Gene editing biotech Amber Bio has emerged with an oversubscribed $26 million seed financing round as it builds editing platforms to reach previously undruggable diseases. The San Francisco-based company says it is developing a new RNA editing platform that can “correct genetic mutations in a safe and reversible way.”
Amber will use the funding to advance development of its RNA editing platform, which enables multi-kilobase edits, allowing a single drug to treat diseases with high allelic diversity. This would benefit patients who suffer from diseases that arise from diverse mutations. Amber Bio says it is developing its own genetic medicine programs in-house.
Amber co-founders Jacob Borrajo (CEO) and Basem Al-Shayeb (CTO) received their PhDs at the Broad Institute and UC Berkeley. Al-Shayeb was advised by Nobel Laureate Dr Jennifer Doudna. He is the inventor of 19 gene-editing patents and was recognized in Forbes’ 30 under 30 for Science in 2021.
“The development of programmable systems such as CRISPR-Cas for gene correction has driven a major biotechnological revolution in the past decade,” said Al-Shayeb. “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.”
Borrajo, previously a co-founder at two biotech start-ups, is a Y Combinator alumnus, and has worked on VLP-based delivery of CRISPR-Cas systems and novel RNA measurement technologies.
“We’re creating an entirely new ability to durably rewrite RNA, and thereby restore human health in many disease settings,” said Borrajo. “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.”
The round was co-led by Playground Global and Andreessen Horowitz Bio + Health. Eli Lilly, RDF (Retinal Degeneration Fund), Hummingbird Ventures, and Pillar VC also participated in the round.
“Amber Bio is pioneering new gene editing methods that will vastly expand the range of treatable diseases and reach previously undruggable patient populations,” said Jory Bell of Playground Global. “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.”
“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,” said Vineeta Agarwala of Andreessen Horowitz. “We are excited to continue working together as Amber advances its technology platform, internal pipeline, and ecosystem partnerships.”