Epicrispr Biotechnologies, a clinical-stage biotechnology company developing programmable epigenetic medicines, has raised $90 million in an oversubscribed Series C financing round to advance its lead clinical program for facioscapulohumeral muscular dystrophy (FSHD) and expand its broader therapeutic pipeline.
The financing was co-led by Octagon Capital and Janus Henderson Investors, with 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.
Epicrispr said the proceeds will primarily support development of EPI-321, its lead clinical candidate currently being evaluated in first-in-human studies for FSHD. The company also plans to use the funding to accelerate additional programmable epigenetic medicine programs, expand its proprietary Gene Expression Modulation System (GEMS) platform and strengthen its manufacturing capabilities.
The company is developing EPI-321 as a potential disease-modifying treatment for FSHD, a genetic muscle disorder associated with progressive muscle weakness and loss of muscle function. Epicrispr’s approach is designed to regulate disease-associated gene expression without permanently modifying the underlying DNA sequence.
According to the company, EPI-321 has demonstrated a favorable safety profile in its ongoing first-in-human clinical trial, alongside early signals that the treatment could modify disease-related biology. Following a single administration, the company reported statistically significant increases in lean muscle volume and biomarker changes consistent with suppression of DUX4, a gene closely associated with the development of FSHD.
Enrollment in the EPI-321 Phase 1/2 trial has now been completed. Epicrispr expects to report additional clinical data later this year, which could provide further insight into the candidate’s safety, biological activity and potential therapeutic benefit.
The latest financing is expected to help the company prepare EPI-321 for later-stage development and potentially pivotal studies. Epicrispr also intends to use the capital to broaden its pipeline of programmable epigenetic medicines targeting serious genetic diseases.
Amber Salzman, Ph.D., CEO of Epicrispr Biotechnologies, described the Series C financing as an important milestone for the company as it moves EPI-321 through clinical development and works to establish programmable epigenetic medicines as a new therapeutic modality.
Epicrispr’s technology is based on its proprietary GEMS platform, which is designed to selectively activate or silence specific genes involved in disease. Unlike approaches that permanently modify DNA, programmable epigenetic medicines aim to alter gene expression while leaving the underlying genetic sequence intact.
The company believes this strategy could have applications across a broad range of serious genetic disorders. By controlling gene activity rather than changing the DNA sequence itself, Epicrispr aims to develop therapies capable of producing durable changes in disease biology while offering a potentially differentiated approach to genetic medicine.
Investor interest in the financing reflects growing attention toward programmable approaches to gene regulation. Octagon Capital said Epicrispr had demonstrated the ability to move its technology from platform development into clinical testing in a relatively short period, highlighting the early clinical data generated by EPI-321.
As part of the financing, Anran Li, Ph.D., of Octagon Capital will join Epicrispr’s Board of Directors. The appointment adds further experience from the investment and biotechnology sectors as the company prepares for the next stage of its development.
EPI-321 currently represents the company’s most advanced program and is described by Epicrispr as the first clinical-stage epigenetic therapy being developed for FSHD. Beyond the lead program, the company is advancing additional candidates across multiple therapeutic areas using its GEMS platform.
With the new $90 million financing, Epicrispr has additional resources to progress EPI-321 toward pivotal development, generate further clinical evidence and expand its pipeline. The upcoming clinical data from the Phase 1/2 study will be an important milestone as the company seeks to demonstrate whether targeted, programmable control of gene expression can translate into meaningful disease modification for patients with FSHD.