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Epic Bio Presents Preclinical Data on EPI-321 for Facioscapulohumeral Muscular Dystrophy at ASGCT 26th Annual Meeting

  • Oral presentation highlights EPI-321 effects on multiple clinically relevant markers
  • IND application for EPI-321 planned for 2023

SOUTH SAN FRANCISCO, Calif. — May 19, 2023 — Epic Bio, a biotechnology company developing therapies to modulate gene expression using compact, non-cutting dCas proteins, today presented promising preclinical data supporting development of EPI-321 for the treatment of facioscapulohumeral muscular dystrophy (FSHD). The data were shared in an oral presentation at the 26th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) taking place May 16-20, 2023, in Los Angeles, Calif.

“We’re proud to share the preclinical data from our lead program, EPI-321, in an oral presentation at the distinguished ASGCT meeting,” said Alexandra Collin de l’Hortet, Ph.D., head of therapeutics at Epic Bio. “We chose FSHD as our lead indication in recognition of the serious unmet need for a therapy that could address the underlying epigenetic cause of the disease. EPI-321’s observed effects on multiple clinically relevant disease measures provide promising direction as we work to translate these findings into the clinic.”

Data being presented demonstrate that a single administration of EPI-321 robustly suppresses pathological expression of the DUX4 gene pathway in multiple FSHD patient-derived myocyte cell lines, as well as in a humanized mouse model of the disease. Additional in vitro studies showed that administration of EPI-321 protected patient-derived myocytes from the high levels of cell death that are characteristic of FSHD. Finally, in a humanized mouse model of FSHD, EPI-321 administration led to a 55 percent increase in the survival of skeletal muscle cells.

“This validation of our GEMS platform’s ability to produce potentially transformative therapies is very exciting,” said Amber Salzman, Ph.D., chief executive officer of Epic Bio. “We are highly focused on progressing EPI-321 toward the clinic with the goal of finally providing people with FSHD a disease-modifying therapeutic option.”

Epic Bio plans to file an IND for EPI-321 later this year and to initiate a first-in-human clinical trial in 2024. The presentation will be made available under the publications section of the Science page on the Epic Bio website.

About Epic Bio
Epic Bio is a leading epigenome engineering company, leveraging the power of CRISPR without cutting DNA. The company is using its proprietary Gene Expression Modulation System (GEMS) to develop therapies. Through the company’s library of the most compact Cas DNA-binding proteins to work on human cells, the company is developing in vivo therapies with delivery via a single AAV vector. Epic Bio has an initial focus on facioscapulohumeral muscular dystrophy (FSHD) and is conducting additional research to address alpha-1 antitrypsin deficiency (A1AD), heterozygous familial hypercholesterolemia (HeFH), as well as other indications. The company is financially backed by Horizons Ventures and other leading investors. Visit for more information or follow us on Twitter and LinkedIn.

Investor Contact

Shawn M. Cox
Epic Bio
Manager, Investor Relations and Corporate Communications

Media Contact

Lisa Raffensperger
Ten Bridge Communications
(617) 903-8783

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"The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to EPI-321 for the treatment of facioscapulohumeral muscular dystrophy (FSHD), the most common form of muscular dystrophy in adults. Read more here: "

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