Developing polytherapy drug treatments for rare genetic diseases, starting with Rett Syndrome.
Uncommon Therapeutics develops drugs targeting the underlying cause of rare diseases using latest biotechnologies including gene therapy and CRISPR. Their first indication is Rett Syndrome, a neurodevelopmental monogenetic disorder affecting 1 in 10,000 girls. The company's polytherapy approach has unlocked multiple candidate therapeutics that together could deliver a cure. Uncommon Therapeutics is a product of Uncommon Therapeutics.