Regulators yesterday approved the first therapy in the western world that can correct errors in a person’s genetic code.
Joern Aldag, chief executive of Amsterdam-based uniQure, said more such treatments would follow and argued a high price was justified because gene therapy restored natural body function and was not just a short-term fix.
The idea of treating disease by replacing a defective gene with a working copy gained credence in 1990 with the success of the world's first gene therapy clinical tests against a rare condition called severe combined immunodeficiency (SCID).
