Gene and cell therapy

Gene therapies act by adding, replacing or editing genetic material; cell therapies treat by administering living cells, engineered or otherwise, of which CAR T-cell treatments are the best known. Both are regulated as advanced therapies, both are frequently given once rather than continuously, and both raise questions about durability that a short trial cannot answer.

Our reporting is careful with the word cure. Some of these treatments have produced results that older medicine could not, and the length of that benefit is in most cases still being measured.