Lilly gains license for Ascidian’s gene-editing tech to develop kidney disease drugs

By Padmanabhan Ananthan

June 3 (Reuters) – Ascidian Therapeutics said on Wednesday it has signed a licensing deal for its gene-editing technology with pharma major Eli Lilly, worth ​up to $1.9 billion, to develop new therapies for ‌rare inherited kidney diseases.

Lilly will get exclusive rights to use the privately held biotech’s RNA exon-editing technology for certain undisclosed kidney disease targets. The technology is designed to correct faulty genes without permanently altering a ‌patient’s ​DNA.

Over the past year, Lilly has ⁠struck an array of ⁠deals to expand its presence in the genetic medicine space, including the $1.13 billion acquisition of Verve Therapeutics and partnerships with AI-first biotech Profluent and Germany-based Seamless Therapeutics.

Ascidian will receive ​up to $1.9 billion, including an undisclosed upfront amount and further payments tied to development, regulatory and sales milestones, ⁠as well as royalties on future ⁠product sales.

“We think (Ascidian’s technology) has many of the ​benefits of a traditional CRISPR or DNA editing technology, but ​with the key advantage of not actually altering the ‌patient’s DNA, only changing RNA,” Daniel Rosan, chief business officer of Ascidian, told Reuters.

CRISPR gene editing is a Nobel Prize-winning technology, which uses molecular “scissors” to trim faulty parts of genes ⁠that can then be disabled or replaced with new strands of normal DNA.

Ascidian will lead early research and some preclinical work, ⁠while Lilly will ‌handle later development, manufacturing and commercialization. Lilly ⁠may add more targets, while Ascidian retains rights ​to ‌pursue other kidney disease programs elsewhere.

“Combined with ​Lilly’s genetic ⁠medicine expertise, we aim to dramatically reduce the burden of genetic kidney disease,” said Ascidian CEO Michael Ehlers.

In 2024, Ascidian had licensed the technology to Swiss drugmaker Roche to develop therapies targeting difficult-to-treat neurological diseases.

(Reporting by Padmanabhan Ananthan in Bengaluru; Editing ​by Sahal Muhammed)