RNA x Cell and Gene Therapy: exploring the Intersections
RNA has stepped from the shadows of vaccine development into the spotlight of next-generation cell and gene therapies. What was once viewed primarily as a rapid-response vaccine modality is now being applied to some of the most complex bottlenecks in cell and gene therapy (CGT) - delivery, safety, scalability, and flexibility.
At Lonrú, we wanted to look systematically at where RNA is already making an impact in CGT, and where the emerging evidence points to new opportunities. The result is an interactive Explorer that maps how RNA technologies intersect with CGT across payload types, delivery systems, and the companies pioneering these approaches.
Why RNA matters in CGT
The unique attributes of RNA make it an enabling technology in multiple ways:
Transient expression: Unlike DNA-based vectors that may persist long term, RNA expression is short-lived. This offers an alternative approach where temporary protein production or cell engineering is desirable, and it can allow for repeat dosing strategies.
Engineering flexibility: Ex vivo, mRNA can reprogram immune cells such as CAR-T or NK cells without the need for viral vectors, streamlining manufacturing.
Editing delivery: Co-delivery of editor mRNA with guide RNAs has become the backbone of in vivo CRISPR approaches, demonstrated clinically in Intellia’s NTLA-2001 program.
Expanding modalities: Self-amplifying RNA (saRNA) and circular RNA (circRNA) extend durability and stability, broadening therapeutic possibilities.
What the data shows
The Explorer collates citable evidence across peer-reviewed publications, clinical trial records, and company disclosures. Key takeaways from the Delivery × Payload Matrix include:
Electroporation + mRNA remains a clinical workhorse, with multiple CAR-T programs trialled using transient mRNA engineering.
LNPs + mRNA+gRNA dominate in vivo editing strategies, from NTLA-2001’s ATTR program (NEJM 2021) to recent prime editing demonstrations.
Custom LNPs designed for immune cells are emerging, pushing RNA delivery beyond the liver into T cells and other compartments.
Polymeric nanoparticles offer early preclinical signals as an alternative to LNPs, but remain less developed.
saRNA and circRNA are present largely at the platform and early translational level, with limited but growing relevance to CGT.
Explore the data
Within the Explorer, you can:
Cross-filter by payloads, delivery systems, or sponsor type.
Drill down into the Delivery × Payload Matrix, with linked examples that connect directly to publications, trial records, or company science pages.
Spotlight BioNTech and Moderna, whose RNA platforms are shaping new intersections with CGT.
Download the Lonrú curated Sources Table for further reading and reference.
Trace partnerships through the network view, with active, paused, and terminated collaborations all clearly mapped.
Why we built it
Our mission at Lonrú Consulting is to bring clarity to enabling technologies in CGT. By collating disparate data points into a single, interactive view, we aim to illuminate how RNA is already shaping the field - and where opportunities for new enabling partnerships lie.
Scroll down to explore the intersection between RNA and Cell and Gene Therapy.
