Ginkgo has hired a new head of Biopharma to revolutionize the gene and cell therapy market

Cell and gene therapies could be the most awaited invention of this century. For many people, these breakthroughs can’t come fast enough: cell therapies could bring hope to people with rapidly progressing cancer, cure genetic diseases like sickle cell anemia, and potentially even treat common conditions like heart disease and diabetes. However, progress is slow and the cost of a single treatment can be as high as $3.5 million, making it inaccessible to most patients. I spoke to Behzad Mahdavi, the new Head of Biopharma Manufacturing & Life Sciences Tools at Ginkgo Bioworks, who will be speaking at SynBioBeta 2023, the Global Synthetic Biology Conference, to understand what needs to be done to make these develop more affordable life-saving therapies.

The traditional model of pharmaceutical drug development is partly responsible for the high price. The industry is highly regulated, so implementing changes takes a long time and requires high upfront investments. Innovation can save money in the long run, but adopting new technologies comes with some risk and it’s hard to say how much cost benefit they can offer. For this reason, most pharmaceutical companies focus on process and operational improvements that can increase efficiency by 10-20%. But making cell and gene therapy treatments more affordable will require leaps and bounds on the biological side to bring the price down by orders of magnitude: “Improvements on the manufacturing side bring only marginal gains,” says Mahdavi. “We need to go back to biology and better understand how it works. Synthetic biology is helping us with this.”

Ginkgo Bioworks has made a name for itself as a synthetic biology platform that helps its customers develop bio-based products using artificial organisms. Ginkgo has partnered with companies covering a wide range of markets from flavors and fragrances to food coloring, cultivated cannabinoids, skin care ingredients, materials and more. But the synthetic biology company doesn’t want to limit itself to ingredients. Ginkgo has expanded its platform into the broad field of biotherapeutics, hence Mahdavi’s appointment. Mahdavi comes to Ginkgo with an impressive track record of commercializing novel therapies. Most recently, he was VP of Global Open Innovation at Catalent Pharma Solutions for almost 14 years and before that VP of Strategic Innovation at Swiss manufacturer Lonza. Now Mahdavi sees a lot of potential to revolutionize the industry by connecting biopharma and synthetic biology. “Pharma has an established model – and it’s good at what it does. But the real improvements have to be made on the biological side,” says Mahdavi. “This requires a better understanding of the biology and higher throughput methods for cell engineering.”

Ginkgo’s platform is a complete solution for therapeutic cell engineering. Their multi-story labs—called “foundries”—are Ginkgo’s biological factories, outfitted with robotics and state-of-the-art instrumentation that enable high-throughput cell engineering. This means Ginkgo can examine hundreds of thousands of cells and molecular compounds to identify the most promising drug candidates Also least expensive to produce on a large scale. For example, in its partnership with Aldevron, Ginkgo increased its performance by a factor of 10 through cell engineering and biological process improvement. “That is, if you want to build a plant [for that product]you can build a facility that is 10 times smaller just through improvements in biology,” says Mahdavi.

Ginkgo has already had initial successes with its platform. During the COVID-19 pandemic, Ginkgo launched a public health initiative, Concentric by Ginkgo, which provided testing services to track emerging infection hotspots. On the vaccine development side, Ginkgo worked with Moderna to quickly find optimal processes for mRNA vaccine production. In addition to COVID-19, Ginkgo recently announced other biopharma partnerships with Optiva, Selecta and Merck, as well as the acquisition of Circularis, a proprietary RNA screening platform.

A major focus of Mahdavi’s work is in the potentially curative (and hugely lucrative) area of ​​cell and gene therapies. One of the first cell therapies to be approved by the FDA was CAR-T cell therapies for leukemia, lymphoma, and myeloma. In this groundbreaking treatment, the patient’s immune cells are taken from their own blood and modified in the laboratory by adding a receptor that can attack and destroy cancer cells. This requires precise genetic modifications to be made to living cells, which is not that easy. Because of this, CAR-T treatments cost between $700,000 and $1 million on average, and most of the cost today comes from manufacturing these modified CAR-T cells. We have come a long way in our ability to engineer cells. But the cellular architecture is still a black box, which makes building these new modalities so challenging.

There are currently over 1,000 cell and gene therapy clinical trials registered with ClinicalTrials.gov, but only 14 have been approved to date. The majority of these trials are still in phase 1 or 2, meaning they are still a long way from commercialization. Given the low success rate of the FDA’s approval pipeline, these potential cures will likely never make it to market. That is why it is so important for the development of successful therapies to invest in early stages in order to discover the best possible targets: “We focus on the optimal production, but not on the production of the most optimal drug,” says Mahdavi. Synthetic biology tools and workflows are designed to address the complexity of biology and increase the chances of success in developing effective cell-based therapies.

Significant advances have been made in high-throughput biology engineering tools that could reduce the cost and timescale for developing new cell and gene therapies. Ginkgo has shown that they can build around 10,000 CAR T cell libraries and see which are the most promising candidates. For example, they found a subset of cells that are much more resistant to fatigue that could potentially be used to develop treatments for solid tumors. Leveraging high-throughput cell engineering combined with bioinformatics can improve the number of shots on goal for each new target: “Ten years ago, saying ‘I’m going to look at 10,000 CAR-T constructs’ was a dream, but today it is no obstacle to even look at 1 million constructs,” said Mahdavi.

For biopharmaceutical companies, this type of high-throughput scaling is much more difficult to achieve without investing heavily in the latest technologies to upgrade their entire R&D infrastructure. Because of this, partnering with synthetic biology companies that have the specific expertise in this field could bring significant savings to biopharmaceuticals. “Ultimately, it’s about getting the optimal product and manufacturing conditions in the shortest amount of time – and that’s what ginkgo is bringing to biopharma,” said Behzad. He envisions that synthetic biology could disrupt biopharma’s drug development pipeline in a systematic way and help bring life-saving therapies to people much faster.

Many thanks to Katia Tarasava for further research and reports on this article. I am the founder of SynBioBeta and several of the companies I write about, including Ginkgo Bioworks, are sponsors of SynBioBeta SynBioBeta Conference and weekly summary.

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