Ep. 261, Chapter 4: Overcoming Regulatory and Manufacturing Challenges With Amber Salzman, Ph.D.

16:56 – 20:07

Epic Bio’s novel epigenetic approach to treat FSHD functions by methylating a specific DNA region, which is expected to reduce the expression of the toxic Dux4 protein. The company seeks to use methylation data as a potential biomarker and regulators have agreed that higher methylation correlates with less disease severity. However, they require human study data to prove that Epic Bio's treatment can successfully increase methylation and change the course of the disease.

In terms of manufacturing, Epic Bio prioritizes a QbD approach has collaborated closely with a CDMO to create a manufacturing process built to commercial standards, preventing the need for costly trial repetitions and ensuring a smoother path to regulatory approval. This forward-thinking strategy has resulted in positive feedback from the FDA regarding the company’s CMC process.