Epicrispr Biotechnologies and Forge Biologics announced on May 5 a strategic partnership to develop and manufacture EPI-321, an investigational gene therapy for facioscapulohumeral muscular dystrophy (FSHD). The collaboration involves process development, cGMP manufacturing, and analytical services provided by Forge at its Columbus, Ohio facility.
The partnership is significant as it supports the production of material for EPI-321, which is being used in a first-in-human clinical trial across the United States, New Zealand, and Australia. This adds to Forge’s experience in supporting clinical programs in the Asia-Pacific region.
Forge’s proprietary FUEL platform will be used in the project. David Dismuke, Ph.D., Chief Technical Officer at Forge Biologics said: “FUEL was designed to enable more efficient manufacturing, delivering more doses per run so partners like Epicrispr can reach more patients. 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 designed as a single-dose gene-modulating therapy that targets aberrant DUX4 expression in skeletal muscle. According to early clinical data from ongoing studies using material produced at Forge’s facility, EPI-321 has been well tolerated with no serious adverse events reported so far. Amber Salzman, Ph.D., Chief Executive Officer of Epicrispr Biotechnologies said: “EPI-321 represents a potential first-and best-in-class therapy for FSHD by addressing the root cause of disease through epigenetic regulation. 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.”
Patients treated with EPI-321 have shown encouraging signals such as improvements in strength measures and increases in lean muscle mass compared to baseline values six months after treatment. The U.S. Food and Drug Administration has granted Orphan Drug, Fast Track, and Rare Pediatric Disease designations to EPI-321.
Epicrispr continues advancing its pipeline using its Gene Expression Modulation System (GEMS) platform aimed at precise regulation of genes without permanently altering DNA.
