Epicrispr Biotech Raises $68M for FSHD Epigenetic Therapy Trial
Epicrispr Biotechnologies, a pioneering biotechnology company dedicated to developing curative therapies, has announced the successful first close of its Series B financing, securing $68 million. The funds will be used to advance the clinical development of EPI-321, a first-in-class, disease-modifying epigenetic therapy for facioscapulohumeral muscular dystrophy (FSHD), a genetic neuromuscular disorder that currently lacks disease-modifying treatments.
Strategic Investment and Industry Support
The Series B financing round was led by Ally Bridge Group, a prominent life sciences investment firm, with participation from SOLVE FSHD, the venture philanthropy organization founded by Chip Wilson, the creator of Lululemon Athletica and an FSHD patient himself. Additional contributions came from new and existing investors, reflecting strong confidence in Epicrispr’s therapeutic approach and scientific advancements. The funding will not only support the upcoming clinical trial for EPI-321 but also help drive the continued expansion of the company’s broader research pipeline.
Regulatory Approval and Clinical Trial Plans
Epicrispr also announced a major regulatory milestone: New Zealand’s Medsafe has approved the company’s clinical trial application (CTA), paving the way for the first-in-human study of EPI-321. This marks the first time an epigenetic therapy is being tested in a neuromuscular disease, underscoring the groundbreaking nature of the company’s work. The clinical trial is expected to commence in 2025 and will assess the safety, tolerability, pharmacodynamics, and biological activity of a single intravenous dose of EPI-321 in adult patients with FSHD.
“FSHD is among the most prevalent adult muscular dystrophies, affecting up to one million individuals worldwide. Unfortunately, no disease-modifying therapy currently exists for this progressive disorder,” said Dr. Richard Roxburgh, Associate Professor of Medicine at the University of Auckland and principal investigator for the EPI-321 trial. The study will be conducted in collaboration with the Pacific Clinical Research Network, a premier clinical research institution in New Zealand. “We are eager to launch this clinical trial, which has the potential to permanently correct the underlying genetic cause of FSHD with a single treatment. This could redefine the therapeutic landscape for genetic diseases.”
Mechanism of Action: EPI-321 as a Novel Gene-Modulating Therapy
EPI-321 is an investigational one-time epigenetic therapy designed to silence the aberrant expression of the DUX4 gene. In individuals with FSHD, the DUX4 gene is mistakenly activated, leading to progressive muscle degeneration. By selectively suppressing DUX4 expression through epigenetic modulation, EPI-321 aims to halt or even reverse disease progression. Delivered systemically using a clinically validated AAV vector, EPI-321 has shown remarkable efficacy in preclinical studies, demonstrating robust suppression of DUX4 expression and significant muscle tissue protection.
Epicrispr’s scientific advancements have garnered recognition from the U.S. Food and Drug Administration (FDA), which has granted EPI-321 Fast Track, Rare Pediatric Disease, and Orphan Drug designations. These regulatory milestones not only validate the therapy’s potential but also facilitate its accelerated clinical development.
Executive and Investor Perspectives
Dr. Amber Salzman, CEO of Epicrispr Biotechnologies, emphasized the transformative nature of the company’s work: “We are developing a first-in-class, one-time epigenetic therapy that directly targets the genetic root cause of FSHD. Securing this Series B financing and obtaining regulatory clearance to initiate our first-in-human trial represent pivotal moments for us as we transition into a clinical-stage company. With strong investor support and key FDA designations, we are fully committed to advancing EPI-321 to provide a much-needed therapy for patients and families facing this debilitating disease.”
Investors also expressed their enthusiasm for Epicrispr’s potential. “With a robust body of data validating the effectiveness of EPI-321 and the broader GEMS platform, Epicrispr has solidified its position as a leader in epigenetic editing,” said Andrew Lam, Pharm.D., Managing Director and Head of Biotech Private Equity at Ally Bridge Group. “We are excited to lead this investment and look forward to supporting the company’s continued success as it pioneers new frontiers in genetic medicine.”
Chip Wilson, the founder and Chairman of SOLVE FSHD and an FSHD patient himself, also shared his perspective: “Living with FSHD, I understand firsthand the devastating impact of this disease and the urgency of finding effective treatments that address its root cause. Epicrispr’s commitment to developing EPI-321 gives hope to patients like me. I am proud to be part of this financing round and to help facilitate the therapy’s transition into clinical trials.”
Strengthening Leadership and Governance
With the successful Series B funding round, Epicrispr has also expanded its Board of Directors, adding key industry leaders who will bring valuable expertise in drug development, strategic growth, and commercialization. New board members include Andrew Lam, Pharm.D., of Ally Bridge Group; Eric Crombez, M.D., Chief Medical Officer of Ultragenyx; and Jennifer King, Ph.D., former SVP of Business Development at Intellia Therapeutics and an expert in rare diseases and strategic partnerships.
A Transformative Era for FSHD Treatment
Epicrispr’s progress marks a transformative era for FSHD research and treatment. With the upcoming first-in-human trial of EPI-321, the company is positioned to lead the next wave of innovation in epigenetic therapeutics. If successful, EPI-321 could establish a new standard for disease-modifying treatments in genetic neuromuscular disorders, offering patients a potentially permanent solution.
As Epicrispr transitions into a clinical-stage company, its pioneering approach to epigenetic medicine stands to redefine the treatment landscape for FSHD and beyond. With robust scientific backing, strong financial support, and regulatory momentum, the company is on a clear trajectory to revolutionize the field of genetic disease therapy.
