- Ascidian Therapeutics and Forge Biologics have formed a strategic partnership to support development and manufacturing of ACDN-01 for Stargardt disease.
- Forge will provide process development, analytical services, toxicology support and cGMP manufacturing at its Columbus, Ohio facility.
Ascidian Therapeutics has entered into a strategic partnership with Forge Biologics to advance the development and manufacturing of ACDN-01, its lead retinal programme for the treatment of Stargardt disease. The agreement brings together Ascidian’s RNA editing technology with Forge’s gene therapy manufacturing capabilities.
Under the collaboration, Forge will provide process and analytical development, toxicology support and current Good Manufacturing Practice (cGMP) manufacturing services. The work will support Ascidian’s clinical development activities for ACDN-01 and forms part of Forge’s CDMO and contract manufacturing offering for gene therapy developers.
Ascidian is using Forge’s proprietary FUEL™ technologies, including HEK293 suspension Ignition Cells™ and the pEMBR™ 2.0 adenovirus helper plasmid, alongside programme-specific optimisations intended to improve manufacturing efficiency. 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.
ACDN-01 is currently being evaluated in the Phase 1/2 STELLAR dose-escalation trial. The therapy uses a single AAV vector to perform in vivo RNA exon editing to restore full-length ABCA4 protein, which is deficient in patients with Stargardt disease. The company states that the approach has demonstrated durable and efficient editing in non-human primate and human retinal models.
“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.”
Michael Ehlers, President and Chief Executive Officer of Ascidian Therapeutics
