Jondo, Did you see the last 5 minutes of Wednesday’s presentation? I try to watch and it freezes on me at 18:37 every time. If so, what does he say?
I love the CAR-T program and progress being made, but gene is where this game is played. The primates are adding up in numbers and years. That is very promising in my eyes. Milestones and CAR-T will not only carry this through but will likely be quite profitable. They only licensed 6 genes, only six. It doesn’t sound like T1DM is one of them???
DMD will be a very nice win, they haven’t said they have a gene yet but SMA has proof of concept and can now be ‘cured’ in babies. Type 1 Babies would show efficacy in weeks. Time is muscle in SMA and every day a Type 1 SMA baby goes without SMN, they lose muscle. Many never raise their heads, cry a cry or lift their arms without the new therapies. Spinraza and Vector gene are available but have drawbacks including IT administration or uncertainty about long term overproduction as their is an adult MD that is caused by over instead of under production. Vector could accumulate risk but natural production should be controlled. Arcus could be tried in utero even some day and they never would have to breath one diminished breath. Imagine how those 1000 parents would feel about never having to worry a single day each year. DMD is bigger. Not as bad, but not good at all. It is tough to watch your child slowly waste away.
aTTR is becoming an established market too. There is big money in curing MD of any kind but those three have cost determination for lesser technology. In the case of DMD, literally no proof of efficacy. The medical costs offset, the cost of current therapy, and the life years of income gained make quite a contribution to society. SMA kids have superior intellect. It is a unique correlation. One of them may just invent the hyperdrive or cold fusion.
