Phacilitate’s annual event dawns as cell and gene therapies reach a new tipping point: the science has hit new heights just as regulatory and government policies spark momentum and frustration.
Advanced Therapies Week, hosted by Phacilitate in San Diego next month, comes at a time of great complexity for the cell and gene therapy sector. We’ve solved for science, proclaims the title of one session, and recent FDA guidance aimed at accelerating the development of CGTs is certainly propelling momentum in the space. However, a spate of rejections and manufacturing woes in 2025 suggests there is still a long way to go before these therapies truly change the face of medicine.
On Feb. 11, I’ll be moderating a panel at the event entitled: Geopolitics, policy, and the new global landscape for Advanced Therapies. Christina Hartman, head of US Government Affairs and the Washington, DC Office at Orchard Therapeutics; Michelle Lynn Hall, general partner at Entrée Bio; and I will discuss how advanced therapies are being reshaped by geopolitics, funding shifts and emerging regional capabilities.
We’ll discuss how U.S. momentum behind mRNA research has taken a hit under Health Secretary Robert F. Kennedy Jr. In August 2025, the Department of Health and Human Services axed 22 mRNA vaccine projects worth around $500 million, and just last week, mRNA specialist Moderna announced it is halting late-stage vaccine trials amid growing U.S. skepticism.
Meanwhile, China is quickly ramping up its biotech presence. The number of novel medicines under development in the country has increased eightfold in the past nine years, nearly equaling U.S. output, Flagship Pioneering CEO Noubar Afeyan wrote in his annual letter earlier this month.
But advanced therapies in the U.S. have certainly seen success as well. Just ask little KJ Muldoon, who in May received the first-ever personalized CRISPR treatment at Children’s Hospital of Philadelphia to treat an ultrarare genetic condition called CPS1 deficiency. KJ featured prominently in the FDA’s “plausible mechanism pathway” guidance, unveiled by Commissioner Marty Makary and Center for Biologics Evaluation and Research head Vinay Prasad in November.
Earlier this month, a group of CRISPR pioneers, including Nobel laureate Jennifer Doudna, launched Aurora Therapeutics to perhaps further capitalize on this regulatory pathway. The biotech’s stated goal: To develop the industry’s first platform technology that can reliably and repeatedly produce gene therapies to address rare disease–causing mutations that would otherwise be implausible to treat at scale. I look forward to hearing about more such endeavors in the Feb. 10 session: Gene therapies’ second act: Scaling sustainable modalities for the future.
“Gene therapy has moved beyond proof-of-concept to a phase where operational reliability is just as critical as scientific innovation,” Andrew Krajewski, director of client development at Forge Biologics and the session’s chair, told BioSpace in an email. “Success now depends on the ability to manufacture AAV therapies consistently, at scale, and under cGMP conditions that support late-stage and commercial programs.”
Other positives have been overshadowed by regulatory uncertainty, however, as the FDA continues to evolve under new leadership. UniQure is a prime example. In September, the company reported that its gene therapy AMT-130 elicited significant slowing of Huntington’s disease in a pivotal trial. Just five weeks later, however, that optimism deflated as the FDA seemingly changed its tune on the evidence required for an approval application for AMT-130—likely setting back the timeline for what could be the first disease-modifying therapy for the devastating neurodegenerative disease. Experts, too, urged caution, raising questions around AMT-130’s data about patient selection and the safety of the required intracranial surgery.
UniQure’s story is not unique. “FDA rejections are on the rise,” notes the write-up for the panel: We’ve solved for science. What now? Capricor Therapeutics’ investigational cell therapy deramiocel, which the biotech is proposing for cardiomyopathy associated with Duchenne muscular dystrophy, and Ultragenyx’s in vivo gene therapy UX111 in Sanfilippo syndrome type A, were both hit with surprise rejections last summer.
Amid all of this, of course, investors are watching closely. Advanced Therapies Week kicks off with an Investment Summit, where speakers will present a data-driven outlook on the current state of CGT investment, and a panel will explore “what fundable companies look like in 2026.”
Biopharma funding is starting to pick up after a drought that extended through the first half of 2025, only thawing in the fourth quarter—but not for early-stage biotechs, many of which play in the CGT space. And while a handful of Big Pharma companies, including Pfizer, Amgen, Novo Nordisk and Eli Lilly, have signed Most Favored Nation drug pricing deals with the White House to avoid tariffs, these same smaller biotechs hang in limbo.
Finally, I’m excited to see so many sessions focused on women’s health and women biotech founders. One roundtable on Feb. 9 plans to “explore strategies for securing funding, support and recognition for women-led initiatives.” Recognizing women-led initiatives and companies is something BioSpace has long prioritized, and I look forward to learning which new initiatives we’ll be writing about this year.
Editor’s note: BioSpace is a media partner of Phacilitate’s Advanced Therapies Week, held Feb. 9–12 at the San Diego Convention Center.
