If cell and gene therapy makers are going to achieve their mission to improve patients’ lives, the industry must come together to share information across stakeholders, from regulators to manufacturers to payers.
COVID-19 broke all the rules in pharma. Everyone worked together, and progress was remarkably fast. Within a year of mapping the virus’ genetic code, scientists had developed and tested multiple vaccines that received emergency use authorization from the FDA.
That kind of collaboration might be just what the doctor ordered for cell and gene therapy (CGT).
The wide range of novel modalities encapsulated within CGT are undeniable scientific breakthroughs with the power to save lives. The trouble is, the industry hasn’t figured out how to develop and produce them in a way that fits into the current financial, regulatory, manufacturing and healthcare systems. To put it bluntly, the economics of CGTs are not in anyone’s favor.
“Commercial is the most important,” AG Capital Advisors CEO Audrey Greenberg told BioSpace. “How quickly are you going to get to approval, and how much are people going to pay for it? How much is it going to cost to make it?”
Bluebird bio still serves as the poster child for a gene therapy maker with multiple products on the market that nevertheless failed to make ends meet. After once being valued at $10 billion, the company was sold to two private equity firms for less than $50 million.
While the reasons for CGT’s struggles are numerous, one pinch point is manufacturing. Great strides have been made toward scaling the production of these therapies, but processes are still largely dependent on paper batch records. “You can’t do this on paper,” Oribiotech CEO Jason Foster lamented during a panel I moderated at last year’s BIO Convention.
Alexander Seyf, CEO of Autolomous, doubled down on the importance of digitizing data, adding that this doesn’t just apply to manufacturing. Documenting failures is critical if the industry is to avoid repeating its mistakes, and data are, of course critical for market approval. “Whatever direction you look into . . . it all is about data,” Seyf said.
Even after a product reaches the market, insurers are going to want to see volumes of aggregated data, said Courtney Rice, principal at Acadia Strategy Partners. Cody Powers, principal at ZS Associates, agreed, telling BioSpace earlier this year that a lack of data-sharing explains why payers “aren’t necessarily convinced” of the “one-and-done model” for CGT reimbursement.
So everyone wants data, yet no one seems to want to share it. And the sector continues to suffer failures that are delaying access to potentially life-saving therapies while burning through capital. Layer on the regulatory uncertainty that has hit CGT like a steam train in the past year and a half, and the result is a hesitant investor base.
“The uncertainty at the FDA and CBER has resulted in . . . a perceived lack of investor confidence, whether that’s true or not,” Greenberg said. “Things that have caused drugs not to get approved result in investors pulling capital, which obviously affects all of us.”
As the industry waits to see who will replace Marty Makary as FDA commissioner, the agency has already begun pushing for data to be shared. Following a public listening meeting in September 2025, the FDA issued draft guidance in February emphasizing the importance of data-sharing for regulatory flexibility.
“Sponsors should seek early feedback on their nonclinical development plans to discuss data leveraging opportunities specific to their program,” the guidance reads. “Shared learning through appropriate data sharing is one opportunity to facilitate continued research.”
Sharing more data makes sense from all sides, according to Seyf—make the information available so “it can be democratized, in a sense, without really infringing their IP.”
Can we make that happen, akin to what went down during COVID? And would it be the difference between a successful CGT and one that never makes it to patients?
Randal Hyer, CEO of Merlin Biotech, sees that collaborative culture as key to success. In a former role at Moderna, Hyer helped bring to market the biotech’s mRNA-based COVID-19 vaccine at the height of the pandemic. At that time, he explained, the NIH and the Foundation for NIH (FNIH) organized a public-private coalition called ACTIV (Accelerating COVID-19 Therapeutic Interventions and Vaccines), recruiting 20 biopharma companies and several nonprofits to join forces with eight U.S. government agencies to accelerate research related to the virus.
“NIH had that ACTIV program where we were able to easily share a lot of information, which would not normally happen in other circumstances,” Hyer said. “And that shared sense of urgency, plus the openness amongst all developers, manufacturers, regulators, KOLs, you name it . . . really did I think contribute.”
Hyer, Seyf, Greenberg and Rice, along with Ronald Li of cardiovascular-focused CGT biotech Medara, will join me at BIO 2026 this week to discuss what needs to happen to accelerate progress in CGT.
