Pioneering Biotech Targets Regulatory RNA to Treat Genetic Disorders
Summary
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.
Key Points
- CAMP4 Therapeutics is targeting regulatory RNA, which plays a role in controlling gene expression, to treat diseases caused by defects in gene expression.
- The company's approach aims to restore healthy protein levels by modulating regulatory RNA using therapeutic molecules called antisense oligonucleotides (ASOs).
- CAMP4's lead drug candidate targets urea cycle disorders, and the company plans to launch preclinical studies for a candidate targeting seizure disorders with a genetic basis this year.