Lilly Bets Big on RNA Editing with $1.9B Deal for Ascidian’s Exon Editing Platform

(Source: Pharmatimes)

In June 2026, Ascidian Therapeutics announced a global research collaboration and licensing agreement with Eli Lilly to jointly develop RNA exon editing therapies for undisclosed monogenic kidney diseases.

Under the agreement, Eli Lilly will obtain exclusive rights to Ascidian’s RNA exon editing technology for an undisclosed kidney disease target. Ascidian will lead early discovery and part of the preclinical studies, while Eli Lilly will be responsible for the remaining preclinical work, clinical development, manufacturing, and commercialization.

In return,Ascidian to Receive Up to $1.9 Billion in Milestone and Royalty Payments, and retains the right to independently or jointly with other partners advance other targets in the nephrology field.

Founded in 2022, this young company draws its name from a simple marine organism: the sea squirt (ascidian). To mature from larva to adult, ascidians remodel their transcriptome through RNA trans-splicing and alternative splicing. Ascidian mimics this mechanism by designing exons with correct RNA sequences that interact with target precursor messenger RNAs.Replacing the cell's endogenous mutant exons via trans-splicing to achieve RNA-level "rewriting"。

Unlike traditional DNA gene-editing technologies (such as CRISPR), Ascidian’s RNA exon editing technology neither modifies genomic DNA nor introduces any exogenous enzymes, but ratherLeveraging the Cell's Endogenous RNA Splicing Machinery for RepairIts RNA exon editor is capable of editing multiple complete exons on a kilobase scale, specifically targeting large genes or genes with high mutational variability, thereby expanding the application boundaries of gene therapies.

Currently, more than 60 genetic disorders are known to affect the kidneys, with over 3.5 million people in the United States alone suffering from severe hereditary kidney diseases. These conditions are typically caused by large genes or heterogeneous mutations, making them difficult to effectively intervene with existing technologies. Ascidian’s technology aims toAchieve durable efficacy of gene therapy while significantly reducing the risks associated with direct DNA editing and gene replacement therapies。

It is worth noting that as early as 2024, Ascidian had alreadyPotential Total Amount: Approximately US$1.8 Billionwith RocheSigned Collaboration on Neural Target Research.

The global RNA editing sector is experiencing a robust rise, with overseas industry leaders such as Wave Life Sciences and Ascidian Therapeutics taking the lead. A cohort of emerging biotech companies continues to secure substantial financing, accelerating both technological iteration and commercialization. The domestic sector in China has likewise entered a phase of rapid development, forming a diversified industrial landscape with multiple areas of growth.

Hui-Gene Therapeutics leverages CRISPR/Cas13 technology to develop multiple RNA-editing therapies, with its core pipeline securing dual Rare Disease Designations from the U.S. FDA;

Boya Ji Yin'sThe RNA base editing platform, based on LEAPER™ technology, is being developed to create in vivo therapies for ophthalmic diseases, neuromuscular disorders, and other genetic and non-genetic conditions;

RecoRNAEstablished the MIMIC RNA editing platform, which utilizes endogenous adenosine deaminases for precise and efficient RNA editing, enabling the repair and optimization of protein sequences and functions without permanent genomic modifications;

RuiZheng GeneFocusing on Intracellular Targets: Developing Innovative Therapeutics for Patients with Congenital Genetic and Acquired Diseases Based on Next-Generation Gene Editing, RNA Editing, and Delivery Technologies with Superior Safety, Efficacy, and Targeting Precision

References:

1.https://ascidian-tx.com/wp-content/uploads/2026/06/Ascidian-Lilly-Partnership_Final-PR_03-June-2026.pdf

2. Other Publicly Available Information

Cover Image Source: DrugTimes Team