Epicrispr Biotechnologies, a South San Francisco-based biotech company, has announced two major advancements in its mission to develop transformative therapies for genetic diseases. The company secured $68 million in Series B financing and received FDA clearance for its Investigational New Drug (IND) application for EPI-321, a first-of-its-kind epigenetic therapy targeting facioscapulohumeral muscular dystrophy (FSHD). These milestones pave the way for global clinical trials of the therapy, which could offer new hope to patients suffering from this debilitating condition.
FSHD is one of the most common forms of adult muscular dystrophy, affecting up to one million people worldwide. This genetic neuromuscular disorder leads to progressive muscle weakening and degeneration. Currently, there are no disease-modifying treatments available for FSHD, leaving patients with limited options to manage symptoms.
EPI-321 is an investigational one-time gene-modulating therapy designed to silence the aberrant activation of the DUX4 gene—a key driver of muscle degeneration in FSHD. Unlike traditional treatments that focus on managing symptoms, EPI-321 targets the root cause of the disease through epigenetic reprogramming. Delivered via a clinically validated adeno-associated virus (AAV) vector, preclinical studies have shown that EPI-321 can suppress DUX4 expression and protect muscle tissue from damage.
Due to potential prior exposure to AAV, each individual must be tested for past exposure and immune response to AAV. It a person tests positive, they may not be eligible to participate in the trial participation.
The therapy has received multiple designations from the FDA, including Fast Track, Rare Pediatric Disease, and Orphan Drug status, highlighting its potential to address unmet medical needs. Amber Salzman, Ph.D., CEO of Epicrispr Biotechnologies, stated that EPI-321 represents a “potentially transformative approach” to halting disease progression in FSHD.
FDA Clearance and Global Trials
The FDA’s IND clearance allows Epicrispr to initiate clinical trials in the United States as part of a broader global development strategy. This follows approval from New Zealand’s Medsafe regulatory agency for a first-in-human trial set to begin in 2025. The Phase 1/2 trial will evaluate the safety, tolerability, and biological activity of a single intravenous dose of EPI-321 in adults with FSHD.
Dr. Russell Butterfield from the University of Utah emphasized the significance of this milestone for patients and families who have long awaited treatment options for this progressive disorder. He expressed optimism about the therapy’s innovative approach and its potential benefits.
$68 Million Series B Financing
To support the clinical development of EPI-321 and advance its pipeline of gene-modulating therapies, Epicrispr secured $68 million in Series B funding. The financing round was led by Ally Bridge Group and included participation from SOLVE FSHD—a venture philanthropy organization founded by Chip Wilson, who is both a patient with FSHD and founder of Lululemon Athletica. The funds will enable Epicrispr to transition into a clinical-stage company and further expand its research efforts.
What does this mean for people with FSHD?
This is undoubtedly an exciting milestone for the FSHD field, but people with the condition should understand that gene therapies like EPI-321 are not for everyone. The adeno-associated virus (AAV) is common and some 30% to 80% of the population has antibodies to AAV, meaning they had a prior infection. These antibodies could target EPI-321 and reduce its efficacy.
What’s more, people participating in the EPI-321 clinical trial will need to take powerful drugs to suppress the immune response both before and after the trial. These drugs increase the risk of infection and cancer, so individuals interested in participating will need to carefully weigh the risks and benefits.
In addition, people who have participated in one gene therapy trial will be ineligible to participate in future gene therapy trials or treatments that use the same viral vector because their bodies will mount an immune response upon being exposed to the second treatment.
If an effective gene therapy for FSHD reaches the market, it could be an appropriate treatment for children and younger patients who have a high chance of developing more severe weakness and disability from FSHD.
Epicrispr’s press releases
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