Amber Bio
June 24, 2026
Theater 3
Type: Company Presentation
Focus Area:
Platform for Therapeutics
Amber Bio is pioneering the next generation of RNA editing. The company is developing Splice Editors—a first-in-class RNA writing platform capable of making multi-kilobase edits, unlocking therapeutic possibilities far beyond the reach of existing modalities. Amber has already demonstrated compelling in vivo proof-of-concept with therapeutically relevant editing and tolerability in small rodents and NHPs. The team is advancing a pipeline targeting severe genetic diseases where multi-kilobase RNA rewriting is uniquely enabling. Amber Bio is advancing a lead candidate forward for USH2A correction, and has selected follow on targets across Ocular, Inner Ear, CNS and other therapeutic areas. Amber Bio is backed by a16z, Playground Global, Eli Lilly, Pillar, Hummingbird, and RD Fund.
Company HQ City:
South San Francisco
Company HQ State:
CA
Company HQ Country:
United States
Year Founded:
2021
Lead Product in Development:
USH2A
CEO
Jacob Borrajo, PhD
Development Phase of Lead Product
Pre-Clinical
Number of Unlicensed Products Looking for Licensing
1
When you expect your next catalyst update?
September 2026
What is your next catalyst (value inflection) update?
NHP Data for CNS programs
Primary Speaker
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