Advanced Therapies (or Regenerative Medicines) including Cell, Gene and Tissue engineered products are attracting record levels of investment and leading to breakthrough ‘curative’ treatments for a range of diseases.
Challenges in Cell and Gene Therapy
The Synthace Solution
Accelerate R&D and product production times
56% of Cell and Gene Therapy Companies agreed automation should be adopted as early as possible in the development process.
To avoid bridging studies or issues when transferring a protocol to a CDMO, companies are looking to standardise methods via automation earlier into the process. Furthermore, by implementing automation into a discovery platform earlier on – time to IND can be accelerated.
Digitally Defined Methods
Digitally defined methods allow for the rapid and accurate sharing of optimised methods via the cloud across teams and sites, as well as with external partners such as CDMOs.
Increase yield, throughput and purity
80% of Cell and Gene Therapy companies recommend automating process development.
Creating processes that robustly and cost effectively scale with volume (allogeneic and gene therapy) or by units (autologous) requires a deeper understanding of the experimental design space. Process Development methods such as PAT (process analytical technology) and DOE are routinely used in CMC to achieve this.
High Level Control
Our platform dramatically reduces the planning and execution time of complex experiments such as a multifactor DOE. This allows complex and labour intensive – but insight rich experiments to become routine.
Processes need to account for donor variability
Autologous cell therapy products are made on a patient to patient basis, with methods often developed on healthy donor material.
A deep multifactorial understanding of the design space is needed to define representative critical quality attributes (CQAs), which then allow for adaptive manufacturing processes.
Enhanced sample traceability and analytics integration
Our platform is built to integrate with advanced analytics and machine learning – producing labelled and structured data sets. This is essential in QC environments, as well as in adaptive manufacturing.
Cost of Goods (CoGs) is still prohibitively high
87% of companies in this space think the CoGs is very important.
The Cost of Goods for a cell or gene therapy product is still prohibitively high preventing front-line usage. Contributing factors include: a global shortage of viral vectors, expensive single-use equipment and the labour-intensive nature of many manual processes.
Simulation of experimental methods before running gives qualified personnel confidence it will work. Important when handling small quantities of variable donor material or in the middle of a time constrained QC process.
Suggested applications in Cell and Gene Therapy
- Optimisation of growth media to drive increases in viral titre of AAV or Lv.
- qPCR automation for higher throughput QC testing in autologous Cell Therapy.
- Transfection and Transduction optimisation