The federal government recently awarded millions of dollars in research funding to develop gene therapies for rare diseases, on an ambitious timeline that calls for human trials in three years. One of them went to scientists at the Children’s Hospital of Philadelphia to continue their work on gene therapy that they recently used to treat a baby with a rare genetic disorder. The other award was given to GEMMABio, the gene therapy company founded by pioneer Jim Wilson, who had a three-decade-long career at the University of Pennsylvania. Wilson explained that patients with rare genetic diseases have problems or mutations in their genetic code that cause health problems, and the traditional approach has been to change the patient’s genes. However, many patients with rare diseases have unique mutations, so each patient might need their own, individualized gene therapy.