Epicrispr Raises $90 Million to Advance Epigenetic Therapy for FSHD

Rare Daily Staff

Epicrispr Biotechnologies has closed a $90 million oversubscribed Series C financing to fund pivotal development of its lead epigenetic therapy for facioscapulohumeral muscular dystrophy (FSHD) and expand a broader pipeline of programmable gene‑modulating medicines.

Octagon Capital and Janus Henderson Investors co-led the round with new participation from Fidelity Management & Research Company, Cormorant Asset Management, Duquesne Family Office, Sanofi Ventures, funds managed by abrdn Inc., Angelini Ventures, Readout Capital, and existing investors. Financial terms beyond the total raise were not disclosed.

Epicrispr said proceeds will support pivotal clinical studies of EPI‑321, the first clinical‑stage epigenetic therapy for FSHD, and further development of the company’s Gene Expression Modulation System (GEMS) platform and manufacturing capabilities.

Epicrispr is one of a small but growing group of companies aiming to harness epigenetic mechanisms—changes in gene expression that do not alter the underlying DNA sequence—to create durable, yet potentially reversible, therapies for genetic disease.

FSHD is a progressive neuromuscular disorder characterized by muscle weakness in the face, shoulders, and upper arms, often driven by inappropriate expression of the DUX4 gene in skeletal muscle. There are currently no approved therapies that directly target the underlying molecular cause of the disease.

Using its proprietary GEMS platform, Epicrispr designs medicines intended to selectively activate or silence disease‑driving genes. EPI‑321 is delivered via a single adeno‑associated virus (AAV) vector that has been clinically validated for muscle targeting, the company said. In preclinical studies, EPI‑321 showed robust suppression of pathological DUX4 expression and reductions in muscle cell death.

Interim data from the ongoing phase 1/2 study previously demonstrated statistically significant increases in whole‑body lean muscle volume measured by MRI, favorable changes in circulating biomarkers consistent with DUX4 suppression, improvements in strength and functional outcomes, and what the company described as a manageable safety profile.

EPI‑321 is currently being evaluated in a first‑in‑human phase 1/2 trial in FSHD. The therapy has shown a favorable safety profile and early evidence of disease modification, including statistically significant gains in lean muscle volume and biomarker changes consistent with suppression of the DUX4 gene following a single administration. Enrollment in the phase 1/2 study has been completed, with additional data expected later in 2026.

“This financing marks a pivotal milestone for Epicrispr as we advance EPI‑321 and the next generation of programmable epigenetic medicines,” said Epicrispr CEO Amber Salzman. “This financing positions us to advance EPI‑321 into pivotal studies, expand our pipeline, and continue building a new class of epigenetic therapies for patients.”

Photo: Amber Salzman, Epicrispr CEO.