Amber Bio, a U.S.-based biotechnology company pioneering the use of a multi-kb gene editing platform, recently launched an oversubscribed $26 million seed round co-led by Playground Global and Andreessen Horowitz Bio + Health. Eli Lilly & Company, RDF (Retinal Degeneration**) Hummingbird Ventures, and Pillar VC also participated in the round.
The funding will be used to advance a first-of-its-kind RNA editing platform that enables multi-kilobase editing, enabling a drug to ** diseases with high allele diversity. This holds great hope for patients who have developed the disease due to multiple mutations. Amber Bio is developing its own gene medicine program internally.
Co-founder and CTO Dr. Basem Al-Shayeb has been mentored by Nobel laureate Dr. Jennifer Doudna, and has published in top journals such as Nature, Science, and Cell**. He is the inventor of 19 pending and granted patents for gene editing, and his research has been highlighted in numerous articles and was named one of Forbes' 30 Under 30 People in Science in 2021. Co-founder and CEO Jacob Borrajo received his Ph.D. from MIT and Harvard's Broad Institute and was a co-founder of two biotech startups. He is an alumnus of Y Combinator and has worked on several projects in industry and academia, ranging from VLP-based CRISPR-CAS systems to novel RNA measurement technologies.
Over the past decade, the development of programmable systems such as CRISPR-CAS for gene correction has driven a major biotechnology revolution. However, there are currently more than 5,000 genetic diseases, many of which are caused by hundreds to thousands of mutations in genes – or different kinds'Typos'--Cause of disease. Current gene editing technology relies on creating solutions based on each mutation, but one mutation at a time cannot be scaled up in the highly diverse patient populations in the U.S. and globally"Dr. Al-Shayeb said.
We are creating an entirely new ability to permanently rewrite RNA to restore human health in a multi-disease setting. Our technical approach utilizes a novel, iteratively engineered CAS system for durable RNA editing, which offers significant advantages over DNA editing that can lead to permanent off-target mutations"Dr. Borrajo said.