Amber Bio, a biotechnology company specializing in multi-kilobase gene editing platforms for previously undruggable diseases, has raised $26 million in a seed financing round co-led by Playground Global and Andreessen Horowitz Bio + Health. Other investors participating in the oversubscribed round include Eli Lilly, RDF (Retinal Degeneration Fund), Hummingbird Ventures, and Pillar VC.
The funding will be used to advance a groundbreaking RNA editing platform that allows multi-kilobase edits, enabling a single drug to treat diseases with high allelic diversity. This has significant potential for patients suffering from diseases caused by diverse mutations. Amber Bio is developing its own genetic medicine programs in-house.
The co-founders of Amber Bio, Basem Al-Shayeb and Jacob Borrajo, both have extensive experience and expertise in the field of gene editing. Basem Al-Shayeb, Ph.D., was advised by Nobel Laureate Dr. Jennifer Doudna and has authored papers in top scientific journals.
He is also the inventor on multiple gene-editing patents. Jacob Borrajo received his Ph.D. at the Broad Institute of MIT and Harvard and has been involved in multiple biotech start-ups and projects related to gene editing.
Amber Bio aims to address the limitations of current gene editing technologies that focus on treating one mutation at a time, which is not scalable for diverse patient populations. The company’s approach leverages novel Cas-based systems for durable RNA editing, offering advantages over editing DNA, which can lead to permanent, off-target mutations.
The investors expressed excitement about supporting Amber Bio and its pioneering gene editing methods that could vastly expand the range of treatable diseases and reach previously untreatable patient populations. The company’s scientific DNA is rooted in the academic centers that were instrumental in developing modern gene editing techniques.