Syngensys
Transforming Patient Care with Synthetic Biology

Syngensys takes patient-centric solutions to new levels with its mammalian expression technology, a cornerstone of its innovative approach to genetic engineering. By integrating its profound expertise in genetic vector design with a commitment to addressing individual patient needs, Syngensys creates solutions essential for the precise control of gene expression needed for tomorrow’s therapies.

“We are confident that our innovative approach will significantly advance the manufacturing processes and enhance the safety and efficacy of novel genetic therapies,” says Professor David James, chief scientific officer.

Traditionally, the biopharma industry has adhered to a ‘one-size-fits-all’ model when employing mammalian expression technology. Generic vectors were the norm, with only the product gene being tailored to the therapeutic target. Syngensys recognised this as a significant limitation—a missed opportunity to enhance patient outcomes by creating more precise therapies. Determined to bring in a change, Syngensys now brings forth the creation of bespoke genetic vectors—through SynGenSys Genetic Information technology that ensures each biologic is produced with the highest fidelity and efficiency, addressing specific therapeutic needs and improving patient outcomes.

The impact of this is seen in two key areas—biologics manufacturing and cell and gene therapy. Its technology accelerates the biologics manufacturing process, facilitating the introduction of new biological medicines to the market.

With respect to recombinant protein manufacturing, as the industry embraces more complex protein formats—such as bi and trispecific antibodies and fusion proteins—the company’s technology plays a pivotal role. Its approach enables the precise design and identification of product-specific genetic expression vectors. This enhances product quality and expedites delivery to the marketplace, clinical trials or healthcare clinics.

The safety and efficiency of genetic medicines is a primary focus for Syngensys. Its proprietary platforms, namely SYNTAX, LEXICON and EXPRESS, grounded in a deep understanding of the human genome at the cellular and tissue levels, enable the company to design genetic components that precisely target specific tissues and deliver the desired dosage of genetic medicine. This allows Syngensys to control therapeutic entity production within the targeted tissue. For instance, in the case of AAV-delivered therapies, synthetic promoters can be engineered to embed tissue and cell type specificity while controlling the expression level of the therapeutic gene encoded on the vector, enhancing patient safety by ensuring that the gene therapy acts precisely where needed.

The precision enables manufacturers to create gene therapies that are more accurately tailored to achieve the intended therapeutic outcomes. One pertinent example is gene therapy for familial hypercholesterolemia. Here, an AAV-mediated therapy delivered a copy of the low-density lipoprotein receptor (LDLR) gene to a patient. Initially, the therapy showed promising results, but within a few weeks, the patient began to experience myopathy, muscle pain, weakness, renal dysfunction and liver stress. The root cause of these adverse effects was using a strong promoter in a genetic vector, which is also activated in muscle and kidney tissues. This highlights the critical need for cell type and tissue-specific genetic vectorology. When combined with AAV tropism, this specificity ensures the correct targeting and physiologically relevant expression strength, increasing the value and safety of the therapy for the patient.

Users are empowered by having control over expression vector design. Through its proprietary informatics platform, it rapidly develops variants with well-defined activities that can be quickly tested for specific applications. Using an engineering design process like those used to manufacture different models of the same car to meet customer specifications, Syngensys designs in silico and quickly tests in vitro or in vivo to produce tailored solutions.

The core strength of Syngensys’s platform lies in its capacity to produce new and effective synthetic DNA sequences, specifically tailored to achieve the desired activity and therapeutic results. A key aspect of its technological approach is the ability to manage multiple projects simultaneously, even when these projects have divergent intended outcomes. This removes much of the uncertainty associated with mammalian or human genetic vector design and allows for the rapid creation of solutions tailored to specific applications.