Epicrispr Biotechnologies and Forge Biologics Announce AAV Development and Manufacturing Pact

Epicrispr Biotechnologies and Forge Biologics are beginning a strategic partnership for development and manufacturing of an adeno-associated virus (AAV) therapy.

The investigational AAV gene therapy, Epicrispr’s EPI-321, intends to treat facioscapulohumeral muscular dystrophy (FSHD). According to the Muscular Dystrophy Association, FSHD most affects the muscles of the face, shoulder blades, and upper arms.

EPI-321’s design silences aberrant expression of double homeobox 4, or DUX4, in skeletal muscle. When untreated, DUX4 drives progressive muscle degeneration in patients with FSHD.

AAV Services Being Deployed

Forge, which manufactures gene therapies, is lending Epicrispr AAV process development, current good manufacturing practice (cGMP) manufacturing, and analytical development. Conversely, Epicrispr—a clinical-stage gene-modulating therapy company—is leveraging Forge’s proprietary platform, with manufacturing services at Forge’s 200,000-square-foot Ohio facility.

Forge Biologics Chief Technical Officer David Dismuke, PhD, commented on the new partnership.

“As [Epicrispr was] an early development partner of our FUEL platform, we’re proud to help advance EPI-321,” Dismuke said.

Dismuke continued that Forge would “enable the scalable delivery of a potentially transformative, curative therapy for patients with FSHD.”

More on Epicrispr and Forge Interaction

Material manufactured by Forge for Epicrispr is being used to evaluate EPI-321 in a first-in-human clinical trial. While this encompasses patients from the United States, New Zealand, and Australia, the U.S. FDA has granted EPI-321 several designations. These include Orphan Drug, Fast Track, and Rare Pediatric Disease status.

“EPI-321 represents a potential first-and best-in-class therapy for FSHD,” said Epicrispr CEO Amber Salzman.

Salzman added that the therapy addresses the root cause of disease through epigenetic regulation.

“Our partnership with Forge strengthens our ability to scale manufacturing as we generate additional clinical data,” Salzman said. “We believe these data support the potential for a durable, one-time therapy for patients with this devastating disease.”

Forge Biologics is a member of the Ajinomoto Bio-Pharma Services group.