Editor’s (re)View: Cell and gene therapy sector regroups amid tough 2025

In welcome remarks at this week’s annual Cell & Gene Meeting on the Mesa, Alliance for Regenerative Medicine CEO Tim Hunt acknowledged the “realities” confronting the cell and gene therapy (CGT) sector, trying to strike a balance between being “too Pollyannaish” and “too gloom and doom.”

On the negative side of the ledger, Hunt said in 2025 the capital markets have continued to be a challenge. The CGT sector has been hit with investment headwinds since the high levels seen in 2020 and 2021, according to Hunt.

“It’s been a tough investor climate for all of biotechnology — and cell and gene therapy has not been immune to that, in fact in some ways it’s been harder over the years,” Hunt lamented. “There have been some safety issues that popped up, including some deaths that took place this year.”

In a report last month, Leerink analysts flagged the fact that investors are worried about potential headwinds from cuts to adeno associated virus-based in vivo gene therapy programs in the wake of patient deaths tied to Sarepta Therapeutics’ Elevidys and other recent cancellations.

Manufacturing challenges persist       

Several panel sessions at the Meeting on the Mesa focused on CGT manufacturing, a major challenge for the industry due to a lack of standardization and the fact that scale-up has often been an obstacle to regulatory approval and commercialization.

A panel on Monday laid out the CGT manufacturing challenges facing the industry as it looks to produce these therapies at scale. “The infrastructure around these therapies just hasn’t kept pace with the pace of the science,” said Ori Biotech CEO Jason Foster. “It’s this kind of difficult tradeoff that developers have to make sometimes between biology and automation or flexibility and the desire to scale at some point.”

A Tuesday panel highlighted the complexity of the supply chain which hinders the sector’s ability to make products efficiently and at-scale. “There is no one-size-fits-all answer for this industry,” said Priya Baraniak, chief commercial and development officer at Pluristyx, pointing out that autologous therapies are fundamentally different from “off-the-shelf” allogeneic products, while cell therapies differ from gene therapies.

Although decentralized manufacturing could improve CGT access by addressing issues related to the high cost of production, scale, and distribution, the panelists agreed the most likely situation over the next 10 years is a hybrid ecosystem of both the centralized and point-of-care manufacturing models.

When it comes to accessing chimeric antigen receptor (CAR)-T cell therapies, Hunt in his welcome remarks at the meeting said approximately 80% to 85% of people eligible to receive these life-saving therapies do not get them. “It’s a very complex puzzle with a lot of pieces,” he commented. 

Cytiva, a Danaher company, announced this week that they received an award from the Advanced Research Projects Agency for Health (ARPA-H) to help transform CAR-T cell therapy for solid tumors. Their goal is to make therapies accessible with an aspirational target to reduce the cost of manufacturing by 10x over current standards. The key, they say, is engineering immune cells inside the body to avoid the autologous ex vivo process that drives up complexity and cost.

“By focusing on rapid development and scalable manufacturing, we aim to help deliver high-quality, innovative therapies that improve patient outcomes, increase accessibility and affordability, and create long-term value for the health system at large,” Sadik Kassim, Danaher’s chief technology officer for life sciences omics solutions and principal investigator on the project, said in a statement.