Gates Foundation supports second generation Covid-19 vaccine from gene therapy pioneers – Endpoints News

The Gates Foundation has supported another early-stage vaccination effort, donating up to $ 2.1 million to a preclinical candidate from a pair of gene therapy pioneers who they believe may offer benefits through compared to the first wave of Covid-19 vaccines currently being deployed around the world. .

The money will help advance a vaccine that Luk Vandenberghe and Jim Wilson, directors of the Massachusetts Eye and Ear and University of Pennsylvania Gene Therapy Centers, have developed over the past year based on the AAV vectors. commonly used in gene therapy. Now, in challenge studies in non-human primates, early data suggests that the vaccine can work in a single dose and remain stable at room temperature, which addresses two of the challenges of deploying the current generation of vaccines around the world. whole.

“When the data came in that we had a number of attributes that could be beneficial from a global logistics deployment perspective, we reached out to Gates,” Vandenberghe said. Endpoints News. These are “essential attributes for releasing these vaccines, especially in areas that are more difficult from an infrastructure perspective.”

Of course, Vandenberghe is not the only vaccine developer trying to make a more scalable and distributable vaccine; Merck was aiming for similar goals with its two experimental inoculations before shutting down both programs earlier this month.

However, hundreds of candidate vaccines are still in development, and the Gates Foundation is supporting a handful that it sees as particularly promising. This includes a synthetic biology candidate from the University of Washington who can also be single dose and shelf stable. And they also gave a $ 20 million grant to the Coalition for Epidemic Preparedness, which selects three to six “second generation” vaccines to support.

Vandenberghe’s vaccine works similarly to the adenovirus-based Covid-19 vaccines developed by J&J and AstraZeneca, using a harmless virus to transfer a gene for the coronavirus spike protein into human cells. Rather than an adenovirus, however, they used a form of the common AAV vector in gene therapy.

The variant of AAV they chose, Vandenberghe said, should express the gene inside cells for about 2 to 3 months – orders of magnitude shorter than what you would want for gene therapy, but also longer than mRNA or adenovirus vector vaccines. In theory, he said, it could produce a more potent and long-lasting immune response.

However, the Gates Foundation dollars will only push them through preclinical studies. Vandenberghe said they are looking for additional funders or pharmacy partners to help bring what has been largely an academic effort into the clinic over the next two months.

They are also looking to expand their manufacturing capacity. So far, they have relied on Novartis, which has AAV facilities to produce the SMA Zolgensma gene therapy. Since their vaccines require about 1 / 10,000 of the dose needed for gene therapy, Vandenberghe said they should be able to extract 100 million doses from current AAV facilities, even without the facility expansions. that other vaccines have relied on.

So far, he said, they have produced four batches of the vaccine – a small number compared to major developers, but significant for an academic effort in the midst of a pandemic.

“It doesn’t correspond to bigger funds and we are certainly not the pioneers here,” said Vandenberghe. “The goal is to address some of the shortcomings of the first wave of vaccines.”

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