Epicrispr Biotechnologies Secures $68 Million to Advance First Epigenetic Therapy for FSHD
核心洞察
Epicrispr Biotechnologies (搜索) has raised $68 million in Series B funding to develop EPI-321, a first-in-class epigenetic therapy targeting facioscapulohumeral muscular dystrophy (搜索) (FSHD (搜索)).
New Zealand's Medsafe has approved Epicrispr's clinical trial application, with the first-in-human study of EPI-321 expected to begin in 2025 to evaluate its safety and biological activity in adults with FSHD (搜索).
EPI-321 uses CRISPR technology to silence the DUX4 (搜索) gene that drives FSHD (搜索) progression and has received FDA Fast Track, Rare Pediatric Disease, and Orphan Drug designations.
Epicrispr Biotechnologies (搜索) announced it has secured $68 million in the first close of its Series B financing to advance clinical development of EPI-321, a first-in-class epigenetic therapy for facioscapulohumeral muscular dystrophy (搜索) (FSHD (搜索)). The funding round was led by Ally Bridge Group (搜索), with participation from SOLVE FSHD (搜索), the venture philanthropy organization founded by Lululemon Athletica founder and FSHD patient Chip Wilson.
Concurrent with the financing, Epicrispr received clinical trial application (CTA) approval from New Zealand's Medsafe to initiate the first-in-human trial of EPI-321, marking a significant milestone as the first epigenetic therapy to enter clinical testing for a neuromuscular disease. The study is scheduled to begin in 2025.
Novel Approach to Treating FSHD
FSHD (搜索) affects approximately 870,000 to 1 million people worldwide and is characterized by progressive muscle weakness that typically begins in the face, shoulders, and upper arms. The disease can eventually lead to wheelchair dependence, debilitating pain, and severe fatigue. Currently, no disease-modifying therapies exist for FSHD patients.
EPI-321 represents a pioneering approach to treating this genetic disorder. Unlike traditional gene therapies that alter DNA directly, Epicrispr's technology uses CRISPR tools to make precise epigenetic modifications that silence the aberrant expression of DUX4 (搜索), the gene incorrectly activated in FSHD (搜索) that leads to muscle degeneration.
"We're going after the absolute root cause of the disease," said Amber Salzman, Ph.D., CEO of Epicrispr Biotechnologies (搜索). "It's a really, really different approach."
The investigational therapy is designed as a one-time treatment delivered systemically via a clinically validated AAV vector. In preclinical models, EPI-321 has demonstrated suppression of DUX4 (搜索) expression and protection of muscle tissue. The U.S. Food and Drug Administration has already granted the therapy Fast Track, Rare Pediatric Disease, and Orphan Drug designations.
Clinical Trial Details
The upcoming first-in-human trial will evaluate the safety, tolerability, pharmacodynamics, and biological activity of a single intravenous dose of EPI-321 in adults with FSHD (搜索). The study will be conducted in partnership with Pacific Clinical Research Network in New Zealand.
Dr. Richard Roxburgh, Associate Professor of Medicine at the University of Auckland and principal investigator for the trial, emphasized the significant unmet need: "FSHD (搜索) is one of the most common adult muscular dystrophies, with estimates of up to 1 million patients affected worldwide. But patients have no disease-modifying therapy for this progressive disease."
He added, "We look forward to advancing this clinical trial which could, with a single treatment, permanently address the disease's underlying cause, and are hopeful that it will pave the way for new standards for therapies in genetic diseases."
Competitive Landscape
Epicrispr's approach enters a field where several companies are pursuing different strategies to address FSHD (搜索). Last year, an oral drug developed by Fulcrum Therapeutics and Sanofi failed in Phase 3 testing. However, other companies including Avidity Biosciences (搜索), Novartis, Arrowhead Pharmaceuticals, and Dyne Therapeutics continue to develop DUX4 (搜索)-targeting drug programs.
According to the nonprofit FSHD (搜索) Society, there are currently more than a dozen active drug development programs targeting DUX4 (搜索). Epicrispr believes its epigenetic editing approach offers distinct advantages by addressing the genetic root cause without the risks associated with directly cutting DNA.
Leadership Expansion
As part of its transition to a clinical-stage company, Epicrispr has expanded its Board of Directors with three key appointments: Andrew Lam, Pharm.D., of Ally Bridge Group (搜索); Eric Crombez, M.D., Chief Medical Officer of Ultragenyx; and Jennifer King, Ph.D., former SVP of Business Development at Intellia Therapeutics.
"With a robust body of data validating the potential of EPI-321 and the GEMS platform broadly, Epicrispr has shown itself to be a leading epigenetic editing company," said Andrew Lam, Managing Director and Head of Biotech Private Equity at Ally Bridge Group (搜索). "We are proud to lead this investment in Epicrispr's future, and we look forward to partnering with their leadership to support their continued success."
Patient Perspective
The investment from SOLVE FSHD (搜索) highlights the personal stakes involved in developing effective treatments for this debilitating condition. Chip Wilson, founder of Lululemon Athletica and an FSHD (搜索) patient himself, has been a vocal advocate for accelerating research in this area.
"As someone living with FSHD (搜索), I know the devastating impact of this disease and the urgent need for treatments that target its root cause," Wilson stated. "We commend Epicrispr's commitment in advancing EPI-321 for FSHD and are glad to be part of this financing to support its transition to the clinic."
Broader Platform Potential
Beyond FSHD (搜索), Epicrispr is leveraging its Gene Expression Modulation System (GEMS) platform to develop treatments for multiple other conditions. The company's pipeline includes programs for heterozygous familial hypercholesterolemia, alpha-1 antitrypsin deficiency, several eye diseases, and certain undisclosed blood cancers.
Epicrispr was co-founded by Stanford researcher Stanley Qi, who previously collaborated with gene editing pioneer Jennifer Doudna at UC Berkeley. The company raised a $55 million Series A round in 2022 before securing this latest financing.
For CEO Amber Salzman, whose personal experience includes advocacy for rare disease treatments after her son and nephews were diagnosed with adrenoleukodystrophy (搜索), Epicrispr's technology represents a significant advancement in genetic medicine.
"All of a sudden, I found a company that had addressed all the limitations I'd come across in genetic medicine," she said, explaining her decision to join the company in 2021 when it was known as Epic Bio.
With the new funding and regulatory clearance to begin clinical trials, Epicrispr is positioned to potentially deliver a transformative therapy for FSHD (搜索) patients while validating its broader epigenetic editing platform.