Amber Bio, a biotechnology company developing multi-kilobase gene editing platforms to reach previously undruggable diseases, has launched with an oversubscribed $26 million (£20.5 million) seed financing round.
The round was co-led by Playground Global and Andreessen Horowitz Bio + Health. Eli Lilly, RDF (Retinal Degeneration Fund), Hummingbird Ventures, with participation from Pillar VC.
The funding will be used to develop a first-of-its-kind RNA editing platform which enables multi-kilobase edits, allowing a single drug to treat diseases with high allelic diversity.
The platform holds great promise for patients who suffer from diseases that arise from diverse mutations.
Co-founder and CTO Basem Al-Shayeb, Ph.D., said:
“The development of programmable systems such as CRISPR-Cas for gene correction has driven a major biotechnological revolution in the past decade.
“But there are over five thousand genetic disorders, many of which are caused by hundreds to thousands of mutations – or different kinds of ‘typos’ – in genes, leading to disease.
“Current gene editing technologies rely on creating solutions on a per-mutation basis, but treating one mutation at a time is not scalable across a patient population which can be highly diverse in the US and globally.”
Al-Shayeb was advised by Nobel Laureate Dr Jennifer Doudna and has co-authored in leading journals including Nature, Science and Cell.
He is also the inventor on 19 pending and issued patents on gene-editing, and his research has been highlighted in numerous articles and was recognised in Forbes’ 30 under 30 for Science in 2021.
Meanwhile, co-founder and CEO Jacob Borrajo, received his Ph.D. at the Broad Institute of MIT and Harvard and was previously a co-founder at two biotech start-ups.
Borrajo is a Y Combinator alumnus, and has worked on a number of projects in industry and academia ranging from VLP-based delivery of CRISPR-Cas systems, to novel RNA measurement technologies.
Dr Borrajo said:
“We’re creating an entirely new ability to durably rewrite RNA, and thereby restore human health in many disease settings.
“Our technical approach leverages novel, iteratively engineered Cas-based systems for durable RNA editing, creating significant advantages over editing DNA, which can lead to permanent, off-target mutations.”
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