– Company’s non-cutting technology, engineered to be even more compact and deliverable, demonstrates synergistic gene reactivation in vitro –
SOUTH SAN FRANCISCO, Calif. — October 22, 2024 — Epicrispr Biotechnologies, a leading epigenetic editing company, presented new data on its hypercompact platform for epigenetic editing, in addition to IND-enabling data for its investigational therapy for facioscapulohumeral muscular dystrophy (FSHD), at the European Society of Gene and Cell Therapy (ESGCT) 31st Annual Congress taking place October 22-25, 2024, in Rome. Epicrispr plans to initiate clinical trials of lead candidate EPI-321 in early 2025.
Among the data presented are results of in vitro studies of a novel epigenetic modulator for gene reactivation, developed through Epicrispr’s rational protein engineering. The ability to reactivate epigenetically silenced genes is critical to fulfilling the therapeutic potential of programmable epigenetic editing. Epicrispr’s data demonstrate that the new modulator can reactivate a silenced gene to near 100% expression, with durability of more than 75 days (over 150 cell divisions).
Additionally, when the modulator is combined with the company’s previously reported epigenetic activators in a single construct, studies demonstrate powerful synergistic activity that substantially exceeds activation achieved with either component singly. This highlights the potential for GEMS therapeutics to robustly reactivate human genes to treat disease.
Full details of the posters are as follows:
Title: “Engineered ultracompact epigenetic editors for DNA and histone modifications enable durable epigenetic gene activation and suppression”
Session: Poster Session III
Presenter: Dan Hart
Date & Time: Thursday, October 24, 2:00-3:30 p.m. CEST
Poster Number: P0523
Title: “Preclinical data supporting efficacy and safety of EPI-321, an investigational epigenetic editing product treating FSHD”
Session: Poster Session I
Presenter: Siddaraju Boregowda
Date & Time: Tuesday, October 22, 7:30 to 9:00 p.m. CEST
Poster Number: P0263
Title: “Preclinical in vitro and ex vivo evaluation of EPI-321, an investigational single dose epigenome editing gene therapy, efficacy for facioscapulohumeral muscular dystrophy (FSHD) treatment”
Session: Poster Session IV
Presenter: Abhinav Adhikari
Date & Time: Thursday, October 24, 6:00 to 7:30 p.m. CEST
Poster Number: P0594
About EPI-321
EPI-321 is an investigational epigenetic therapy that aims to address the underlying molecular mechanisms of FSHD by restoring methylation to the D4Z4 region of chromosome 4 and halting toxic expression of the DUX4 gene. EPI-321 is delivered to muscle tissue within a single AAV vector (AAVrh74) which has been clinically validated for muscle delivery. Preclinical studies on EPI-321 have demonstrated its ability to robustly suppress pathological expression of the DUX4 gene and reduce muscle cell death.
About Epicrispr Biotechnologies
Epicrispr Biotechnologies 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. In addition to the lead program, EPI-321, additional programs seek to address alpha-1 antitrypsin deficiency (A1AD), heterozygous familial hypercholesterolemia (HeFH), and other indications. Visit https://epicrispr.com/ for more information or follow us on LinkedIn.
Investor Contact
Benson Cheng
benson.cheng@epic-bio.com
Media Contact
Lisa Raffensperger
Ten Bridge Communications
lisa@tenbridgecommunications.com