Fierce Biotech Summit: Disruptive gene technology is here—and society seems willing to pay for it

As the cell and gene field builds off a quarter of great scientific progress, the industry may be teetering on the brink of transforming medicine as we know it.

At the Fierce Biotech Summit last week, Avrobio CEO Geoff MacKay shared his insight on the current cell and gene landscape, and what the outlook is like for reimbursement once more programs pass the FDA threshold. He pointed to an estimated15 companies in the field preparing to file for approval with the agency in 2023.

“You can slice and dice that any way you want,” the CEO of the Massachusetts-based biotech said of the expected FDA submission number. “If half of those actually lead to approvals, that's a measurable change in healthcare.”

Those submissions include Vertex and CRISPR's exa-cel, an ex-vivo gene-edited therapy for patients with sickle cell disease and transfusion-dependent beta thalassemia. If given the green light, it would be the first FDA-approved gene therapy to treat sickle cell disease, not to mention the first CRISPR therapy for a genetic disease.

Further up the cell and gene pipeline are around 200 companies actively conducting phase 3 clinical trials, MacKay said. If even a third of those late-stage products make it to market, the healthcare system is poised for a “very, very, very big change,” he explained, with single infusions potentially replacing a multitude of lifelong therapies for chronic conditions.

“The disruptive technology is here,” McKay said. “It's proven it works.” 

As recently as 2019, the FDA said it was expecting to approve 10 to 20 cell and gene products a year starting in 2025. The estimation was based on an analysis of the industry pipeline at the time and clinical success rates.

Avrobio's CEO thinks the industry is now working to soften some of the rough edges unique to cell and gene therapies, such as addressing safety signals as well as chemistry, manufacturing and control issues. Amid work to overcome such science-specific challenges, an even greater obstacle looms over the sector—who will pay for the therapies?     

Since the FDA approved Novartis' Zolgensma in 2019, society has grappled with the high price of such therapies. In the past few months, bluebird bio has surpassed that price marker, with its beta thalassemia therapy Zynteglo’s $2.8 million tag quickly outdone by Skysona's $3 million label. The biotech has struggled in some regions due to these high costs, winding down its operations in Europe due to a so-called "broken market."

While there’s no guarantee, or as MacKay puts it, no “god-given right to get reimbursement,” the CEO believes companies that demonstrate an attractive cost offset and health benefit for their therapies are likely to get some sort of compensation.

“It's wonderful to be sitting here in September of 2022,” MacKay said, noting that his outlook is very different from a year ago. The CEO told the summit that society has proven its willingness to reimburse for safe and effective therapies, pointing to several recent examples including Orchard Therapeutics’ Libmeldy in Germany and BioMarin’s Roctavian across Europe.

For now, privately owned biotechs with earlier-stage assets have to focus on securing investor funds amid the bear market as others push their later-stage programs to the FDA’s doors.