- Forge’s FUEL™ platform and manufacturing services support the production of AAV for EPI-321, Epicrispr’s investigational gene therapy for FSHD
- Material manufactured at Forge for Epicrispr is being used in a clinical trial across the U.S., New Zealand, and Australia, adding to Forge’s experience in supporting clinical programs in APAC
SAN FRANCISCO & COLUMBUS, Ohio--(BUSINESS WIRE)--Epicrispr Biotechnologies (“Epicrispr”), a clinical-stage company pioneering gene-modulating therapies, 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 support the development and manufacturing of EPI-321, Epicrispr’s investigational AAV gene therapy for facioscapulohumeral muscular dystrophy (FSHD).
Through this collaboration, Forge is providing Epicrispr with AAV process development, current Good Manufacturing Practices (cGMP) manufacturing, and analytical development services. Epicrispr is also leveraging Forge’s proprietary FUEL™ platform, including its HEK293 suspension Ignition Cells™, pEMBR™ 2.0 adenovirus helper plasmid, and optimized AAVrh74 rep/cap plasmid. All manufacturing activities occur at the Hearth, Forge’s 200,000-square-foot facility in Columbus, Ohio.
Material manufactured at Forge for Epicrispr is being used in evaluating EPI-321 in a first-in-human FSHD clinical trial across the U.S., New Zealand, and Australia, adding to Forge’s experience in supporting clinical programs in the Asia-Pacific (APAC) region.
“FUEL™ was designed to enable more efficient manufacturing, delivering more doses per run so partners like Epicrispr can reach more patients,” said David Dismuke, Ph.D., Chief Technical Officer at Forge Biologics. “As an early development partner of our FUEL™ platform, we’re proud to help advance EPI-321 and enable the scalable delivery of a potentially transformative, curative therapy for patients with FSHD.”
EPI-321 is an investigational, single-dose gene-modulating therapy designed to silence aberrant DUX4 expression in skeletal muscle, which drives progressive muscle degeneration in patients with FSHD. Delivered intravenously via a clinically validated AAV vector, it has demonstrated robust DUX4 suppression and protection of muscle tissue in preclinical models. Material produced at Forge demonstrates high purity and consistent quality, with optimized capsid composition. Early clinical data from the ongoing first-in-human study demonstrate that EPI-321 had favorable clinical efficacy and was well tolerated, with no serious adverse events reported to date. EPI-321 has received Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration
“EPI-321 represents a potential first-and best-in-class therapy for FSHD by addressing the root cause of disease through epigenetic regulation,” said Amber Salzman, Ph.D., Chief Executive Officer, Epicrispr Biotechnologies. “Our partnership with Forge strengthens our ability to scale manufacturing as we generate additional clinical data showing early signs of improved muscle function and increased muscle volume following a single dose. We believe these data support the potential for a durable, one-time therapy for patients with this devastating disease.”
About EPI-321
EPI-321 is an investigational epigenetic therapy designed to treat facioscapulohumeral muscular dystrophy (FSHD), as a potential one-time curative therapy. Delivered intravenously via a clinically validated AAV vector targeting muscle tissue, EPI-321 has shown early signals of biological activity and clinical benefit, including improvements in strength and function, increased lean muscle volume on whole-body MRI, and reductions in circulating DUX4 biomarkers. At six months post-treatment, patients demonstrated gains in muscle volume versus baseline, contrasting with declines typically seen in natural history datasets. EPI-321 has received Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration, and is part of Epicrispr’s broader GEMS platform for precise gene regulation without altering DNA.
About Epicrispr Biotechnologies
Epicrispr Biotechnologies is a biotechnology company pioneering gene-modulating therapies, leading with treatments for neuromuscular diseases. The company’s proprietary Gene Expression Modulation System (GEMS) enables precise and durable control of gene expression, unlocking first-in-class treatments for previously untreatable conditions. Epicrispr’s lead program, EPI-321 is in clinical trials for FSHD, and the company is advancing additional gene-modulating therapies. Learn more at www.epicrispr.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.
Contacts
Media Inquiries
Epicrispr Biotechnologies:
Kimberly Ha
KKH Advisors
kimberly.ha@kkhadvisors.com
Forge Biologics:
Media
Marina Corleto
Associate Director, Marketing & Communications
media@forgebiologics.com
Client Development
Taleen Barsoumian
Senior Vice President, Client Development
CD@forgebiologics.com
