NEW YORK – Epicrispr Biotechnologies on Wednesday said it has raised $68 million in Series B financing to support a planned clinical trial of the company's lead program EPI-321 for the genetic neuromuscular disease facioscapulohumeral muscular dystrophy (FSHD).
The Series B funding round was led by Ally Bridge Group, a healthcare investment management firm. Other investors that participated in the funding round include SOLVE FSHD, a venture philanthropy organization founded by Lululemon Athletica's founder, Chip Wilson, who's also an FSHD patient.
South San Francisco, California-based Epicrispr will use the funding to advance EPI-321 and its broader pipeline, the company said.
Epicrispr is developing EPI-321 as a one-time gene-modulating therapy that aims to suppress abnormal expression of DUX4, a gene that's usually silenced in adult cells but is activated in FSHD. It's unknown how abnormal DUX4 activity leads to the progressive muscle degeneration characteristic of FSHD, but it's thought to influence the activity of other genes in muscle cells.
The epigenetic therapy is administered intravenously and delivered to muscle tissue using an adeno-associated virus vector.
The New Zealand Medicines and Medical Devices Safety Authority (Medsafe) recently approved a clinical trial application to initiate a first-in-human study of EPI-321, which is expected to begin this year. Investigators will evaluate the safety, tolerability, pharmacodynamics, and biological activity of a single dose of EPI-321 in adults with FSHD.
The clinical trial will be conducted in partnership with Pacific Clinical Research Network, a clinical research center in New Zealand, Epicrispr said.
"Patients have no disease-modifying therapy for this progressive disease," Richard Roxburgh, an associate professor of medicine at the University of Auckland and principal investigator for the clinical trial, said in a statement. "We look forward to advancing this clinical trial which could, with a single treatment, permanently address the disease's underlying cause."