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Epic Bio Reports New Data on Hypercompact Cas Proteins Optimized for Broad Therapeutic Applicability

Data being presented at ESGCT reflect improvements upon CasMINI, previously the smallest Cas protein known to work in human cells –
Company’s additional poster presentations report on progress of epigenetic editing platform and lead candidate EPI-321

SOUTH SAN FRANCISCO, Calif. — October 26, 2023 — Epic Bio, a biotechnology company developing therapies to modulate gene expression using compact, non-cutting dCas proteins, is presenting new data reflecting advances in its Gene Expression Modulation System (GEMS) platform, alongside additional preclinical data supporting the company’s lead program, EPI-321, in development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). These data are being presented at the European Society of Gene and Cell Therapy (ESGCT) 30th Annual Congress taking place October 24-27, 2023, in Brussels, Belgium.

Among the data being presented are new details on Epic’s in-house engineered suite of Cas molecules derived from CasMINI, the smallest Cas protein known to work in human cells. The company’s lead Cas effectors — dCasONYX, dCasRUBY, and dCasTOPAZ — have boosted editing activity and are each less than 500 amino acids in size, enabling them to be delivered within a single AAV. In new data at ESGCT, Epic reports that these three Cas effectors also display greater flexibility in the genetic sequences, called PAMs, that they can target. This flexibility enables epigenetic editing therapies to be targeted to an even wider range of disease-causing genes.

“As we prepare to become a clinical-stage company, we’ve also continued to build upon the powerful GEMS platform with the components necessary to bring epigenetic editing to the many diseases and patients who stand to benefit,” said Amber Salzman, Ph.D., chief executive officer of Epic Bio. “These data highlight why GEMS is an unparalleled therapeutic platform, and how our team’s unique expertise is expanding the state of the science in epigenetic editing every day.”

The ESGCT posters will be made available on the Science page of the Epic Bio website following the conference. 

About Epic Bio
Epic Bio is a leading epigenetic editing company, leveraging the power of CRISPR without cutting DNA. The company’s proprietary Gene Expression Modulation System (GEMS) includes the smallest Cas protein known to work in human cells, enabling in vivo or ex vivo delivery via a single viral vector. Epic’s lead program, EPI-321, is in IND-enabling studies for treatment of facioscapulohumeral muscular dystrophy (FSHD); additional programs seek to address alpha-1 antitrypsin deficiency (A1AD), heterozygous familial hypercholesterolemia (HeFH), and 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

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Ten Bridge Communications
(617) 903-8783

The latest from Epic

16 Nov

"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: "

7 Nov

"We are pleased to share that Epic Bio CEO Dr. Amber Salzman has been invited to present at the 2023 Stiefel Healthcare Conference. Read more here: "

31 Oct

"@Epicbiotech partners with @KitePharma to extend the potential application of Epic’s platform for cancer treatment. Epic’s approach is promising for in vivo medicines and may enable next-generation cell therapies #epigenetics #CellTherapy #Cancer "