BOSTON — Ascidian Therapeutics and Forge Biologics announced a manufacturing partnership to support the development and production of ACDN-01, Ascidian’s lead retinal program for the treatment of Stargardt disease.
Under the agreement, Forge will provide process and analytical development, toxicology support, and current Good Manufacturing Practice manufacturing for the adeno-associated virus used in ACDN-01. The collaboration is intended to secure the clinical supply needed to advance the program into late-stage studies.
Forge is supporting the program through its proprietary FUEL platform technologies, including HEK293 suspension Ignition Cells and the pEMBR 2.0 adenovirus helper plasmid, along with program-specific optimizations designed to improve manufacturing efficiency. All development and manufacturing activities are being conducted at the Hearth, Forge’s 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 chief executive officer of Forge. “Forge’s FUEL platform technologies and manufacturing capabilities were built to enable partners like Ascidian as they advance their work through clinical development and beyond.”
Ascidian is currently evaluating ACDN-01 in the Phase 1/2 STELLAR dose-escalation trial. ACDN-01 is designed to address the underlying cause of Stargardt disease using a single AAV vector to perform in vivo RNA exon editing, restoring production of the full-length ABCA4 protein that is deficient in patients with the disease. The approach has demonstrated durable and efficient editing in both non-human primate and human retinal models.
“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, M.D., Ph.D., president and chief executive officer of Ascidian. “Partnering with Forge Biologics provides us with deep AAV expertise, scalable manufacturing capabilities, and significant capacity, enabling us to rapidly advance ACDN-01 into late-stage clinical development.”
