Epicrispr Secures $68M for Pioneering Epigenetic Editing Approach to FSHD Treatment

Epicrispr Biotechnologies, a San Francisco Bay Area-based biotech startup, has raised $68 million in a Series B funding round to advance its innovative epigenetic editing approach for treating facioscapulohumeral muscular dystrophy (FSHD). The company's lead program, EPI-321, aims to be the first-of-its-kind genetic medicine for this rare neuromuscular disorder.

Novel CRISPR-Based Approach Targets Root Cause of FSHD

Epicrispr's technology uses CRISPR tools to stop the errant expression of the DUX4 gene, which is implicated in FSHD. Unlike traditional gene editing approaches, Epicrispr's method focuses on turning genes on or off without directly altering DNA. This epigenetic editing strategy involves binding to a specific region of the DUX4 gene and making a chemical modification to halt protein expression.

Amber Salzman, CEO of Epicrispr, emphasized the uniqueness of their approach: "We're going after the absolute root cause of the disease. It's a really, really different approach." Preclinical tests have shown potential to impact muscle function and prevent the DUX4 protein from "seeping out," according to Salzman.

Clinical Trial and Industry Competition

Epicrispr plans to initiate a Phase 1 trial for EPI-321 in New Zealand this year. The company joins a competitive field of drugmakers targeting FSHD, with more than a dozen active DUX4-targeting drug programs currently in development. Notable competitors include Avidity Biosciences, Novartis, Arrowhead Pharmaceuticals, and Dyne Therapeutics.

The recent failure of Fulcrum Therapeutics and Sanofi's oral drug in Phase 3 testing last year highlights the challenges in developing effective treatments for FSHD. However, this setback has not deterred other companies from pursuing various approaches, including small molecule drugs and gene therapies.

Funding and Future Prospects

The $68 million Series B round was led by Ally Bridge Group and included participation from Solve FSHD, an advocacy group founded by Lululemon Athletica founder Chip Wilson. This latest funding follows Epicrispr's $55 million Series A round in 2022.

In addition to FSHD, Epicrispr is developing treatments for heterozygous familial hypercholesterolemia, alpha-1 antitrypsin deficiency, eye diseases, and undisclosed blood cancers. The company's epigenetic editing platform, developed by co-founder and Stanford researcher Stanley Qi, has the potential to address a wide range of genetic disorders.