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Dyne is eyeing an accelerated approval filing for DYNE-251 in early 2026 that would pit the asset against Sarepta’s Exondys 51 in a patient population amenable to exon 51 skipping.

WVE-N531, an oligonucleotide, elicited significant functional benefit and reversal of muscle damage in the Phase II FORWARD-53 trial. Wave plans to file for accelerated approval of the candidate in 2026.

Ascidian Therapeutics today announced a research collaboration and licensing agreement with Roche (SIX: RO, ROG; OTCQX: RHHBY) for the discovery and development of RNA exon editing therapeutics targeting neurological diseases.

Exonate Ltd., a biotechnology company developing novel, non-invasive, small-molecule therapeutics for patients with retinal vascular diseases, announced the expansion of its Board with the appointment of Dr Rafiq Hasan as Non-Executive Director.

Ascidian Therapeutics will receive $42 million upfront from Roche, and up to $1.8 billion in milestone payments and royalties, to discover RNA exon editing candidates for neurological diseases.

Ascidian Therapeutics, a biotechnology company seeking to treat human diseases by rewriting RNA, today announced that it will share preclinical data that enabled IND clearance for Phase 1/2 testing for its lead RNA exon editing therapy, ACDN-01, in a poster session at the American Society of Gene & Cell Therapy (ASGCT) annual meeting in Baltimore, MD, May 7-11, 2024.