SHERIDAN, WYOMING – August 9, 2025 – The cell and gene therapy (CGT) sector stands at a pivotal moment. Once buoyed by multibillion-dollar valuations, companies like Sarepta Therapeutics and bluebird bio have faced sharp declines, underscoring the sector’s commercial growing pains. Industry leaders now agree: sustainable success will depend on advancing manufacturing, aligning global regulations, and better communicating value to payers.
Next-Generation Manufacturing as a Growth Engine
Panelists at the recent BIO Convention in Boston pointed to decentralized, modular manufacturing as a critical enabler for CGT expansion. The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) has taken the lead with formal guidelines for point-of-care production, a move welcomed by innovators like Ori Biotech CEO Jason Foster. In the U.S., the FDA has yet to implement a comparable framework.
Distributed manufacturing offers the potential to cut vein-to-vein times, avoiding cell freezing and improving access for critically ill patients. But standardization remains a hurdle. “You need automation, you need digitization. You can’t do this on paper,” Foster stressed, highlighting the need for digital batch records to ensure quality across sites.
Regulatory Harmonization and Quality Control Challenges
A fragmented global regulatory landscape adds cost and complexity. Mayo Clinic’s Audrey Greenberg warned that evolving potency assay requirements have delayed approvals for leading players like Sarepta and Novartis. Without harmonization, manufacturers face costly delays and uncertain pathways to market.
Greenberg emphasized that coordinated standards would benefit patients, accelerate approvals, and improve transparency for investors—potentially unlocking more capital for the sector.
Market Expansion into Larger Patient Populations
Cell therapies are moving beyond oncology into autoimmune conditions such as multiple sclerosis and lupus, opening doors to much larger patient pools. “All the big boys—BMS, Novartis, AstraZeneca—they all have plays in autoimmune,” Foster noted. This expansion mirrors the monoclonal antibody market’s growth, where manufacturing innovations eventually lowered costs and broadened access.
Industry leaders predict automation and process advances could bring CGT production costs down to $30,000–$35,000 per treatment, making front-line therapy more financially viable.
Communicating Value to Payers
While regulatory approval marks one milestone, reimbursement is the true commercial finish line. Courtney Rice of Acadia Strategy Partners argued the sector has failed to tell a compelling value story—one that frames high upfront costs against the long-term savings from reduced patient care needs. “We need to do a better job of packaging [the initial high price tag] in a palatable way to payers,” she said.
External Pressures: Tariffs and Onshoring
Trade policy uncertainty adds another layer of complexity. A new U.S.–EU agreement imposes 15% tariffs on pharmaceutical imports, with the possibility of much higher levies. While some manufacturers may offset costs through process efficiencies, industry leaders expect the onshoring trend in U.S. manufacturing to continue regardless of political cycles.
“Tariffs are geopolitical warfare,” Greenberg stated, calling them a lever for U.S. dominance in targeted modalities.
From Promise to Practicality
Despite market turbulence, panelists remain optimistic that CGT can scale successfully. As automation, AI-driven process control, and regulatory adaptation converge, the industry may finally be able to “mass-produce” personalized medicine—bringing it within reach for more patients, at sustainable costs.
“The scale problem,” concluded ElevateBio’s Mike Paglia, “is starting to get solved.”
Use the company’s official site for accuracy: https://www.bluebirdbio.com.