Epicrispr raises US$68M for pioneering FSHD treatment set for 2025 trial

USA—Epicrispr Biotechnologies, a pioneering biotechnology company located in South San Francisco, has achieved a significant milestone in the development of groundbreaking therapies by securing US$68 million in its Series B financing round.

 This substantial investment will primarily support the clinical development of EPI-321, a groundbreaking, first-in-class therapy designed to treat facioscapulohumeral muscular dystrophy (FSHD), a genetic neuromuscular disorder affecting approximately 870,000 people worldwide.

The financing round was led by Ally Bridge Group, with notable participation from SOLVE FSHD, a venture philanthropy organization founded by Chip Wilson, the founder of Lululemon Athletica and an FSHD patient himself.

Other new and existing investors also contributed to this round, underscoring the strong support for Epicrispr’s innovative approach to treating FSHD.

This funding not only propels EPI-321 toward clinical trials but also fuels the advancement of Epicrispr’s broader pipeline of therapeutic candidates.

EPI-321 represents a pioneering effort in the field of epigenetic therapies, as it is the first such treatment to enter clinical trials for a neuromuscular disease.

The therapy is designed to address the root cause of FSHD by silencing the aberrant expression of the DUX4 gene, which is incorrectly activated in patients with this condition, leading to progressive muscle degeneration.

Delivered via a clinically validated AAV vector, EPI-321 has shown promising results in preclinical studies, demonstrating robust suppression of DUX4 expression and protection of muscle tissue.

In conjunction with the funding, Epicrispr has received approval from New Zealand’s Medsafe to initiate a first-in-human trial of EPI-321.

This trial, expected to commence in 2025, will evaluate the safety, tolerability, pharmacodynamics, and biological activity of a single intravenous dose of EPI-321 in adults with FSHD.

 The study marks a crucial milestone for Epicrispr as it transitions into a clinical-stage company.

Commenting on this development, Dr. Richard Roxburgh, Associate Professor of Medicine at the University of Auckland and principal investigator for the EPI-321 clinical trial, highlighted the significance of this trial.

He noted that FSHD is one of the most common adult muscular dystrophies, with no current disease-modifying therapies available.

The potential of EPI-321 to permanently address the disease’s underlying cause with a single treatment offers hope for a new standard in therapies for genetic diseases.

Amber Salzman, Ph.D., CEO of Epicrispr Biotechnologies, emphasized the company’s focus on developing a first-in-class, one-time epigenetic therapy that targets the genetic root cause of FSHD.

With strong investor support and recent FDA designations recognizing EPI-321’s potential, the team is committed to advancing this therapy into the clinic to provide much-needed relief to patients and families affected by FSHD.

Andrew Lam, Pharm.D., Managing Director and Head of Biotech Private Equity at Ally Bridge Group, expressed pride in leading this investment, citing Epicrispr’s robust data and innovative GEMS platform as key factors in its success.

Chip Wilson, founder and Chairman of SOLVE FSHD, commended Epicrispr’s dedication to advancing EPI-321, highlighting the urgent need for treatments that target the root cause of FSHD.

Concurrent with the funding, Epicrispr has expanded its Board of Directors, welcoming Andrew Lam and Eric Crombez, M.D., Chief Medical Officer of Ultragenyx, and Jennifer King, Ph.D., former Senior Vice President of Business Development at Intellia Therapeutics.

This strategic expansion positions Epicrispr for continued success in developing innovative therapies for previously untreatable conditions.

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