Ascidian Therapeutics, a biotechnology company seeking to treat human diseases by rewriting RNA, and Forge Biologics, a manufacturer of gene therapies and member of the Ajinomoto Bio-Pharma Services group, have partnered to advance the development and manufacturing of ACDN-01, Ascidian’s lead retinal program for the treatment of Stargardt disease.

Ascidian is currently evaluating ACDN-01 in the Phase 1/2 STELLAR dose-escalation trial. ACDN-01 uses a single AAV vector to perform in vivo RNA exon editing, restoring full-length ABCA4 protein that is deficient in Stargardt patients. This approach has already demonstrated durable, efficient editing in both non-human primate and human retinal models.

Through this collaboration, Forge is providing Ascidian with process and analytical development services, toxicology, and current Good Manufacturing Practice (cGMP) manufacturing. Ascidian will utilize Forge’s proprietary FUEL technologies, including HEK293 suspension Ignition Cells and pEMBR 2.0 adenovirus helper plasmid, along with program-specific optimizations designed to drive manufacturing efficiencies. All development and manufacturing activities will take place at the Hearth, Forge’s 200,000-square-foot gene therapy development and manufacturing facility in Columbus, Ohio.

“We are proud to partner with Ascidian Therapeutics on this program, which reflects our shared commitment to bringing together scientific and manufacturing innovation to support patients,” said John Maslowski, President and CEO of Forge.

“ACDN-01 represents a fundamentally new approach to treating diseases driven by large, complex genes like ABCA4 which have historically been difficult to address with traditional gene therapies,” said Michael Ehlers, MD, PhD, President and CEO of Ascidian. “Partnering with Forge Biologics equips us with deep AAV expertise, scalable manufacturing capabilities, and significant capacity, enabling us to rapidly advance ACDN-01 into late-stage clinical development.”