4basebio UK Societas is developing next generation gene therapy technologies and solutions.
4basebio started with a focus on the manufacture of synthetic DNA for use in gene therapies; our synthetic DNA has clear safety, efficacy, and cost benefits over current plasmid DNA. Our near-term objective is the development of Good Manufacturing Practice (“GMP”) grade synthetic DNA as a key milestone in the commercialisation of this core technology.
In validating and scaling our DNA, we have identified opportunities to address complementary areas along the gene therapy value chain.
Our recently established discovery business focusses on the testing and development of nanoparticles for use in non-viral delivery of nucleic acids. Alongside this, 4basebio is collaborating across a range of indications with partners who have achieved gene expression in relevant cell types in laboratory settings. Our ambition is to combine this know-how with our delivery technology and synthetic DNA to advance new potential therapies through proof of concept, pre-clinical and beyond.
At the heart of the business will be our ability to manufacture GMP grade synthetic DNA and non-viral nanoparticles for third party customers; and develop the combination of technologies and expertise into gene therapy solutions for further clinical development and commercialisation.
4basebio owns proprietary technology which will place it at the forefront of synthetic DNA manufacture for gene therapy and vaccine applications. We aim to combine this technology with an innovative non-viral DNA delivery platform to offer better and safer gene therapies and DNA vaccines.
4basebio focus of activity: DNA manufacture & gene delivery
4basebio aims to become a preferred supplier of proven high- quality DNA products and is therefore establishing a cost-effective process for large scale manufacture.
4basebio seeks to collaborate with partners to facilitate the functional validation of its nucleic acid payloads and in doing so develop a pipeline of promising gene therapy candidates.
4basebio UK Societas has two core business units:
The MANUFACTURING unit offers services for customers such as biotech and pharma in gene therapy and gene vaccine development. 4basebio can help with the design of genomic vectors and then enzymatically synthesise hpDNATM constructs or mRNA in large quantities with high purity in a relatively short timeframe. After the production process, 4basebio can package the genetic payload into non-viral delivery systems.
4basebio will further assist clients with the GMP & CMC process development of their end product.
The PRODUCT DISCOVERY unit’s main objective is to validate and advance our innovative gene therapy DNA payloads and delivery systems both in vitro and in vivo. This unit will also focus on combing these delivery systems with clinically relevant payloads with a view to progressing them from proof of concept through to phase I clinical trials.
Transcending Adeno-associated viral vectors (AAV)
The recent approval and success of several AAV based products has propelled the vector system to a preferred choice for genetic payload delivery. Significant numbers in discovery and clinical trial projects are currently using this vector system.
The AVV system however has got some serious drawbacks. Most notably is its immunogenicity, which compromises repeat dosing strategies. It also shows levels of toxicity particularly when higher viral loads are required to effectively delivery a gene of interest. Inherent of this approach is the small packing capabilities limiting the genes that can be targeted. Finally, the manufacturing of AAV vectors remains a difficult and expensive process resulting in expensive products for patients.
4basebio’s Trueprime driven hpDNATM synthesis technology enables production of otherwise difficult to make large gene constructs. We combine our capabilities to make big genes with our HermesTM nanoparticle technology to effectively package these large DNA constructs in stable and safe vectors. This circumvents the major drawbacks of plasmid/AAV vectors which are inherently limited to 4.5kb payloads and carry significant safety concerns.
The ability to efficiently deliver much larger payloads enables 4basebio to explore a whole new array of therapies where AAV vectors are unable to be effective. 4basebio strives to expand into the gene therapy field with its high performant, safe and cost-effective hpDNATM constructs and its novel non-viral delivery platform.