The Cell and Gene Therapy Catapult (CGT Catapult), an independent innovation and technology organisation specialising in the advancement of the cell and gene therapy industry, has established a cross-industry consortium to improve the understanding and process optimisation of adeno-associated virus (AAV) formulation.
AAV-based gene therapies are being developed to treat a broad range of inherited and chronic diseases and several of these therapies have already been approved for clinical use. Additionally, AAVs are the most popular gene delivery system currently used in gene therapy clinical trials. However, there are significant challenges during the development and manufacturing of these therapies that can impact both their shelf-life and potency.
To overcome these challenges, the CGT Catapult has set up a consortium comprising Croda, ikarovec and UCL’s department of Biochemical Engineering. The expert organisations will work together, alongside the CGT Catapult, to increase understanding of AAV formulation, characterisation and stability. The work of the consortium will include optimising AAV formulations for two different types of AAV, including a serotype that is currently being developed for clinical use.
“We look forward to working with our partners in this consortium to improve understanding of a vital component for many gene therapies, AAV. This consortium will help Croda accelerate the development of a systematic understanding of the impact of existing and novel excipients on the formulation of AAV therapies, looking to overcome some of the known challenges of this delivery system. For ikarovec, it will help accelerate the development of its gene therapies while creating capability to drive forward the industry more generally.”
Matthew Durdy, Chief Executive of the Cell and Gene Therapy Catapult