Ascidian Therapeutics and Forge Biologics Announce Manufacturing Partnership to Advance RNA Editing Therapy for Stargardt Disease

• Forge’s FUEL™ platform and cGMP manufacturing services support the production of AAV for Ascidian’s ACDN-01, the most advanced genetic therapy targeting the underlying cause of Stargardt disease
• Collaboration secures clinical supply needed to advance ACDN-01 into late-stage studies

BOSTON & COLUMBUS, Ohio--(BUSINESS WIRE)--Ascidian Therapeutics (“Ascidian”), a biotechnology company seeking to treat human diseases by rewriting RNA, and Forge Biologics (“Forge”), a leading manufacturer of gene therapies and member of the Ajinomoto Bio-Pharma Services group, today announced a strategic partnership to advance the development and manufacturing of ACDN-01, Ascidian’s lead retinal program for the treatment of Stargardt disease.

Through this collaboration, Forge is providing Ascidian with process and analytical development services, toxicology, and current Good Manufacturing Practice (cGMP) manufacturing. Ascidian is utilizing 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 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 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—the most advanced genetic therapy targeting the underlying cause of Stargardt disease—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 first-in-class approach has already demonstrated durable, 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, MD, PhD, President and Chief Executive Officer 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.”

About Ascidian Therapeutics

Ascidian Therapeutics is redefining the treatment of disease by rewriting RNA. By editing exons at the RNA level, Ascidian therapies enable precise post-transcriptional editing of genes, resulting in full-length, functional proteins at the right levels, in the right cells, at the right time. With discovery, preclinical, and clinical programs in retinal, neurological, neuromuscular, and genetically defined diseases, Ascidian’s approach has the potential to treat patients with one dose of an RNA exon editor, opening new therapeutic possibilities for patients and their families who are seeking breakthroughs. To learn more about Ascidian, visit www.ascidian.com. To learn more about the STELLAR clinical trial, visit www.StellarStargardtTrial.com.

About Forge Biologics

Forge Biologics, a member of Ajinomoto Bio-Pharma Services, is a gene therapy contract development and manufacturing organization (CDMO) enabling access to life-changing gene therapies by bringing them from concept to reality. Forge’s 200,000 square foot facility, the Hearth, is headquartered in Columbus, Ohio, and houses 20 custom-designed cGMP suites with 20,000L of bioreactor capacity. Forge’s end-to-end, scalable plasmid and AAV manufacturing services include research-grade manufacturing, process and analytical development, cGMP manufacturing, fill and finish, and integrated regulatory support to help accelerate the timelines of transformative medicines for patients with genetic diseases. To learn more, visit www.forgebiologics.com.