What does 4basebio do?
4basebio is developing a production method for next generation synthetic DNA (hairpin-DNA, hp-DNA). Our products will be the active ingredient in future gene therapies and gene vaccines. We are currently working to scale up our proprietary processes and are testing our hp-DNA products with a range of different delivery systems. Our products are currently available for research and pre-clinical use and we expect GMP certified product to become available in the near term.
What makes the synthetic DNA from 4basebio special?
Most DNA for therapeutic purposes is produced as plasmid DNA (pDNA) through biological fermentation, which is slow, costly and also has some safety concerns. Synthetic DNA could solve cost, speed and safety issues.
We call the DNA we produce hairpin DNA (hp-DNA). This is a synthetic form of DNA which is made as a double stranded linear but covalently closed DNA molecule. The DNA is closed DNA with unique proprietary loops or hairpins which offer significant flexibility in how our DNA can be used in therapies and vaccines.
What is the market for this DNA?
Synthetic DNA production/gene synthesis for gene therapy and gene vaccines applications. In all of these applications, a target molecule of DNA different for each therapy or vaccine needs to be synthesized to be delivered efficiently into the patient’s body. As of today, the main DNA synthesis method is based on complex plasmid DNA constructs where the target DNA molecule is combined with additional DNA fragments needed for downstream steps of gene therapy and gene vaccines workflows which could be harmful for patients. hp-DNA will be produced using a new and advantageous method that will considerably simplify the production process, which will translate into reduced costs and turn-around times.
hp-DNA could be used too in the future for other recently developed synthetic biology applications such as genome engineering.
How big is the market for this DNA?
DNA represents a significant part of the manufacturing cost for a therapy and can comprise between 20 – 40% of the total manufacturing cost. The average cost of plasmid DNA is currently around €5,000 per dose. On this basis a therapy for 100,000 patients would generate revenue from the sale of DNA of €0.5B. The number of gene therapy clinical trials continue to increase rapidly, and the expectation is that by 2025 the FDA will approve 20 new therapies per annum.
Who are your competitors?
The area of synthetic DNA is relatively new with only few companies active in this space. However, we believe our product has certain technical benefits which in turn is expected to make it more adaptable for a wider range of different DNA therapies and delivery systems.
In any event, the demand for synthetic DNA is expected to grow exponentially as its benefits over plasmid DNA are recognised.
Do you develop the gene therapy or vaccine?
No, our role is to supply the DNA required in therapies at discovery, pre-clinical, clinical trial and eventual commercial clinical stages. Whilst we are not a therapy development company we may decide to support and invest in certain companies we supply should the opportunity arise.
How do you plan to fund your development?
4basebio is extremely well funded following the sale of its proteomics and immunology businesses to Abcam plc on 1 January 2020.