Calculating viral vector yields is essential to determine how much drug product is needed for a specific gene therapy. This calculation will determine either how much internal manufacturing capacity is needed or how much it may cost to manufacture at an external contract development and manufacturing company (CDMO).
Data collected from trials as well as REGENEX's gene therapy programs for the treatment of other rare lysosomal storage diseases have the potential to broaden our understanding of AAV gene therapy in the CNS.
On the heels of a 2020 study predicting substantial job growth in the cell and gene therapy sector in Greater Philadelphia, industry leaders in our region have created the Life Science Talent Pipeline Collaborative to ensure a strong talent pool for the future.
With a small number of approved cell therapies/ex-vivo gene therapies, and with those approved therapies reaching small patient populations, there is not a proven strategy to answer the questions about large-scale commercial manufacturing. The developers of these processes are taking different strategies for commercial manufacturing as they weigh several factors.
With several gene and cell therapies currently in development and even more rapidly moving toward the development stage, a big bottleneck in this evolving field is identifying sites with the capability and capacity to conduct these resource-intensive studies.
Over the past 12 months the news has been dominated by the worldwide COVID-19 pandemic, but the work to bring gene and cell therapies to patients has not stopped. Here is a rundown of some of the most interesting developments in gene and cell therapy for the year.
For so many of us in the cell and gene therapy industry, we entered this field because we were deeply motivated by the life-changing potential such therapies, if approved, could have for patients, and by extension, their families, and communities.