The number of [nucleic-acid-delivered] gene therapies in Phase II & Phase III trials right now is huge - because of this progress in delivery [of nucleic acids]. Gene therapies for the eye, for hemophilia, for sickle cell, many many cancer therapies all rely on the ability to 'deliver' nucleic acid payloads to cells. Of those, only 3 or 4 have been approved - and all in the past 2 years, but there are a huge number that are behind that tip of the iceberg - quite precisely because it's relatively straightforward to do 'same thing but with a different sequence' once the first one works.
No comments yet.