Amber Bio – a biotechnology company pioneering multi-kilobase gene editing platforms to reach previously undruggable diseases – recently launched with an oversubscribed $26 million seed financing round co-led by Playground Global and Andreessen Horowitz Bio + Health. And Eli Lilly, RDF (Retinal Degeneration Fund), Hummingbird Ventures, and Pillar VC also participated in the round.
The funding round will be used to advance a first-of-its-kind RNA editing platform that enables multi-kilobase edits, allowing a single drug to treat diseases with high allelic diversity. And this holds great promise for patients who suffer from diseases that arise from diverse mutations. Amber Bio is developing its own genetic medicine programs in-house.
Amber Bio Co-founder and CTO Basem Al-Shayeb, Ph.D., was advised by Nobel Laureate Dr. Jennifer Doudna and has co-authored in top journals, including Nature, Science and Cell. And he is the inventor on 19 pending and issued patents on gene editing, and his research has been highlighted in numerous articles and was recognized in Forbes’ 30 under 30 for Science in 2021. Amber Bio 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. He 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.
KEY QUOTES:
“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.”
— Dr. Al-Shayeb
“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.”
— Dr. Borrajo
“Amber Bio is pioneering new gene editing methods that will vastly expand the range of treatable diseases and reach previously undruggable patient populations. We’re thrilled to have the opportunity to support Amber Bio, a founder-led biotech whose scientific DNA is rooted in the two academic centers that birthed modern gene editing.”
— Jory Bell, General Partner at Playground Global
“It has been a pleasure to work closely together with Jacob and Basem since day one as they have built a fast-moving, intensely creative, and yet product-focused team at Amber Bio. We are excited to continue working together as Amber advances its technology platform, internal pipeline, and ecosystem partnerships.”
— Vineeta Agarwala, MD, PhD., General Partner at Andreessen Horowitz
