Eli Lilly signs US$1.9B deal with Ascidian Therapeutics for RNA kidney disease therapies

USA—Eli Lilly has strengthened its growing presence in genetic medicine through a new research and licensing agreement with Massachusetts-based biotech company Ascidian Therapeutics, a deal valued at up to US$1.9 billion.

The partnership will focus on developing innovative therapies for inherited kidney diseases using Ascidian’s proprietary RNA exon editing technology.

Targeting rare genetic kidney diseases

Under the agreement, the two companies will initially work on therapies for undisclosed monogenic kidney diseases caused by mutations in a single gene.

Lilly also retains the option to expand the collaboration to additional kidney disease targets in the future.

In return for an undisclosed upfront payment, development and commercial milestone payments, and tiered royalties, Lilly will receive exclusive rights to apply Ascidian’s RNA exon editing platform to selected kidney disease programs.

Ascidian will lead target discovery and selected preclinical activities, while Lilly will oversee additional preclinical development, clinical research, manufacturing, regulatory activities, and global commercialization.

The collaboration addresses a significant unmet medical need. More than 60 inherited kidney diseases affect millions of patients worldwide, many of whom currently have limited treatment options and face progressive kidney failure.

RNA editing technology at the center

The partnership is built around Ascidian’s RNA exon editing platform, which aims to correct disease-causing genetic instructions at the RNA level rather than permanently altering DNA.

The technology can replace multiple defective exons at the kilobase scale, potentially restoring normal protein production without introducing permanent genomic changes.

According to Ascidian President and Chief Executive Officer Michael Ehlers, the approach could unlock treatment possibilities for diseases that have remained difficult to address using traditional gene-editing technologies.

Company executives have also highlighted the potential safety advantages of RNA editing, which avoids permanent DNA modifications while delivering durable therapeutic effects.

Lilly accelerates dealmaking strategy

The Ascidian agreement is the latest addition to Lilly’s aggressive business development campaign.

It follows several high-profile transactions announced in recent months, including a licensing agreement with Hanmi Pharm for the GLP-2 agonist sonefpeglutide and a collaboration with Haisco Pharmaceutical to develop innovative therapies.

The company has also expanded its genetic medicine portfolio through acquisitions and partnerships with firms such as Verve Therapeutics, Profluent, and Seamless Therapeutics.

Alongside these partnerships, Lilly continues to invest heavily in manufacturing capabilities.

In May 2026, the company committed US$4.5 billion to expand its genetic medicines manufacturing facility at its Lebanon, Indiana campus, supporting future production of advanced therapies.

Building a broader pipeline

Lilly’s investment strategy reflects its efforts to diversify beyond its highly successful obesity and diabetes franchises.

Strong revenue growth from its weight-loss portfolio has provided the company with additional capital to pursue acquisitions, licensing agreements, and next-generation therapeutic platforms across multiple disease areas.

Beyond genetic medicine, Lilly has remained active in mergers and acquisitions throughout 2026.

Among its largest transactions this year are the US$7.8 billion acquisition of sleep-disorder specialist Centessa and a US$8.8 billion collaboration with Innovent to develop new therapies for cancer and immune-mediated diseases.

With the addition of Ascidian’s RNA exon-editing technology, Lilly continues to expand its portfolio of advanced genetic therapies while targeting diseases with few effective treatment options.