– Epicrispr’s non-cutting technology to modulate the epigenome advances with the addition of new hypercompact Cas effectors and potent gene activators –
SOUTH SAN FRANCISCO, Calif. — August 17, 2023 — Epicrispr Biotechnologies, a biotechnology company developing therapies to modulate gene expression using compact, non-cutting dCas proteins, today presented new data on its epigenetic editing platform at Cold Spring Harbor Laboratory’s Genome Engineering: CRISPR Frontiers conference, being held August 16-20, 2023, in Cold Spring Harbor, NY.
Epicrispr’s six poster presentations detail a variety of discoveries that expand upon the capabilities of the company’s powerful Gene Expression Modulation System (GEMS) platform. Specifically, Epicrispr is presenting the first disclosures of the results of their in-house protein engineering efforts to improve upon CasMINI, the smallest Cas protein known to work in human cells. Epicrispr generated novel Cas effectors even smaller than CasMINI, under 500 amino acids in size, and with improved epigenetic editing activity. These hypercompact CRISPR components can enable packaging within a single AAV and delivery of multiple modulators simultaneously.
Additionally, Epicrispr will highlight new research identifying combinations of epigenetic activators that display synergistic activity, increasing gene expression more than the sum of their parts. Epicrispr screened hundreds of transcription-activating peptides for robust combinatorial activity, then applied machine learning to predict combinations with the greatest effect on gene expression. These insights will allow Epicrispr to design multiplexed GEMS constructs with improved activity.
Full details of the posters are as follows. The presentations will be made available under the publications section of the Science page on the Epicrispr Biotechnologies website.
Title: “Discovery and engineering of hypercompact transcriptional modulators for robust and durable target gene activation”
Poster #: 71
Date & Time: Thursday, August 17, 2:00-4.30 p.m. ET
Title: “EPI-321 — A promising CRISPR epigenome engineering therapy for facioscapulohumeral muscular dystrophy (FSHD)”
Poster #: 80
Date & Time: Thursday, August 17, 2:00-4.30 p.m. ET
Title: “Combinatorial screening of transcriptional activation domains for improved activity provides insights into biophysical properties of strong activators”
Poster #: 100
Date & Time: Thursday, August 17, 2:00-4.30 p.m. ET
Title: “Gene Expression Modulation Systems (GEMs): Optimized CRISPR-based epigenome editing platform for versatile epigenome modulation”
Poster #: 108
Date & Time: Thursday, August 17, 2:00-4.30 p.m. ET
Title: “Accelerating protein engineering with machine learning: A few-shot transfer learning approach to designing novel gene activators”
Poster #: 117
Date & Time: Thursday, August 17, 2:00-4.30 p.m. ET
Title: “Engineering of a compact CRISPR-Cas ribonucleoprotein complex for epigenome editing”
Poster #: 215
Date & Time: Friday, August 18, 7:30-10:30 p.m. ET
About Epicrispr Biotechnologies
Epicrispr Biotechnologies is a leading epigenome editing company, leveraging the power of CRISPR without cutting DNA. The company is using its proprietary Gene Expression Modulation System (GEMS) to develop therapies. Through Epicrispr’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. Epicrispr’s lead program, EPI-321, is in IND-enabling studies for treatment of facioscapulohumeral muscular dystrophy (FSHD); additional programs in preclinical development 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 www.epicrispr.com for more information or follow us on Twitter and LinkedIn.
Investor Contact
Shawn M. Cox
Epicrispr Biotechnologies
Manager, Investor Relations and Corporate Communications
shawn.cox@epic-bio.com
Media Contact
Lisa Raffensperger
Ten Bridge Communications
lisa@tenbridgecommunications.com
(617) 903-8783