Advanced Therapies Week, organized by Phacilitate, will take place in San Diego from February 9 to 12. The event comes as the cell and gene therapy sector faces both scientific progress and ongoing challenges. A session titled "We’ve solved for science" reflects recent advances, including new FDA guidance aimed at accelerating the development of cell and gene therapies (CGTs). However, industry setbacks such as regulatory rejections and manufacturing issues in 2025 indicate that there are still significant hurdles before these therapies can have a broad impact on medicine.
A panel discussion scheduled for February 11 will focus on how geopolitics, policy changes, and shifting funding are influencing advanced therapies. Panelists include Christina Hartman of Orchard Therapeutics and Michelle Lynn Hall of Entrée Bio. The discussion will address recent developments such as reduced U.S. momentum behind mRNA research following decisions by Health Secretary Robert F. Kennedy Jr., including the cancellation of 22 mRNA vaccine projects worth about $500 million in August 2025. Moderna also recently announced it is halting late-stage vaccine trials amid increasing skepticism in the United States.
In contrast, China has rapidly expanded its biotech sector over the past nine years, with the number of novel medicines under development nearly matching that of the U.S., according to Flagship Pioneering CEO Noubar Afeyan.
There have been notable successes in advanced therapies within the U.S., such as a personalized CRISPR treatment administered to KJ Muldoon at Children’s Hospital of Philadelphia for CPS1 deficiency—an ultrarare genetic disorder. This case was highlighted in new FDA guidance on personalized therapies known as the “plausible mechanism pathway,” introduced by Commissioner Marty Makary and Center for Biologics Evaluation and Research head Vinay Prasad last November.
Industry analysts from William Blair noted that this regulatory pathway "could accelerate gene therapy/editing development," but said more clarity is needed.
Earlier this month, a group including Nobel laureate Jennifer Doudna launched Aurora Therapeutics with plans to develop platform technology for producing gene therapies targeting rare disease mutations at scale.
Andrew Krajewski, director of client development at Forge Biologics and chair of an upcoming session on scaling sustainable modalities for gene therapy, told BioSpace: “Gene therapy has moved beyond proof-of-concept to a phase where operational reliability is just as critical as scientific innovation. Success now depends on the ability to manufacture AAV therapies consistently, at scale, and under cGMP conditions that support late-stage and commercial programs.”
Despite some progress, regulatory uncertainty remains a challenge. For example, UniQure reported positive trial results for its Huntington’s disease gene therapy AMT-130 but later faced setbacks when FDA requirements changed regarding approval evidence—delaying what could be a first-in-class treatment.
Other companies have experienced similar obstacles; Capricor Therapeutics’ cell therapy deramiocel for Duchenne muscular dystrophy cardiomyopathy and Ultragenyx’s UX111 gene therapy for Sanfilippo syndrome type A were both unexpectedly rejected by the FDA last summer.
Capricor Therapeutics met with regulators after its rejection and plans to resubmit its application based on existing data from deramiocel trials.
Investor interest remains high despite these challenges. Advanced Therapies Week will begin with an Investment Summit analyzing current CGT investment trends. While biopharma funding began recovering late in 2025 after a slow start earlier in the year, early-stage biotechs continue to face difficulties accessing capital—even as large pharmaceutical firms like Pfizer, Amgen, Novo Nordisk, and Eli Lilly secured drug pricing agreements with the White House to avoid tariffs.
The event will also feature sessions dedicated to women’s health initiatives and supporting women-led biotech startups—a priority area highlighted by BioSpace coverage of high-profile acquisitions involving women-fronted companies in recent years.
BioSpace is serving as a media partner for Advanced Therapies Week at the San Diego Convention Center next month.
