for the headline, this story was not edited by GossipMantri staff and posted from a syndicated feed.)