Regulatory Challenges in the Development of an Extracellular Vesicle-Based Clinical Product
Time: 1:05 pm
day: Pre-Conference Workshop Day
Details:
In this workshop we will discuss how to navigate the intricate regulatory requirements to translate extracellular vesicles loaded with PTEN-siRNA (ExoPTEN) from preclinical to clinical phase.
Strategies for the manufacturing process and controls include:
- Scaling up the bioprocess to produce higher yields and quantities.
- Development of a proprietary moiety for efficient loading of siRNA
- Establishing a matrix array of multiple potency assays to measure several attributes related to ExoPTEN’s therapeutic activity
The following preclinical aspects will be considered:
- Biological relevancy of the animal model
- Dose finding and quantification studies. How will the therapeutic unit be defined, for example, EVs concentration, total RNA content
- Safety studies for off‐target assessments of ExoP
- Biodistribution and pharmacokinetics profiles.
