Formulation development for ATMPS in high demand
R&D efforts for ATMPs have increased dramatically over the past decades, owing to recent breakthroughs in genetic engineering and improved gene vectors. By the end of 2020, 17 ATMPs have received market approval in Europe (8 of those since 2018), and the European Commission foresees new promising therapies in their recently published Pharmaceutical Strategy for Europe. This statement does not appear exaggerated, since more than 1,200 clinical trials (150 in Phase III) with gene-, cell-, or tissue-based therapeutics are ongoing. Worldwide, developers of ATMPs raised $20 billion in financing in 2020, almost doubling the previous record from 2018.
Advancing ATMP stability
Despite the promising forecasts for ATMPs, the reality is that formulations of viral vectors used for gene therapy are far from advanced when it comes to stability. This directly impacts the potency / infectivity, and thereby potentially the safety of the therapy. For long-term stability of viral vectors, frozen storage at (ultra) low temperatures is often the only solution, similar to mRNA vaccines. Furthermore, cell-based gene therapy medicinal products commonly contain DMSO for stabilization via cryopreservation, i.e., storage in the vapour of liquid nitrogen. DMSO, however, is toxic for cells and can cause adverse effects in patients. A tailor-made formulation development program, which also explores lyophilization as an option, has the potential to remarkably improve the situation.
At Coriolis, we apply our scientific expertise and 13+ years of experience in liquid and lyophilized formulation development to obtain stable (bio)pharmaceutical drug products. This includes vaccines, viral vectors, nucleic acids, gene- and cell-based medicinal products, for which the demand for such services is on the rise. To test and optimize critical quality attributes (potency / infectivity, purity, empty vs. full capsids, etc.), we apply and develop dedicated methods for more than 100 specialized analytical techniques in-house. For viral vectors, analytical ultracentrifugation, qPCR, TCID50, ELISA and many particle characterization and identification techniques are of particular relevance. To meet the growing demand, we will further expand our ATMP development facilities under biosafety level S2 in 2021. A new building close to Coriolis’ headquarters in Munich is being reconstructed and will be ready within Q4/2021. In addition, we have been intensifying the research in our scientific unit to stay ahead of the growth curve and deliver science-driven solutions for ATMPs, following Coriolis’ mission: formulating innovation!
Coriolis Pharma Research GmbH
Dr Stefan Heindl
82152 Martinsried-Munich, Germany