The development of gene therapies holds immense significance for medical innovation. Yet, the realization of their full therapeutic impact hinges on the urgent need for technological advancements that can further improve drug product (DP) quality and efficacy. Formulation, the penultimate operation prior to final fill, is crucial for robust stability that can impact product shelf-life, therapeutic delivery and potency. However, the complexity and resource investment required, coupled with a lack of comprehensive understanding of formulation science and best-practice for stability characterisation to ensure appropriate product quality control, have hindered progress. To overcome and address formulation and stability characterisation challenges, the Cell and Gene Therapy Catapult (CGTC) established a consortium leveraging expertise of key industry stakeholders, including a therapy developer (Ikarovec), specialist chemicals company (Croda), and academic research institution (UCL), each contributing their expertise, capabilities, and assets to deliver tangible solutions that will benefit the wider cell and gene therapy (CGT) industry. Following industry assessment, this project's activities represent novelty, depth and scale beyond what is currently being performed and will be foundational for future consortium activities aiming to benefit manufacturers across the industry.

"Age-related macular degeneration (AMD) is an eye condition affecting the central part of the retina (the macula), which can make it difficult for patients to read, drive or recognise people's faces. The wet form of AMD (wAMD) can develop quickly, causing serious changes to central vision in a short period of time -- over days or weeks as the retina starts growing new blood vessels to try and address the problem. However, these new blood vessels do not fully develop and instead cause swelling and bleeding underneath the macular which leads blank patches in the centre of an individual's sight. Around 600,000 mainly elderly people in the UK currently have sight loss caused by AMD, with around 70,000 new cases diagnosed each year. Globally the incidence of wAMD affects around 170 million people. The current treatment for wAMD on the NHS is with a group of medicines called anti vascular endothelial growth factor (anti-VEGF) antibody-based drugs that are injected directly into the eye every 4-6 weeks to stop the growth of new blood vessels and preventing further damage to the macula. Treatment approaches require frequent visits to ophthalmic surgeons for intraocular injections, which are unpleasant, expose the patient to risk of infection, can cause corneal scarring and over time retinal/macular fibrosis, which limits the long-term improvements associated with their use. In addition to the burden on patients, the burden on the NHS is considerable, with costs currently estimated at around £1.6 billion annually. The aim of this project is to develop a new gene therapy for wAMD that can be safely administered with one single injection resulting in long-term, 24 hour stable treatment that would be expected to last for many years and even decades, thereby improving the quality of life for AMD patients while at the same time significantly reducing costs to the NHS."