Are Cell and Gene Therapies Overpromised or Facing Growing Pains | Asembia's AXS25 Summit

At the 2025 Asembia Specialty Pharmacy Summit in Las Vegas, Managed Healthcare Executive spoke with Joe DePinto, MBA, head of cell, gene, and advanced therapies at McKesson, about the challenges facing the cell and gene therapy (CGT) space.

Although CGTs have typically been celebrated for their potential to cure or dramatically improve rare and serious diseases, the actual number of patients receiving these treatments remains low.

DePinto discussed the importance of differentiating between cell therapies and gene therapies, which can often get grouped together despite having distinct development and delivery challenges.

He pointed out that one key to closing the gap between potential and reality is ensuring manufacturers deeply understand the value of their therapies and who should receive them.

“It’s really important that the manufacturers, as they move into the market, have a really intimate understanding of the value proposition for their product and the appropriate patients to utilize this product,” he said.

Part of the challenge, DePinto said, lies in aligning the right patient with the right site of care, such as a Center of Excellence.

These steps, along with medical and financial-related issues, make an already challenging area even more complicated.

In addition, the recent decision by Pfizer to pull its hemophilia B gene therapy, Beqvez (fidanacogene elaparvovec-dzkt), from the market highlights another critical issue in CGT: whether the product can succeed in the market.

DePinto suggested that product success is usually less about a therapy’s inherent value and more about the broader therapeutic landscape.

He said that organizations should evaluate whether the need is truly unmet, how crowded the competitive space is and whether their product clearly stands out.

“You really have to look at what the market needs, what’s already out there, and what makes your therapy different,” DePinto added. “It’s not just about having a great clinical story—it’s about how that story fits into real-world practice, payer expectations, and whether systems are ready to support it.”

This slow uptake in CGT does not mean the therapies are oversold, he said.

It reflects how tricky and ever-evolving this space is, which requires clear planning and connections across the system to make these therapies work.