Does gene therapy have the answer to vaccinating the world?


The COVID-19 pandemic is still raging around the world ... and the only weapon we have to wipe it out are vaccines

Does gene therapy have the answer to vaccinating the world?

We’ve invented the weapon. We’ve just not worked out how to make enough of them to win the war.

At current rates only half the world will be vaccinated by the end of the year. A partially vaccinated world is a dangerous place, for everyone.

The economic cost of locked-down economies and disrupted global supply chains is immense. The health and social implications of existing and new strains erupting, is catastrophic. The war is not won until people everywhere are vaccinated.

Yet, while Big Pharma and governments scramble to meet intense demand, innovative gene therapy manufacturers may have the answer. And not just for COVID-19, but for future pandemics, and many of the diseases that blight humanity.

Why aren’t we making vaccines faster?

Big Pharma has done an incredible job of developing a number of viable vaccines against COVID-19, in less than a year. A huge feat of scientific endeavour.

However, pharmaceutical companies are not set up for producing billions of doses in a few months. The world needs 10 billion doses from firms such as Johnson & Johnson or Oxford/AstraZeneca, to give two shots to 5 billion people — enough for global herd immunity.

At current levels, they’re on target to produce half this amount, this year.

The industry, and the world, has a fundamental question to answer. How do we make the vaccine faster? After all, a vaccine, no matter how effective, is only as good as the number of people vaccinated by it.

Viral vector-based vaccines are expensive to manufacture. With only 10-15% of new drugs getting approved by the FDA in the US, there’s no incentive to invest in manufacturing while still in clinical trials. This chronic under-investment in manufacturing makes it harder to ramp up production quickly, once it is approved.

It is more profitable for pharmaceutical companies to make the vaccine more slowly, using existing facilities, than build a new facility that would stand empty 6 months later.

This is not unique to vaccines. Viral vector-based gene therapies for cancers and diseases such as muscular dystrophy and arthritis, face similar obstacles.

In fact, the lack of availability of COVID-19 vaccines, is similar to the capacity issue for innovative treatments like cell and gene therapy.

In both cases it is simply a matter of production capacity. Pharma companies are struggling to produce the very large quantities of COVID-19 vaccines needed. On the other hand, because of inefficient production processes gene therapies are too expensive to be supported sustainably by our healthcare systems.

For instance, a single dose of the CAR-T therapy Yescarta can cost $373,000, whereas Zolgensma can be as much as $2.1 million, depending on the country or region. Yet, happily, the solution to solving both problems is the same.

A billion doses in a couple of months, from one facility?

Although COVID-19 has been ravaging the world, agile CDMOs (contract development and manufacturing organisations), such as Exothera, have been using highly advanced biopharmaceutical development and manufacturing programmes to solve a different problem - manufacturing viral vectors for cell and gene therapies.

The gene therapies are not designed to tackle COVID-19, but treat diseases such as cancer, infectious diseases, genetic disorders and cardiovascular and autoimmune diseases.

However, the process Exothera uses for developing gene therapies, makes vast volumes of viral vectors, at speed - just what the world needs to tackle the global vaccine shortfall.

By developing highly concentrated batches of viral vectors the company is able to ramp up yields while keeping volumes down. That means less space and less capex.

By bringing together data-driven processes that enhance performance and cut cost, with state-of-the-art manufacturing technology, high concentrations of viral vectors can be produced at warp speed.

Exothera is now turning its guns to making high concentrations of viral vectors for vaccines, as well. This can then be used for making millions of doses of vaccines using a fraction of the space of conventional vaccine production – thereby drastically reducing timelines.

“In cell and gene therapy there is no immediate need to produce millions of doses. But there is a need to produce very large doses per patient. So conceptually the challenge around production capacity is the same,” said Thibault Jonckheere, Deputy Chief Executive Officer of Exothera.

“That is what we are trying to solve with intelligent approaches to bioprocess development. By using state-of-the-art bioreactor technology, coupled with our bioprocess approaches, we estimate that with 5-10 bioreactors running in parallel, we could produce a billion doses in a few months, from a single facility.”

Beyond COVID

The innovative, optimised bioprocessing expertise of firms such as Exothera is not only of value to solving the immediate demand for more COVID vaccines. As COVID mutates, the need for further capacity will only continue.

COVID has woken up the world to the serious threat posed by pandemics and the need to be prepared. It has also made us more aware of global diseases and their impact on our economies, healthcare systems and way of life.

From cancers to rare diseases, the lives of millions of people could be transformed by effective gene therapies. Just as with COVID, the solutions lie in robust, agile and productive processes that tailor to the problem in front of them.

The sooner we address the need for fast, effective development and manufacture of viral vectors, the sooner we can meet head on, not just COVID, but some of the viruses and diseases that affect millions of people’s lives every day.

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So, as we have the world’s attention, maybe now is the time to start investing in future technologies and scientific endeavours that hold the solution to COVID and beyond.