Epicrispr Biotechnologies Raises $68M for FSHD Therapy Trial

Epicrispr Biotechnologies, based in South San Francisco, has announced a significant achievement in its journey to develop groundbreaking therapies. The company has successfully raised $68 million in the initial close of its Series B financing round. This financial boost is aimed at advancing the clinical development of their pioneering treatment, EPI-321, designed to combat facioscapulohumeral muscular dystrophy (FSHD), a genetic disorder affecting the neuromuscular system.

The Series B financing was spearheaded by Ally Bridge Group, alongside contributions from SOLVE FSHD, a venture philanthropy organization initiated by Chip Wilson, the founder of Lululemon Athletica and an FSHD patient himself. The funding will empower Epicrispr to initiate a critical clinical trial for EPI-321 and support the expansion of the company's overall pipeline of therapies.

A significant development in Epicrispr's journey is the approval of their clinical trial application by New Zealand's Medsafe. This approval paves the way for a first-in-human trial of EPI-321, marking the inaugural clinical application of epigenetic therapy for a neuromuscular disease. Scheduled to begin in 2025, the trial will assess various parameters including the safety, tolerability, pharmacodynamics, and biological activity of a single intravenous dose of EPI-321 in adults diagnosed with FSHD.

FSHD is recognized as one of the most common muscular dystrophies affecting adults, with an estimated global patient base of up to one million individuals. Despite its prevalence, patients currently have no access to disease-modifying treatments for this progressive disorder. Dr. Richard Roxburgh, Associate Professor of Medicine at the University of Auckland, and the principal investigator for the EPI-321 trial, expressed optimism about this advancement. The trial, conducted in collaboration with the Pacific Clinical Research Network, aims to tackle the disease's underlying cause with potentially a single treatment, setting a new benchmark for genetic disease therapies.

EPI-321 stands as an investigational gene-modulating therapy aiming to silence the abnormal expression of the DUX4 gene, which is erroneously activated in FSHD, leading to muscle degeneration over time. Delivered via a clinically tested AAV vector, EPI-321 has shown significant promise in preclinical studies, demonstrating its ability to suppress DUX4 expression and protect muscle tissue. The therapy has also secured FDA Fast Track, Rare Pediatric Disease, and Orphan Drug designations, highlighting its potential impact.

Epicrispr's CEO, Amber Salzman, Ph.D., emphasized the importance of the Series B financing and regulatory approvals in transitioning Epicrispr into a clinical-stage company. With a robust investor base and recent FDA recognitions, the company remains focused on bringing EPI-321 into clinical use, aiming to offer a much-needed therapeutic solution to patients and families impacted by FSHD.

Andrew Lam, Pharm.D., Managing Director and Head of Biotech Private Equity at Ally Bridge Group, praised Epicrispr for its leadership in epigenetic editing, expressing pride in supporting the company's future endeavors. Chip Wilson, founder and Chairman of SOLVE FSHD, also acknowledged the urgent need for treatments targeting the root cause of FSHD, commending Epicrispr's efforts in advancing EPI-321.

In alignment with this financing, Epicrispr has expanded its Board of Directors by welcoming new members, including Andrew Lam from Ally Bridge Group, Eric Crombez, M.D., Chief Medical Officer of Ultragenyx, and Jennifer King, Ph.D., an expert in rare diseases and strategic partnerships.

Epicrispr Biotechnologies continues to pioneer gene-modulating therapies, targeting neuromuscular diseases with a focus on precision and durability through their proprietary Gene Expression Modulation System (GEMS). Their lead program, EPI-321, is a testament to their commitment to developing transformative treatments for conditions previously deemed untreatable.