ARC Collaborates with Invest NI to Advance Gene Therapy Testing

ARC has successfully completed a 12-month research and development project aimed at improving the safety and success of emerging gene therapies, supported by almost £100,000 in funding from Invest Northern Ireland.  

Gene therapies using adeno-associated viruses (AAV) are transforming the treatment landscape for many genetic and rare diseases. However, the presence of pre-existing neutralising antibodies in some patients can significantly reduce the effectiveness of these therapies. Addressing this challenge requires reliable testing tools that help identify suitable patients before treatment begins. 

Through this project, ARC established and validated its first in-house ELISA platform method for the detection of Anti-AAV9 antibodies in human serum. This assay provides an important pre-screening capability for gene therapy programmes, supporting appropriate patient selection, and improving the likelihood of successful treatment outcomes. 

The funding enabled ARC to accelerate the development and commercialisation of its laboratory capabilities while reducing the risks associated with early-stage assay development. As part of the work, the ARC laboratory team installed specialist ELISA equipment, developed and optimised the assay workflow, and completed an extensive validation programme to demonstrate the precision, accuracy and robustness of the method. 

The new ELISA platform represents a significant technical milestone for ARC and provides a foundation for further expansion of the company’s testing capabilities. In addition to serum-based testing, feasibility work has already begun to explore the use of alternative sample types, including saliva and urine. 

Beyond the technical achievements, the project has also supported wider organisational growth. ARC has strengthened its laboratory infrastructure, expanded its scientific team, and increased engagement with pharmaceutical and biotechnology partners seeking advanced testing capabilities for gene therapy and precision medicine programmes. 

Alongside these developments, ARC has continued to strengthen its quality and regulatory credentials. The ARC Laboratory has recently achieved College of American Pathologists (CAP) accreditation, a globally recognised benchmark for laboratory quality and operational excellence. ARC now joins a small number of laboratories across the UK and Ireland to hold this prestigious accreditation. 

In addition, ARC has received confirmation from the MHRA that its laboratory can self-designate as a Health Institution under EU IVDR Article 5(5), enabling the organisation to manufacture and use in-house assays to accelerate early-phase precision medicine trials. 

Together, these achievements position ARC as a provider of clinically accredited testing services supporting the development of innovative therapies and clinical research programmes.

ARC extends its thanks to Invest Northern Ireland for its support in enabling innovation, strengthening scientific capability, and helping to advance Northern Ireland’s life sciences sector. 

Contact us to discuss how ARC Laboratory can support your current or upcoming trials – [email protected]