Supporting Clinical Trials Across Multiple Countries
Epicrispr Biotechnologies and Forge Biologics have announced a strategic partnership to develop and manufacture AAV gene therapy for EPI-321, targeting facioscapulohumeral muscular dystrophy (FSHD). This collaboration aims to advance the potential treatment for this progressive muscular disorder.
Forge is providing Epicrispr with AAV process development, current Good Manufacturing Practices (cGMP) manufacturing, and analytical development services. Epicrispr is leveraging Forge’s FUEL™ platform, including HEK293 suspension Ignition Cells™ and the pEMBR™ 2.0 adenovirus helper plasmid, to enhance production efficiency. With these advanced tools, the partnership aims to streamline the manufacturing process.
Clinical trials are underway in the U.S., New Zealand, and Australia. These efforts contribute to Forge’s growing experience in supporting clinical programmes in the Asia-Pacific region. The collaboration not only benefits Epicrispr but also strengthens Forge’s presence in international markets.
David Dismuke, Chief Technical Officer at Forge Biologics, emphasised the efficiency of the FUEL™ platform, “FUEL™ was designed to enable more efficient manufacturing, delivering more doses per run so partners like Epicrispr can reach more patients.” This statement highlights the platform’s potential to meet patient needs by increasing production capacity.
EPI-321: A Promising Gene Therapy
EPI-321, developed by Epicrispr, is an investigational gene-modulating therapy aimed at silencing DUX4 expression in skeletal muscle, a key driver of FSHD. Delivered intravenously through a validated AAV vector, preclinical models have shown robust suppression of DUX4 and muscle tissue protection. The therapy addresses the root cause of FSHD, offering hope for patients.
Manufactured material at Forge has demonstrated high purity and consistent quality, crucial for clinical success. Early clinical data indicate EPI-321 is well tolerated with no serious adverse events, showing favorable efficacy. The therapy’s promising results suggest its potential as a transformative treatment.
Epicrispr’s CEO, Amber Salzman, Ph.D., stated, “EPI-321 represents a potential first-and best-in-class therapy for FSHD by addressing the root cause of disease through epigenetic regulation.” This underscores the therapy’s groundbreaking potential in the field of gene therapy.
Notably, EPI-321 has received Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration. These designations reflect the unmet medical need for FSHD treatments and the therapy’s potential to fill this gap.
The partnership between Epicrispr and Forge Biologics aims to advance gene therapy for muscular dystrophy. By combining Epicrispr’s therapy with Forge’s manufacturing expertise, the collaboration seeks to deliver a scalable and effective treatment for patients worldwide.
