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Pioneering Biotech Targets Regulatory RNA to Treat Genetic Disorders

Pioneering Biotech Targets Regulatory RNA to Treat Genetic Disorders

Apr 18, 2025
MIT News | Massachusetts Institute of Technology

CAMP4 Therapeutics is pioneering a novel approach to treating genetic disorders by targeting regulatory RNA, which controls gene expression, using therapeutic molecules called antisense oligonucleotides to restore healthy protein levels, with lead candidates targeting urea cycle and seizure disorders.

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