Ascidian Therapeutics, a bitechnology company seeking to treat human diseases by rewriting RNA, has announced a global research collaboration and licensing agreement with Eli Lilly and Company to discover and develop therapies for undisclosed monogenic kidney diseases, with the option to expand to additional targets.
Ascidian’s RNA exon editors are capable of editing multiple whole exons at the kilobase scale to repair genetic instructions causing disease. Designed to address large genes or genes with high mutational variance, Ascidian’s RNA exon editors expand the boundaries of genetic medicines.
‘Lilly and Ascidian believe that patients with serious monogenic kidney diseases deserve effective treatment options and that an RNA-based approach is a compelling strategy for those diseases’, said Michael Ehlers MD, PhD, President and Chief Executive Officer of Ascidian Therapeutics. ‘RNA exon editing gives us the ability to rewrite genes at their source, without altering DNA, opening the door to diseases long out of reach. Combined with Lilly’s genetic medicine expertise, we aim to dramatically reduce the burden of genetic kidney disease.’
Under the agreement, Lilly will receive exclusive, target-specific rights to Ascidian’s RNA exon editing technology for undisclosed kidney disease targets. Ascidian will lead discovery and selected preclinical activities, with Lilly responsible for additional preclinical work, clinical development, manufacturing, and commercialisation. Ascidian is eligible to receive up to $1.9 billion, including an upfront payment, development and commercial milestone payments, and tiered royalties on commercial sales worldwide. Ascidian retains the right to pursue other targets in the kidney independently or with additional partners.
