ADARx Pharmaceuticals
San Diego, United States· Est.
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Total funding raised: $246M
Overview
A biotech leveraging engineered RNA editing to correct mutations and treat genetic diseases at the RNA level.
Genetics & GenomicsRare Disease
Technology Platform
An RNA editing platform that uses engineered ADAR enzymes and guide RNAs to make precise, single-nucleotide corrections on target mRNA transcripts.
Funding History
2Total raised:$246M
PIPE$200M
Series B$46M
Opportunities
Could provide a safer, more flexible alternative to permanent DNA editing for a wide range of genetic disorders.
Risk Factors
The technology is unproven in humans, with significant delivery, specificity, and durability challenges to overcome.
Competitive Landscape
A key player in the nascent but highly competitive RNA editing space, racing to achieve clinical proof-of-concept.