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Ascidian to receive $42 million in initial payment, and up to $1.8 billion in research,clinical and commercial milestone payments, as well as commercial royalties
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Per-target agreement enables Ascidian to pursue additional internal and collaborativeprograms within neurology and other therapeutic areas
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Combines RNA Exon Editors with next generation CNS delivery capabilities of Roche todevelop novel medicines for difficult to treat neurological diseases
BOSTON, June 18, 2024 – Ascidian Therapeutics, a biotechnology company seeking to treathuman diseases by rewriting RNA, today announced a research collaboration and licensingagreement with Roche (SIX: RO, ROG; OTCQX: RHHBY) for the discovery and development ofRNA exon editing therapeutics targeting neurological diseases.
Ascidian’s RNA exon editing platform is designed to advance the therapeutic possibilities ofRNA medicine and treat diseases not addressed by today’s gene editing technologies. Thecompany designs and develops RNA exon editing therapeutics that edit RNA exons at thekilobase scale.
Under the agreement, Ascidian will provide Roche exclusive, target-specific rights to Ascidian’sRNA exon editing technology for undisclosed neurological targets. Ascidian will conductdiscovery and certain preclinical activities in collaboration with Roche, and Roche will beresponsible for certain preclinical activities, and further clinical development, manufacturing, andcommercialization. Ascidian will receive an initial payment of $42 million and is eligible toreceive up to $1.8 billion in research, clinical, and commercial milestone payments, as well asroyalties on commercial sales worldwide. Based on the terms of the agreement, Ascidian is freeto develop programs against other neurological targets internally or with other collaborators.
“Roche is known and respected worldwide for their expertise in complex neurological diseases,and I am proud of the scientific rigor and quality of the work done at Ascidian that has led to thispartnership,” said Michael Ehlers, M.D., Ph.D., President and Chief Executive Officer ofAscidian Therapeutics. “The potential of treating disease by large-scale exon editing of RNA isvast. We look forward to working with the Roche team to develop first-in-class RNA exon editingmedicines for multiple neurological diseases, with a mission and passion to relieve suffering andimprove lives.”
“Our partnership with Ascidian is an opportunity to harness advanced RNA exon editingtechnology, which has the potential to deliver transformative one-time therapeutics by editingmultiple whole exons at the RNA level with a single treatment,” said James Sabry, M.D., Ph.D.,Global Head of Pharma Partnering at Roche.
Ascidian’s platform enables targeting of large genes and genes with high mutational variancewhile maintaining native gene expression patterns and levels. By rewriting RNA, Ascidian’sexon editing technology is designed to provide the durability of gene therapy, while sharplyreducing risks associated with direct DNA editing and gene replacement.
About Ascidian Therapeutics
Ascidian Therapeutics, an ATP company, is redefining the treatment of disease by rewritingRNA. By editing exons at the RNA level, Ascidian therapies enable precise post-transcriptionalediting of genes, resulting in full-length, functional proteins at the right levels, in the right cells, atthe right time. With discovery, preclinical, and clinical programs in retinal, neurological,neuromuscular, and genetically defined diseases, Ascidian’s approach has the potential to treatpatients with one dose of an RNA exon editor, opening new therapeutic possibilities for patientsand their families who are seeking breakthroughs. Earlier this year, Ascidian announced U.S.FDA IND clearance for the first-ever RNA exon editing candidate, ACDN-01, which targetsStargardt disease and other ABCA4 retinopathies. Ascidian is currently executing the Phase 1/2STELLAR clinical trial to evaluate the safety and efficacy of ACDN-01. For more information,visit www.ascidian.com.