Synthetic biology: Innovating in drug discovery

Genetic research

Synthetic biology is increasingly important to drug discovery and development. Reece Armstrong examines recent activity within the sector and highlights some of the key advancements and innovation.


Earlier this year cell-engineering company MaxCyte signed a strategic platform license (SPL) with biotechnology company Catamaran Bio.

The agreement will see Catamaran gain access to MaxCyte’s technology platforms which enable the complex engineering of cells. In return MaxCyte will receive platform licensing fees and program-related revenue.

The deal makes sense given Catamaran’s focus on using synthetic biology and non-viral cell engineering to develop highly potent allogeneic, cryopreserved CAR-NK cell therapies that target challenging cancers, including solid tumours. The company is combining new functional attributes with the cancer-fighting properties of natural killer cells.

To expand and process NK cells into safe and effective cell therapies, Catamaran has developed an integrated suite of technologies that specifically address the end-to-end methods of engineering, processing and manufacturing.

Speaking about the partnership, Doug Doerfler, President and CEO of MaxCyte says: “The ability to leverage synthetic biology to engineer cell therapies will help drive innovative solutions for solid tumour cancer treatment. At MaxCyte, we are passionate about driving the discovery, development and manufacturing of next-generation, cell-based medicines to tackle complex diseases and cancer. We are excited to support Catamaran with the advancement of their pipeline of differentiated CAR-NK cell therapies into and through the clinic.”

Meanwhile, synthetic biology company Evonetix completed a $24 million financing round to help it continue the development of its semiconductor chips. Evonetix will use the funding to brings its semiconductor chips to commercial scale and extend gene assembly capabilities for its binary assembly technology to deliver accurate, gene-length DNA in a benchtop instrument. Evonetix hopes the platform can change the way DNA is prepared and delivered to users within synthetic biology and helps accelerate research across life sciences.

Colin McCracken, Chief Executive Officer at Evonetix, comments: “This substantial investment round demonstrates continued confidence in the progress of our technical development and its potential to revolutionise the accessibility of gene synthesis. Evonetix is in a very exciting phase, having recently opened our early access program. This investment will support us as we execute on our commercial strategy to put benchtop gene synthesis in the hands of users.”

Towards the end of last year, Kite, a Gilead company, entered into an exclusive license agreement with synthetic biology company Refuge to access its gene expression platform to develop potential treatments for blood cancers.

Refuge’s synthetic biology system uses an expression modulation strategy to repress or activate transcription of target genes. Pre-clinical data has shown that the platform could regulate target antigen- dependent gene expression as a means to improve upon both the efficacy and safety of first-generation CAR-T cell therapies.

Speaking about the partnership Francesco Marincola, MD, Global Head of Cell Therapy Research, Kite says: “First-generation autologous CAR-T cell therapies have dramatically changed outcomes for people with certain blood cancers yet more work needs to be done to reach additional patients. As the global CAR-T cell therapy leader, Kite is working on the next generation of cell therapies with the goal to improve upon the great results we have today so more patients can benefit. Through this exclusive license agreement with Refuge’s platform, coupled with our unique in-house research capabilities, we aim to create a new generation of CAR-T cell products to induce long-term remissions for more patients.”

Drug development

Kite later announced results from a Phase III trial of its CAR-T cell therapy Yescarta (axi-cel) in which it was compared against standard of care (SOC) treatment in patients with relapsed or refractory large B-cell lymphoma (R/R LBCL). The company states the therapy is the first treatment in almost 30 years to demonstrate an improvement in survival for this patient population.

For patients with this type of cancer, the SOC therapy has included multiple steps such as high-dose chemoimmunotherapy and stem-cell transplants. In the ZUMA-7 study, 94% of patients who received Yescarta were able to make it through to their stem cell transplant, compared with only 40% receiving SOC therapy.

Speaking about the study, Jason Westin Principal ZUMA-7 Principal Investigator said: “As the first treatment in nearly three decades to significantly improve survival for patients with relapsed/refractory large B-cell lymphoma, axi-cel can potentially change the standard of care for these patients who previously had very limited options for successful curative therapy.”

Advancing research

This year too saw pharmaceutical company Roche open its Institute of Human Biology (IHB) to help advance research into human model systems such as organoids. The IHB will leverage human model systems to accelerate drug discovery and development by improving our understanding of organs and how diseases develop.

Human model systems have emerged as an alternative to animal model systems to give researchers a more accurate reflection of biology and how therapies work within the body. Scientists use human stem cells to create miniature 2D or 3D living systems of tissues and organs. It’s thought that these ‘replicas’ can enable the discovery of new human biology and identify drug targets that might otherwise be impossible to find using conventional methods.

“Human model systems such as organoids are the future of our industry. They have the potential to enhance almost all the steps involved in the research and development of an innovative medicine,” says Professor, Dr Hans Clevers, Head of Pharma Research and Early Development (pRED) at Roche and a pioneer in the field of organoids. “The IHB will address long-standing and urgent challenges in drug discovery and development with the ambition to bring more effective and safer medicines to patients faster.”

“The work at the IHB has the potential to redefine how we discover and develop medicines over the next decade,” says Dr Matthias Lutolf, Head IHB at Roche. “The institute is uniquely positioned in bringing together biology, bioengineering and data science around human model systems and applying them to real-world challenges in drug discovery and research.”

DDW Volume 24 – Issue 3, Summer 2023

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