Companies in #Rare Genetic Disease

Atrium Therapeutics is a clinical-stage biopharmaceutical company developing RNA-targeted therapeutics for cardiovascular diseases. Lead program ATR 1072 is being advanced for PRKAG2 syndrome, a rare genetic cardiac condition.

Gene-editing biotech launched in January 2026 with $16 million in seed financing from Menlo Ventures, focused on N-of-1 therapies for ultrarare genetic diseases. Its initial lead program targeted phenylketonuria (PKU) before the company pivoted away from it in August 2026 amid growing competition.