Basel, Switzerland, 2025 — The American Society of Gene & Cell Therapy (ASGCT) Annual Meeting continues to be a key indicator of where the field is heading. This year, one trend was unmistakable: RNA–LNP therapeutics are gaining momentum, and the field is advancing at an accelerated pace.
Here are the major developments that caught our attention:
RNA–LNP (lipid nanoparticle) therapeutics are solidifying their role as a leading platform for nucleic acid therapeutics. Several features explain their increasing adoption:
RNA provides transient expression, meaning the encoded protein or tool (e.g., CRISPR/Cas9) is expressed for a limited time, reducing the risk of off-target effects and prolonged exposure
LNPs promote cellular uptake while minimizing immune responses, supporting a favorable safety profile.
Together, these features address critical challenges in safety, precision, and flexibility, especially when compared to viral vectors systems. At ASGCT 2025, it was evident that these advantages are increasingly influencing both clinical development decisions and regulatory strategies.
In his keynote address, Dr. Drew Weissman—Nobel Prize in Physiology or Medicine for their discoveries concerning nucleoside base modifications that enabled the development of effective mRNA vaccines against COVID-19—emphasized the potential of in vivo CAR-T cell therapies through mRNA–LNP delivery.
Traditional CAR-T therapies involve collecting patient T cells, engineering them ex vivo, and reinfusing them, a process that is both resource-intensive and time-consuming. In contrast, in vivo CAR-T generation using mRNA–LNP to deliver the genetic payload directly to T cells within the body has the potential to:
Eliminate complex manufacturing steps
Reduce treatment timelines
Make personalized immunotherapies more accessible at scale
Additionally, it includes advantages associated to RNA-LNP therapeutics, such as improved safety due to mRNA transient expression and refined dosage control.
It represents a meaningful shift in how cell therapies might be designed and delivered in the coming years.
The ability to target LNPs to specific tissues or cell types is becoming increasingly important. At the conference, several presentations focused on next-generation LNP formulations that improve tissue specificity, cell uptake, and delivery efficiency.
Targeted LNPs can reduce off-target effects, minimize systemic toxicity, and enable precision medicine applications in oncology, rare diseases, and beyond. This level of control will be essential as RNA therapeutics move into more complex indications.
Artificial intelligence (AI) is no longer theoretical in Life Science R&D. AI is actively being integrated into workflows across the discovery and development spectrum.
At ASGCT, several groups presented tools leveraging AI to:
Optimize RNA sequence design for expression and immunogenicity
Predict LNP formulation performance across biological systems
Model biodistribution and delivery kinetics with greater accuracy
By reducing trial-and-error and accelerating iteration, AI is helping teams shorten timelines and de-risk decision-making in preclinical and clinical stages alike.
A major highlight of the meeting was the announcement of the first successful base editing treatment in a human patient, a child referred to as KJ Muldoon.
This patient was treated for a previously incurable metabolic disorder using base editing, a technology that allows for precise single-nucleotide changes in DNA without double-strand breaks, making it a safer alternative for gene editing clinical applications.
In KJ’s case, the success of this therapy was made possible through the use of RNA–LNP delivery systems. Instead of delivering the base editing components via DNA (which could integrate into the genome) or using viral vectors (which pose immunogenicity and manufacturing challenges), the editing tools were encoded as mRNA and packaged into lipid nanoparticles (LNPs) for delivery.
The success of this intervention marks a milestone in genomic medicine and underscores the translational potential of RNA-guided gene editing systems delivered through lipid nanoparticles.
At CATUG, we specialize in contract research, development, and manufacturing (CRDMO) services for RNA- and DNA-based therapeutics, with a deep focus on LNP formulation and analytics. Our integrated capabilities allow us to support innovators from early discovery through scalable manufacturing.
The 2025 ASGCT Annual Meeting is a testament to the speed with which RNA therapeutics are moving from research into the clinic and the real impact they can have on patients’ lives—not only at the vaccine level but also for gene editing and cancer treatment.