One company at the forefront of this shift is 4basebio, a UK-based platform technology company specializing in the production of synthetic DNA tailored for advanced therapies. The company recently achieved a major milestone with good manufacturing practice (GMP) certification, further enabling its support across the full drug development lifecycle.

To learn more about the unique advantages of synthetic DNA and what this means for the future of gene therapy, Drug Discovery News spoke with Amy Walker, Chief Operating Officer at 4basebio, who leads the development and commercialization of the company’s synthetic DNA and non-viral delivery platforms.

For those unfamiliar with 4basebio, how would you describe your mission and what sets your synthetic DNA technology apart from conventional plasmid approaches?

4basebio is a platform technology company specializing in the enzymatic production of synthetic DNA for the cell and gene therapy and vaccine sectors. Our mission is to accelerate the development of next generation therapies by delivering high-performance synthetic DNA with significantly shorter lead times and performance benefits as compared to traditional plasmid-based methods.

You’ve developed multiple DNA formats, hpDNA, osDNA®, opDNA®, and oeDNA®. Could you walk us through how these differ and the specific advantages each offers for therapeutic or research applications?

What sets 4basebio apart is our ability to tailor DNA formats to specific therapeutic needs. Take opDNA®, for example, it’s optimized for mRNA production, featuring an open 3' end that removes the need for enzymatic linearization. This simplifies workflows, reduces costs, and improves overall quality. hpDNA is ideal for viral vector production, particularly in AAV applications, offering a safer, more cost-effective alternative by eliminating bacterial backbones and antibiotic resistance genes. osDNA® gives researchers control over immunostimulatory responses, allowing them to dial the innate immune activation up or down depending on their application. All 4basebio molecules can facilitate DNA dose reductions in downstream applications due to lack of bacterial backbone.

You’ve recently received GMP certification. What does this mean for your capabilities and how will it impact customers working on advanced therapies?

Our GMP certification marks a significant milestone, it means we can now support customers from early discovery through to clinical trials. It validates that our manufacturing processes meet the highest regulatory standards, offering peace of mind when it comes to quality, compliance, and patient safety. Critically, it enables faster access to GMP-grade synthetic DNA, simplifies regulatory pathways, and reduces development risk, advantages that are essential in the fast-paced field of advanced therapies.

What are some of the biggest challenges in sourcing high-quality DNA for drug development, and how are newer platforms helping to overcome them?

One of the most persistent challenges our customers face is the long lead time associated with plasmid DNA production, often stretching from nine to twelve months. Our enzymatic approach removes the need for master cell banks and fermentation, allowing us to deliver GMP-grade DNA in as little as 8 weeks from sequence to product.

Another major advantage is flexibility. Unlike traditional plasmid manufacturers who rely on large bioreactors, our enzymatic process allows us to scale production according to customer needs, minimizing waste and cost. This is especially valuable in personalized medicine and mRNA-based therapies, where relatively small amounts of DNA may be required.

Additionally, because our DNA is entirely enzymatically synthesized, it contains no bacterial backbone or antibiotic resistance genes, mitigating contamination risks and enabling dose reduction due to increased purity and safety.

How does 4basebio support researchers and developers across different stages of the drug discovery process, from early exploration to preclinical development?

4basebio provides comprehensive support across the drug development continuum. In early research phase settings, scientists have the opportunity to evaluate our synthetic DNA either through off-the-shelf constructs suitable for mRNA and AAV production or through 1mg quantities of custom sequences at research use only (RUO) grade. Such studies can accelerate discovery timelines. As projects progress, we can provide high-quality DNA formats for late pre-clinical studies, including investigational new drug-enabling studies or good laboratory practice toxicology studies. This enables a smooth transition into regulated development by using high-quality material that is representative of GMP quality material that will be ultimately required for clinical application.

Our recent GMP certification gives our customers confidence that we can support their programs from discovery through to clinical application. This continuity reduces risk, saves time, and ensures consistent quality from research to clinical application. Our team works closely with customers to tailor formats and workflows to their specific therapeutic goals, ensuring optimal performance at each development stage.