The development of allogeneic therapies designed to fight certain cancers is capturing attention with a robust share of the pipeline, while simultaneously, the methodologies for commercially scaling these therapies are being optimized. The global pressures of COVID-19 have also increased the number of fast-tracked allogeneic therapies worldwide. For instance, in April 2020, the FDA granted immediate authorization for a team of University of Miami doctors to run trial-based stem cell therapy on patients suffering severe lung inflammation from COVID-19, building on a promising but limited 10-patient study in China.2 Championed by Israeli biotech, Pluristem Therapeutics, doctors at a Bergen County, New Jersey hospital injected cells from a placenta into a critically ill coronavirus patient for the first time, in the hope they would bolster his immune system and save his life.3
As the demand continues to grow and as allogeneic therapies progress towards late-stage maturity, the benefits of engaging an expert partner to scale up allogeneic cell therapy are coming into sharper focus. Whether it’s big pharma, or an emerging or virtual biotech, drugmakers large and small who are looking for venture capital investment need to show potential investors they have a manufacturing plan.6Manufacturing scalability challenges, especially with T-cell allogeneic therapies, continue to highlight the opportunity to engage a skilled CDMO partner who is prepared to address such challenges from raw material through commercial supply. A solid understanding of regulatory requirements around the handling of human-sourced material and managing time-sensitive processing, storage, and transportation of cells, are critical to the success of any advanced therapeutic. Coupled with supply chain complexity is the inherent challenge that each cell type has its unique biological blueprint making it difficult to move towards a standardized manufacturing line that achieves instant scalability.
For example, stem cell manufacturing in contrast to allogeneic immunotherapy may require further customized workflow, or a gene-modified immune cell may need added upstream processes vs. a non-modified cell. Engaging early with a partner who has the expertise to balance manufacturing processes for a variety of cell types (e.g. iPSCs, MSCs, etc) with customized biological blueprints can help overcome challenges for late-phase and commercial manufacturing.
Every cell therapy project is customized by the service provider and extensively evaluated from both a scientific and manufacturing perspective. The financial burden of continuous innovation for each project as well as moving biological materials through each step of the operations process exacerbates the need for efficiency within the manufacturing model. Engaging an expert CDMO partner who has the infrastructure and expertise in place from process development, manufacturing, and fill & finish thereby accelerates the path to commercialization.
Emerging trends of increased costs associated with commercial autologous CAR-T therapies are also paving the way for an allogeneic CAR-T treatment that can potentially add cost efficiencies while delivering similar efficacy. Innovative MSC allogeneic treatments will also need new capacity requirements in anticipation of these therapeutics hitting the market.4 US.1
Opportunity is knocking: By 2025 the FDA expects it will be reviewing and approving between 10 and 20 cell and gene therapies per year.7
Catalent has made significant investments in the last year in the cell and gene therapy space with acquisitions of both cell and gene therapy businesses to provide viral vector scale-up and production in combination with autologous and allogeneic development and manufacturing. This experience and expanding footprint is an optimal solution to the various scale-up challenges in the industry today.
References
- https://www.smh.com.au/business/companies/mesoblast-treatment-gets-us-tick-of-approval-20200813-p55liw.html
- https://www.miamiherald.com/news/health-care/article242008576.html
- https://www.nj.com/coronavirus/2020/04/nj-hospital-tries-experimental-placenta-therapy-on-critically-ill-coronavirus-patient.html
- https://www.pharmaceutical-technology.com/comment/gene-therapy-manufacturing-2019/
- www.fiercepharma.com/manufacturing/novartis-new-cell-therapy-facility-could-ease-manufacturing-squeeze-for-car-t-med
- https://www.dcatvci.org/5933-cell-and-gene-therapies-a-manufacturing-view
- https://www.biopharmadive.com/news/fda-gene-therapy-guidance-sameness-durability/571225/