Epicrispr Biotechnologies has secured an additional $90 million in financing to speed development of EPI-321, an experimental therapy designed to treat the rare genetic muscle disorder facioscapulohumeral muscular dystrophy (FSHD).
EPI-321 uses an emerging genetic medicine approach known as epigenetic editing. Rather than changing the underlying DNA sequence, the technology uses CRISPR-based tools to regulate whether particular genes are active or inactive. Epicrispr is applying the approach to suppress the genetic mechanism responsible for FSHD, a progressive neuromuscular disease. The company has completed enrolling participants in an early-stage clinical trial of the therapy.
Chief Executive Officer Amber Salzman described the Series C financing as an important milestone for the company as it advances EPI-321 and works toward developing additional programmable epigenetic medicines.
More on Epigenetic Editing
Epigenetic editing has attracted growing interest from researchers, biopharmaceutical companies and investors because it can modify gene activity without cutting or rewriting DNA. Supporters of the technology believe this more targeted approach could potentially offer safety advantages over conventional DNA-editing methods while expanding the number of diseases that can be addressed through genetic medicine.
In FSHD, excessive activity of the DUX4 gene drives muscle wasting and degeneration. Instead of directly modifying the gene, EPI-321 attaches to a specific region associated with DUX4 and introduces a chemical modification designed to reduce production of the protein encoded by the gene.
By suppressing DUX4, Epicrispr hopes to prevent further muscle-cell damage and preserve or improve muscle function. Preliminary clinical findings have indicated potential increases in muscle volume along with changes in biological markers associated with DUX4 suppression.
Salzman said the initial findings, although based on a small patient population and relatively short follow-up period, provide early evidence that EPI-321 may be capable of targeting the underlying biological mechanisms involved in FSHD.
The company expects to report results from six months of patient follow-up in early October.
FSHD Drug Development Landscape
Epicrispr is competing in an increasingly active FSHD drug development landscape. Several other companies are developing therapies designed to inhibit DUX4 through different mechanisms, with programs from Dyne Therapeutics, Sarepta Therapeutics, Novartis and Arrowhead Pharmaceuticals already being tested in humans.
The latest financing brings Epicrispr’s total funding since its founding to $213 million. Octagon Capital and Janus Henderson Investors jointly led the Series C, with participation from additional investors including Cormorant Asset Management, Fidelity Management & Research and Sanofi Ventures. Anran Li, an investment analyst at Octagon Capital, will also join Epicrispr’s board of directors.
Salzman said the new capital gives Epicrispr the financial resources and operational flexibility needed to continue developing EPI-321, invest in additional pipeline programs and build the company with a long-term outlook.
Joining Epicrispr in the latest wave of biotech financing, Alameda, California-based Infinimmune has raised $75 million in Series A funding to support the clinical development of two experimental treatments for atopic dermatitis. The company plans to use the new capital to advance both candidates into first-in-human clinical studies, which are expected to begin next year.
Epicrispr Raises $90M to Advance Its Pipeline
Epicrispr has secured $90 million in new funding to advance its epigenetic medicine platform and clinical development of EPI-321, an investigational treatment for facioscapulohumeral muscular dystrophy (FSHD).
The financing gives Epicrispr additional resources to progress its rare disease programs and expand research into epigenetic approaches that regulate disease-associated genes.
Epicrispr Targets FSHD With EPI-321
Epicrispr is developing EPI-321 for FSHD, a genetic muscle disorder associated with progressive muscle weakness and loss of physical function. The therapy is designed to target DUX4, a gene believed to play a central role in FSHD.
Unlike approaches that permanently alter DNA, Epicrispr is exploring epigenetic editing to regulate the activity of disease-driving genes.
