SOUTH SAN FRANCISCO, Calif. – October 13, 2022 – Epicrispr Biotechnologies, a biotechnology company developing therapies to modulate gene expression using compact, non-cutting dCas proteins, today announced a presentation on the company’s unique engineering platform, called the Gene Expression Modulation System (GEMS), at the co-hosted Solebury / BMO Fall Private Company Showcase.
“We are excited to show the scientific community a new approach to treating genetic and non-genetic diseases. Our dCas proteins anchor our GEMS platform and have been uniquely designed to work in tandem with our customized library of modulators (gene activators and suppressors),” said Amber Salzman, Ph.D., Chief Executive Officer of Epicrispr Biotechnologies. “I am excited by the data we will present today on Facioscapulohumeral Muscular Dystrophy (FSHD), the second most prevalent form of muscular dystrophy. Notably, our in vitro results demonstrate the utility of our dCas to suppress DUX4 expression by up to 95%. We view this to be a tremendous step forward to finding a one-and-done treatment for this debilitating disease. Following on from this, we have submitted a request to the FDA for a pre-IND meeting to align on the IND submission package and Phase 1 clinical design.”
Epicrispr Biotechnologies’ GEMS platform integrates its dCas proteins with customized guide RNA and modulator proteins to develop effective and safe approaches to treat diseases. Epicrispr Biotechnologies’ dCas proteins are half the size of standard Cas9 proteins, thus enabling Epicrispr Biotechnologies to deliver single AAV vectors to all tissues and organs.
About FSHD
Facioscapulohumeral Muscular Dystrophy is estimated to be the second most common muscular dystrophy, affecting 1 in 8,000 patients1,2. The genetic muscle disorder primarily affects the face, scapula, and the humerus region. The loss of movement is accompanied with chronic pain, anxiety, and depression. Symptoms usually start before age 20 and may lead to being wheelchair-bound by age 50. There are no treatment options for FSHD, and patients manage symptoms through the use of assistive devices, physical therapy, and orthopedics. For more information on FSHD, please visit www.epicrispr.com/pipeline.
About Epicrispr Biotechnologies
Epicrispr Biotechnologies is a leading epigenetic 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. Epicrispr Biotechnologies has an initial focus on Facioscapulohumeral Muscular Dystrophy (FSHD) and is conducting additional research to address 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 www.epicrispr.com for more information or follow us on Twitter and LinkedIn.
1 Cohen J, et al. Trends Mol Med. 2021 Feb; 27(2):123-137
2 Schatzl T, et al. Orphanet J Rare Dis. 2021 Mar; 16(129)
Contacts
Investor Contact
Jessica Vo
Gilmartin Group, LLC
jessi@gilmartinir.com
Media Contact
Lisa Raffensperger
Ten Bridge Communications
lisa@tenbridgecommunications.com
(617) 903-8783