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Epicrispr lands $90M to advance epigenetic editing drug for rare muscle disease
['Delilah Alvarado']
BioPharma Dive - Latest News
Epicrispr Biotechnologies has raised another $90 million to accelerate development of a first-of-its-kind treatment for a rare genetic muscle disorder.
With EPI-321, Epicrispr is using this strategy to silence the genetic driver of facioscapulohumeral muscular dystrophy, or FSHD, a progressive neuromuscular condition.
In FSHD, for instance, the overexpression of a gene called DUX4 causes muscle atrophy and degeneration.
Rather than cut into that gene, EPI-321 binds to a specific area of DUX4 and makes a chemical modification that suppresses production of its encoded protein.
Early clinical data have shown the potential to boost muscle volume and impact biological markers associated with DUX4 suppression.