Companies join forces to speed production of 1-time treatment for FSHD
Epicrispr's epigenetic therapy aims to 'turn off' gene underlying MD type
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Epicrispr Biotechnologies is partnering with Forge Biologics to support the development of EPI-321, Epicrispr’s one-time epigenetic treatment candidate for facioscapulohumeral muscular dystrophy (FSHD).
The treatment uses epigenetics — chemical modifications that can alter gene activity without changing a gene’s DNA sequence — to turn off the abnormally-activated gene that underlies FSHD. According to the developer, EPI-321 is a potential curative therapy for the condition, which is marked by muscle weakness that primarly affects the face and upper body.
“EPI-321 represents a potential first-and best-in-class therapy for FSHD by addressing the root cause of disease through epigenetic regulation,” Amber Salzman, PhD, CEO of Epicrispr, said in a press release from the two companies announcing the collaboration.
“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,” Salzman said. “We believe these data support the potential for a durable, one-time therapy for patients with this devastating disease.”
FSHD is a form of muscular dystrophy marked by muscle weakness and wasting that mainly manifest in the face, shoulders, and upper arms. It is caused by mutations that lead to the abnormal activation of the DUX4 gene in muscle cells.
EPI-321 is designed to silence that gene, essentially turning it off. According to the company, the experimental therapy has “shown early signals of biological activity and clinical benefit, including improvements in strength and function” for people with FSHD.
Trial now testing EPI-321 in adults with FSHD
A Phase 1/2 clinical trial (NCT06907875) testing EPI-321 in adults with FSHD is now underway, with a main goal of evaluating the therapy’s safety. The study is still enrolling about a dozen participants, ages 18 to 75, all of whom will receive a one-time infusion of EPI-321. Participants are being recruiting at sites in the U.S., Australia, and New Zealand.
To deliver its genetic payload to cells, EPI-321 uses a so-called viral vector, which is basically a virus that has been engineered to deliver therapeutic genes instead of causing an infection. The vector used in EPI-321 is specifically derived from a virus called adeno-associated virus, or AAV, which is commonly used as a gene therapy vector because.
Under the new collaboration, Forge will be providing Epicrispr multifaceted support to manufacture the AAV vector that’s used in the therapy.
We’re proud to help advance EPI-321 and enable the scalable delivery of a potentially transformative, curative therapy for patients with FSHD.
Materials created at Forge will be put to use in the ongoing Phase 1/2 trial. Through this collaboration, Epicrispr will have access to Forge’s proprietary FUEL platform for AAV manufacturing.
“FUEL was designed to enable more efficient manufacturing, delivering more doses per run so partners like Epicrispr can reach more patients,” said David Dismuke, PhD, Forge’s chief technical officer. “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.”
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