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Roche partners with Ascidian Therapeutics to develop exon-editing drugs for neurological diseases
Human Gene Editing
Jun 18, 2024
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Human Gene Editing

Human Gene Editing

Jun 18, 2024

Roche partners with Ascidian Therapeutics to develop exon-editing drugs for neurological diseases

Partnerships

  • RNA-based therapeutic development company Ascidian Therapeutics has entered an agreement with global healthcare company Roche to give Roche access to Ascidian's exon-editing platform to develop drugs for undisclosed neurological targets.

  • As per the agreement, Roche will gain exclusive rights to Ascidian's RNA exon-editing technology, which the companies will develop in collaboration. Roche will continue certain preclinical studies and further clinical development, manufacturing, and commercialization. In return, Ascidian will receive a USD 42 million upfront payment, milestone payments up to USD 1.8 billion, and royalties on sales.

  • Ascidian Therapeutics is focused on leveraging its proprietary RNA exon-editing technology to target genetic diseases by precisely editing and replacing faulty exons in RNA. Its lead product, ACDN-01, is the first RNA exon editor to enter clinical trials and targets Stargardt disease, a genetic eye disorder. This technology aims to offer the benefits of gene therapy without the risks associated with direct DNA editing, ensuring that normal protein functions are maintained.​

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