USPs and efficacy driving CGT competitiveness

Highlighting the need for efficient animal models, the panel discussed how the potency and efficacy of a therapy in its preclinical phase can reduce high manufacturing costs and make the process sustainable.

Uta Griesenbach, professor of molecular medicine at the National Heart and Lung Institute, Imperial College London, said given a choice to pick a modality, she would pick the one with a good animal model to prove efficacy in vivo as early as possible.

“I would pick a disease indication that has good biomarkers that could be followed up in clinical trials,” Griesenbach said, emphasizing that the disease should be one that is easy and fast to measure.

Fellow panelist and chief technical officer for AAVantgarde, Nina Kotsopoulou said, “I think in discovery it's important to get efficacy as your first goal. You don't always have the luxury of choosing between a number of modalities. You might end up choosing just the one that is efficacious, then you do have to work very hard to commercialize it.”

The panel pointed out the importance of having a unique selling point for the therapy and being aware of the competitive landscape in a particular disease indication.

“It is very important, particularly if you're trying to get funding,” Griesenbach said. “Being ahead of competitors or having a unique selling point that makes you stand out from the crowd is essential for attracting investment and for licensing.”

Additionally, the panel discussed how costs of manufacturing are impacted by a lack of potency assays on the chemistry, manufacturing, and controls (CMC) side, which delays therapies from moving to Phase III clinical trials.

“You need to have a validated potency assay before you go into Phase III,” said Franz Gerner, independent consultant for South Mountain Advanced Therapies Consultants. “There have been multiple delays in several gene-therapy trials because there was not a good potency assay, or the regulatory agency didn't accept that.”

With the cost of goods for rare diseases remaining high, Kotsopoulou added, “We do want the cost of goods to go down, and for rare indication, you will still need a significant number of people to support the product,” she said, citing post approval commitments that keep products on the market, quality organizations, and manufacturing facilities.