Topic: phenylketonuria (pku)

Crispr Pioneer Launches Startup for Custom Gene-Editing Therapies

Aurora Therapeutics, co-founded by CRISPR pioneer Jennifer Doudna, aims to scale personalized gene-editing treatments for rare diseases using a novel FDA regulatory pathway that allows approval based on data from a small number of patients. The company's initial focus is on phenylketonuria (PKU),...