A shared scalable suspension platform for cost effective manufacture of novel AAV gene therapies

"The rapid development of Gene Therapy Medicinal Products (GTMPs) requires new manufacturing processes and facilities. Gyroscope Therapeutics Ltd and Freeline Therapeutics Ltd are UK-based SMEs developing gene therapies for different indications but which are both delivered via adeno-associated viral (AAV) vectors. Gyroscope and Freeline intend to co-develop an innovative, suspension-based viral vector manufacturing process, which will provide a shared platform for the manufacture of each company's GTMP products. The major challenge facing commercialisation of gene therapies is the manufacture and supply of products at a commercial scale. Gyroscope and Freeline will develop a proprietary, serum-free, scalable, suspension based manufacturing platform, which will improve product quality, safety, robustness and reduce the costs of goods at commercial scale. Working together with process development specialists at partner organisations the Centre for Clinical Biotechnology (CBC) and the Centre for Process Innovation (CPI), Gyroscope and Freeline will:

1. Establish ways to reduce risk of genetic instability of plasmid starting materials in an integrated approach;
2. Establish a proprietary animal component free and 'chemically defined' suspension cell culture manufacturing process which is scalable and which minimises product heterogeneity while maximising safety, quality and yield;
3. Supply batches of material to confirm comparability of product made using suspension platform material to that produced by current adherent cell culture platforms and so enable process changes to be implemented
4. Generate data to facilitate technology transfer of the developed process at a representative scale to GMP manufacturing facilities for subsequent qualification as a GMP manufacturing process.

Ultimately, a successful outcome will address a significant manufacturing challenge for GTMPs, strengthen the UK position as a leader in scientific innovation, offer scope for investment and employment into UK Pharmaceutical manufacturing and bring healthcare benefits to patients."