Learning through time

Gene therapy was supposed to be easy: find a disease caused by a single gene defect and replace the gene. After the NIH showed proof-of-principle with severe combined immune deficiency (SCID) in 1990, the diseases of choice were those caused by single gene mutations: hemophilia, sickle cell anemia and cystic fibrosis.

Targeted Genetics Corp. was one of the early players in CF. It was spun out of Immunex Corp. in 1992 with an exclusive license to a gene therapy technology portfolio and rights to related gene identification and cloning, cell lines, immunological assays and certain recombinant cytokines...