Key highlights

• The first-in-human, in vivo base editing therapy was unveiled at the 2025 ASGCT Annual Meeting.
• RNA platforms enable rapid, personalized treatments: the k-abe therapy moved from diagnosis to IND in under eight months for KJ.
• Modular RNA technologies support both single patient (N-of-1) and large population (N-of-many) therapeutics, accelerating development while maintaining safety.
• True progress requires collaboration among academia, biotechs, biopharma, and manufacturers to translate innovation into accessible RNA medicines.

Introduction

The field of RNA therapeutics continues to evolve at a rapid pace, and with it, the tools we have to treat genetic diseases are becoming not only more precise but more accessible and scalable.

This year, at the ASGCT Annual Meeting, I had the opportunity to attend a presentation that left a lasting impression: the k-abe case study. It detailed the first known in vivo base-editing therapy designed specifically for a newborn, KJ, diagnosed with CPS1 deficiency—a rare and often fatal urea cycle disorder.

In under eight months, the team went from diagnosis to dosing using a therapy composed of an mRNA-encoded adenine base editor and guide RNA (gRNA) delivered via lipid nanoparticles. The patient received two doses before turning one year old. That timeline is almost unheard of in traditional drug development, and it's a direct result of having platform-based technologies already in place.

A timeline that redefines what is possible

The development path for k-abe is not just clinically impressive. It’s a case study in what happens when modular platforms are fully leveraged:

Time Point	Milestone
Birth (Month 0)	Diagnosis of CPS1 deficiency confirmed.
Months 1-2	Guide RNA and base editor optimization completed.
Months 3-5	Preclinical validation in mice and NHPs; off-target studies performed.
Month 6	IND approved; clinical batch manufactured.
Months 7-8	Two in vivo doses administered using mRNA and LNP delivery.

The therapeutic components, editor mRNA, gRNA, LNP formulation, were assembled rapidly because the foundational systems already existed. This wasn’t just about speed. It was about precision, reuse, and the power of platform thinking.

Platform technologies enable scale without sacrificing precision

As former head of CBER, Peter Marks, M.D., Ph.D., noted in his accompanying editorial, the path forward for personalized therapies lies in leveraging the information that is used repeatedly from product to product. Regulatory frameworks are beginning to recognize the value of platform technologies, where the delivery method, quality controls, and even components like the mRNA-encoded editor can remain constant and only the guide sequence or target shifts.

We’re already seeing this mindset take shape across the field:

• Verve Therapeutics is advancing VERVE-102, an in vivo base editing therapy targeting PCSK9 for heterozygous familial hypercholesterolemia, delivered via mRNA and lipid nanoparticles. In March 2025, the company announced FDA clearance of its IND, following encouraging safety and LDL-C–lowering results from its Phase 1b program. This milestone signals growing momentum for RNA-encoded gene editors in common, chronic conditions and validates the regulatory and clinical potential of mRNA-LNP–delivered gene editing in cardiovascular medicine.
• CASGEVY, approved in 2023, marked the first CRISPR-based gene editing product to reach the market. While ex vivo, it helped pave regulatory and clinical paths that are now benefiting in vivo and RNA-delivered approaches.
• With patient-specific efforts like k-abe, we’re seeing that RNA technologies can span the full spectrum from personalized therapeutics to population-scale interventions.

This changes the landscape of gene therapies:

• You can develop N-of-1 therapies on timelines that match the urgency of disease.
• You can scale the same platforms to serve N-of-many populations.
• You can do so with repeatable, modular manufacturing and data-backed regulatory confidence.

Reflections from the room

What made the ASGCT session even more memorable was the energy in the room. The sense of purpose was unmistakable. Not just from the presenting team, but from everyone who contributed behind the scenes.

The success of k-abe was built on collaboration across the entire ecosystem. As soon as the lead gRNA and base editor were identified, teams at IDT, Aldevron, Danaher, and many others moved quickly and collaboratively to support the patient. This wasn’t a routine project—it was a coordinated effort involving hundreds of people who were deeply invested in the outcome.

One of the standout contributors was the Innovative Genomics Institute (IGI), led by Jennifer Doudna. IGI played a critical role in the early design and evaluation phases, developing key methodologies that will help streamline future N-of-1 programs. For example, they used the patient’s own genome to guide off-target screening, a practice that not only improved safety for this case but also pointed toward more scalable platform approaches for future therapies. Their foresight in building tools for modular, repeatable development underscores just how aligned this work is with the future of programmable, RNA-delivered medicines.

It was, as I said in a post shortly after:

“An incredible story that celebrates years of brilliant, hard and dedicated teamwork from a literal army of people across academia, biopharma, and the NIH.”

Our role in what comes next

At Eclipsebio, we see our impact in enabling these types of efforts. We’re not building therapies ourselves; we instead support our drug development partners by providing our sequencing assays and analytical insights to help advance programs faster and with greater confidence.

One way we enable this progress is through eMERGE, our sequencing-based platform for characterizing and optimizing RNA therapeutics, including mRNA, saRNA, and circRNA therapies. As programmable medicines become more modular and complex, eMERGE provides the critical data drug developers need to make informed decisions at every stage, from early sequence optimization to IND-enabling studies. The platform is designed to be consistent, repeatable, and modular, supporting each phase of development with assays that stay robust across applications.

This is where we believe we can make the greatest difference by building infrastructure and insights that help accelerate both personalized and broadly applicable RNA-based therapies.

We’re entering a new phase of gene editing. One where RNA is not just the delivery mechanism, but the common denominator across an entire generation of programmable medicines.

And we’re excited to help make that future possible.