3 ms·
How can you say we made enormous progress when it appears this is still an untested therapy.
by postalrat 6y ago
How can you say we made enormous progress when it appears this is still an untested therapy.
- jfarlow 6y agoThe 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.