HQ Team
June 18, 2024: Ascidian Therapeutics, a US-based biotechnology company announced a $42 million research pact with Swiss multinational Roche AG for developing gene editing therapies.
The Boston headquartered Ascidian’s pact involves a research collaboration and licensing agreement with Roche for the discovery and development of RNA exon editing therapeutics targeting neurological diseases, according to a statement.
Ascidian will receive an initial payment of $42 million and is eligible to receive up to $1.8 billion in research, clinical, and commercial milestone payments, as well as royalties on commercial sales worldwide.
Ascidian’s RNA exon editing platform is designed to advance the therapeutic possibilities of RNA medicine and treat diseases not addressed by today’s gene editing technologies, the company stated.
Target-specific rights
Under the agreement, Ascidian will provide Roche exclusive, target-specific rights to Ascidian’s RNA exon editing technology for undisclosed neurological targets.
Genes in the genome consist of exons and introns. An exon is a region of the genome that ends up within an mRNA molecule. Some exons are coding, in that they contain information for making a protein, whereas others are non-coding.
Exons are the part of the RNA that codes for proteins.
Ascidian will conduct discovery and “certain preclinical activities” in collaboration with Roche, and Roche will be responsible for certain preclinical activities, and further clinical development, manufacturing, and commercialization.
Free to collaborate
Ascidian will be free to develop programs against other neurological targets internally or with other collaborators.
“The potential of treating disease by large-scale exon editing of RNA is vast,” said Michael Ehlers, MD, PhD, President and Chief Executive Officer of Ascidian Therapeutics.
“We look forward to working with the Roche team to develop first-in-class RNA exon editing medicines for multiple neurological diseases, with a mission and passion to relieve suffering and improve lives.”
Right cells at right time
Ascidian’s gene editing platform enables the targeting of large genes and genes with high mutational variance while maintaining native gene expression patterns and levels.
By editing exons at the RNA level, the company’s therapies enable precise post-transcriptional editing of genes, resulting in full-length, functional proteins at the right levels, in the right cells, at the right time.
Ascidian’s exon editing technology is designed to provide the durability of gene therapy, while sharply reducing risks associated with direct DNA editing and gene replacement.
