– $55 million Series A financing will advance the company’s robust preclinical pipeline and ongoing discovery efforts –
– Epicrispr holds exclusive license to smallest known Cas protein, enabling in vivo delivery via AAV vector to wide range of target organs –
– Lead program in facioscapulohumeral muscular dystrophy (FSHD) on track for clinical initiation in 2023 –
SOUTH SAN FRANCISCO, Calif. — July 12, 2022 — Epicrispr Biotechnologies, a biotechnology company developing ultracompact therapies to modulate gene expression in vivo, today announced its launch and Series A financing of $55 million. Epicrispr was founded by Stanley Qi, Ph.D., a renowned bioengineer and named co-inventor on the CRISPR patent held by the University of California. The company’s mission is to develop a new class of genetic medicines that act on the epigenome, a naturally occurring mechanism that determines which genes are expressed and at what levels.
Epicrispr has developed the GEMS (Gene Expression Modulation System) platform to precisely modify gene expression. GEMS includes the largest known library of novel modulators combined with advanced functional and computational genomics capabilities to rapidly design guide RNAs that are highly specific to the targeted genes.
The company additionally launches with an exclusive license from Stanford University to the ultracompact DNA-binding protein CasMINI for human use. CasMINI is the smallest Cas protein created to date, developed in the lab of Dr. Qi. It is less than half the size of Cas9 and Cas12a and is engineered to function robustly in mammalian cells. The small size of CasMINI enables delivery of Epicrispr’s therapeutic candidates in vivo via a single AAV vector to a wide range of target organs.
Epicrispr is led by chief executive officer Amber Salzman, Ph.D., a biotechnology executive with more than 30 years of experience in the pharmaceuticals industry. Alongside Dr. Salzman the company has assembled a diverse leadership team with deep scientific acumen and industry experience.
“Our vision at Epicrispr Biotechnologies is nothing short of transformative: To create a new class of genetic medicines that can treat diseases for which there’s no effective treatment today. We have a unique platform that enables in vivo delivery of our compact gene modulation components using AAV vectors that have already been de-risked in the clinic,” said Dr. Salzman. “Our lead program in FSHD has a clear line of sight to clinical entry next year, and other programs are close behind. We are excited to realize the sweeping impact epigenetic engineering could have on the standard of care for many serious diseases.”
“The platform that Epicrispr Biotechnologies has built is unparalleled in the field of epigenetic engineering. With the largest existing library of differentiated modulators and a proprietary guide design system, Epicrispr’s technology enables fine-tuning of gene expression to a degree never before possible,” said Patrick Zhang, investor at Horizons Ventures. “This promise is already visible in the company’s robust pipeline. We are eager to explore and develop the potentiality of epigenetic engineering to benefit patients in need.”
The Series A will support Epicrispr’s preclinical programs in five initial indications that are insufficiently addressed by today’s genetic medicines — Facioscapulohumeral Muscular Dystrophy (FSHD), Heterozygous Familial Hypercholesterolemia (HeFH), Alpha-1 Antitrypsin Deficiency (A1AD), Retinitis Pigmentosa 4 (RP4), and Retinitis Pigmentosa 11 (RP11) — as well as the ongoing development of the company’s platform and discovery efforts.
Epicrispr is also engaging with academic and industry partners to increase access to epigenetic engineering and ensure that the vast therapeutic potential of the GEMS platform can be explored for the benefit of patients with a wide range of conditions.
Founder and Scientific Advisors
Epicrispr Biotechnologies was founded by Stanley Qi, Ph.D., whose fundamental work in epigenetic engineering includes the invention of CRISPRa (activation) and CRISPRi (interference). Dr. Qi is a co-inventor on the University of California’s Nobel Prize-winning CRISPR patent. He is an associate professor in the Department of Bioengineering and the Department of Chemical and Systems Biology at Stanford University, where his lab has invented numerous technologies combining genome engineering with synthetic biology to probe biological processes and discover new therapeutic approaches.
Epicrispr additionally launches with a world-class Scientific Advisory Board featuring deep expertise in genetic medicine:
- David Schaffer, Ph.D., is Professor of Chemical and Biomolecular Engineering, Bioengineering, and Neuroscience at University of California, Berkeley, and a leading expert in genetic engineering
- Claire M. Fraser, Ph.D., is director of the Institute for Genome Sciences at the University of Maryland School of Medicine, and a renowned pioneer in genetic medicine
- Yang Shi, Ph.D., is Professor of Epigenetics at the Ludwig Institute for Cancer Research, University of Oxford, and a world leader in the field of epigenetics
About Epicrispr Biotechnologies
Epicrispr Biotechnologies is building the world’s leading therapeutic platform based on epigenetic engineering. The company was founded by CRISPR pioneer Stanley Qi, Ph.D., to develop ultracompact therapies to modulate gene expression in vivo. Its Series A financing was backed by Horizons Ventures and other leading investors around the globe. The company is based in South San Francisco. Visit www.epicrispr.com or follow us on Twitter and LinkedIn.
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