RARE Daily

Amber Bio Launches with $26 Million in Seed Capital for RNA Editing

August 3, 2023

Rare Daily Staff

Amber Bio, which is developing a first of its kind RNA editing platform to reach previously undruggable diseases, launched today with $26 million in seed financing.

Playground Global and Andreessen Horowitz Bio + Health co-lead the round with participation from Eli Lilly, the Retinal Degeneration Fund, Hummingbird Ventures, and Pillar VC.

The funding will be used to advance its platform, which enables multi-kilobase edits, allowing a single drug to treat diseases that arise from diverse mutations. Amber Bio is developing its own genetic medicine programs in-house.

“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,” said co-founder and chief technology officer Basem Al-Shayeb. “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 U.S. and globally.”

Co-founder and CEO Jacob Borrajo, received his doctorate 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.

Al-Shayeb, who was advised by Nobel Laureate Jennifer Doudna, has co-authored articles in top journals including Nature, Science, and Cell. He is the inventor on 19 pending and issued patents on gene-editing, and his research has been highlighted in numerous articles.

“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.”

Photo: Co-founder and CEO Jacob Borrajo and co-founder and chief technology officer Basem Al-Shayeb

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