Roche Partners with Ascidian Therapeutics to Develop Gene Therapies

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Roche Partners with Ascidian Therapeutics to Develop Gene Therapies

June 18, 2024 – Swiss pharmaceutical giant Roche has announced a partnership with Boston-based biotech startup Ascidian Therapeutics to develop gene therapies targeting hard-to-treat neurological diseases. This collaboration, revealed on Tuesday, is part of Roche’s broader strategy to diversify its therapeutic portfolio amid declining oncology sales.

Key Points of the Partnership:

Technology and Focus: Roche will gain exclusive rights to Ascidian’s RNA exon editing technology, which is aimed at developing treatments for undisclosed neurological conditions. This technology focuses on editing RNA to replace mutated gene sections with healthy ones, offering a long-lasting therapeutic effect with fewer risks compared to traditional gene editing.
Financial Terms: Ascidian will receive an upfront payment of $42 million. Additionally, the company could earn up to $1.8 billion through research, clinical, and commercial milestone payments, along with royalties on global sales of any resulting therapies.
Strategic Importance: This collaboration is a significant step for Roche as it seeks to rejuvenate its development pipeline following setbacks in Alzheimer’s and cancer immunotherapy trials in 2022. The partnership reflects Roche’s commitment to investing in innovative technologies to address unmet medical needs.

Background on Ascidian Therapeutics:

Ascidian is currently conducting an early-stage trial for a genetic eye disease therapy based on its RNA exon editing technology. The company, backed by venture capital firm Apple Tree Partners, raised $40 million in its most recent funding round in November 2023. The therapy candidate, ACDN-01, exemplifies Ascidian’s approach to gene therapy, aiming for a safer and more effective treatment method compared to direct DNA editing.

Expert Insight: “Our partnership with Ascidian is an opportunity to harness advanced RNA exon editing technology, which has the potential to deliver transformative one-time therapeutics by editing multiple whole exons at the RNA level with a single treatment,” said James Sabry, global head of pharma partnering at Roche.

This strategic move by Roche underscores its dedication to expanding into new therapeutic areas and leveraging cutting-edge technologies to create impactful treatments for complex diseases.