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

The Gates Foundation supported another early-stage vaccination effort, delivering $ 2.1 million to a preclinical candidate from a pair of gene therapy pioneers who they think may offer benefits over the first wave of Covid-19 vaccines. now launched worldwide.

Luk Vandenberghe

The money will help develop a vaccine that Luk Vandenberghe and Jim Wilson, directors of gene therapy centers in Massachusetts Eye and Ear and the University of Pennsylvania, have been developing for the past year based on the AAV vectors commonly used in gene therapy. Now, in challenging studies with non-human primates, initial data suggests that the vaccine can function as a single dose and remain stable at room temperature, addressing two of the difficulties with the launch of the current generation of vaccines worldwide.

“When the data arrived that we had a number of attributes that could be beneficial from a global logistics deployment perspective, we contacted Gates,” said Vandenberghe News from Endpoints. These are “essential attributes for the distribution of these vaccines, particularly in areas that are more challenging from the point of view of infrastructure”.

It is clear that Vandenberghe is not the only vaccine developer trying to make a vaccine more scalable and distributable; Merck pursued similar goals with its two experimental vaccinations before ending both programs earlier this month.

However, there are still hundreds of vaccine candidates in initial development, and the Gates Foundation has supported some that it finds particularly promising. This includes a candidate for synthetic biology from the University of Washington who can also be a single, stable dose. And they also gave a $ 20 million grant to the Coalition for Epidemic Preparedness, which is selecting three to six “second generation” vaccines.

The Vandenberghe vaccine operates similarly to Covid-19 adenovirus-based vaccines developed by J&J and AstraZeneca, using a harmless virus to carry a gene for the coronavirus peak protein in human cells. Instead of adenovirus, however, they used a form of the common AAV vector in gene therapy.

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

The Gates Foundation dollars, however, will only lead to pre-clinical studies. Vandenberghe said they are looking for additional sponsors or pharmaceutical partners to help bring what has been largely an academic effort to the clinic in the coming 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 expansions of facilities on which other vaccines depended.

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 tied up in the middle of a pandemic.

“This is not in line with larger 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.”