26-03-2025
Epicrispr banks $68M to test epigenetic editing on rare muscle disease
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Epicrispr Biotechnologies has raised $68 million in pursuit of a first-of-its-kind genetic medicine for a rare neuromuscular disorder called facioscapulohumeral muscular dystrophy.
EPI-321, the startup's lead program, uses CRISPR tools to stop errant expression of a gene implicated in the muscle-wasting condition. Epicrispr will start a Phase 1 trial this year in New Zealand, according to the company's Wednesday statement.
The San Francisco Bay Area biotech's Series B round was led by Ally Bridge Group and involved Solve FSHD, an advocacy group formed by Lululemon Athletica founder Chip Wilson.
FSHD is a rare neuromuscular disorder estimated to affect about 870,000 people worldwide. The disease is characterized by progressive muscle weakness that begins in the face, back and upper arms and can leave people in wheelchairs or with debilitating pain and fatigue.
Though there are no available medications for FSHD, drugmakers in recent years have zeroed in on a gene called DUX4. In FSHD, a genetic error causes DUX4 to be overexpressed, eventually resulting in muscle degeneration and atrophy. Biotech companies have been working on various ways, from small molecule drugs to gene therapies, to stop that from happening.
One high-profile effort, an oral drug developed by Fulcrum Therapeutics and Sanofi, failed in Phase 3 testing last year. But other companies, including Avidity Biosciences, Novartis, Arrowhead Pharmaceuticals and Dyne Therapeutics have drugs in development as well. There are currently more than a dozen active DUX4-targeting drug programs, according to the nonprofit FSHD Society.
Epicrispr says its approach is unique among that group. The company is using CRISPR tools to turn genes on or off instead of altering DNA directly. In FSHD, it's harnessing CRISPR to bind a precise region of the DUX4 gene and make a chemical modification. The hope is doing so might stop expression of the encoded protein, without the health risks associated with cutting into DNA.
According to Amber Salzman, the company's CEO, that strategy has shown potential in preclinical tests to impact muscle function and block the DUX4 protein from 'seeping out.'
'We're going after the absolute root cause' of the disease, she said. 'It's a really, really different approach.'
Salzman has for years worked on genetic disorders as a biotech executive and patient advocate. While at GSK many years ago, her son and two nephews were diagnosed with a rare disease called adrenoleukodystrophy. She connected with prominent gene therapy researcher Jim Wilson, met several other experts in the field and started the nonprofit Stop ALD Foundation.
One of Salzman's nephews died from ALD in 2004. But her son and second nephew received a treatment that was later approved as Skysona. In the meantime, Salzman worked at multiple biotech startups, including eye gene therapy developer Adverum Biotechnologies. In 2021, a recruiter gauged her interest in leading Epicrispr, which was then known as Epic Bio.
By then, the startup had already started working on FSHD — a disease that affected her husband's family, Salzman said. That, and the potential to use epigenetic editing against a wide range of diseases, convinced her to take the job.
'All of a sudden, I found a company that had addressed all the limitations I'd come across in genetic medicine,' she said.
Epicrispr raised a $55 million Series A round in 2022. Along with FSHD, it's working on drugs for heterozygous familial hypercholesterolemia, alpha-1 antitrypsin deficiency, a pair of eye diseases and certain undisclosed blood cancers. All of its work is preclinical.
The company was co-founded by Stanford researcher Stanley Qi, who worked closely with gene editing pioneer Jennifer Doudna at UC Berkeley.
