Ascidian Therapeutics has appointed Murray Abramson as chief development officer. This news comes alongside the company forming its first Ophthalmology Clinical Advisory Board to guide the development of its lead RNA exon editor, ACDN-01, for Stargardt disease and other ABCA4-related retinal conditions.

Dr Abramson brings over 25 years of experience in clinical development and operations, having contributed to the approval of more than six NDAs and BLAs, including Spinraza and Tecfidera. At Ascidian, he will lead clinical strategy and execution across the company’s growing RNA-based therapeutic pipeline.

The new advisory board includes seven globally recognized retinal disease experts who will provide strategic input into clinical trial design and development for ACDN-01. The program is currently in a Phase 1/2 trial evaluating its safety and efficacy in Stargardt disease, a progressive inherited retinal disorder for which there are no approved treatments.

“Ascidian is pushing the boundaries of RNA medicine with a novel exon editing platform,” said Dr Abramson. “I’m excited to help advance this work and contribute to translating cutting-edge science into clinical solutions for patients.”

The company’s approach enables precise, large-scale RNA editing in vivo without the need to introduce foreign enzymes or make permanent changes to the genome. This modality may offer advantages over conventional gene therapies, particularly for large, mutation-rich genes such as ABCA4.

Board member Mark Pennesi, director of the inherited retinal degeneration division at the Retina Foundation of the Southwest, said: “RNA exon editing is a promising new tool for treating diseases like Stargardt, where conventional gene therapy approaches face technical limitations. I look forward to collaborating with Ascidian to help move this important work forward.”

Dr Abramson was previously SVP, head of clinical development at Precision Biosciences and has held senior roles at Tempus AI, Biogen and Merck. Before joining the pharmaceutical industry, he was on the faculty at Duke University School of Medicine and led infectious disease research at the Duke Clinical Research Institute.