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Only two grade 5? most level 3/4? Well...

Grade 1 Mild AE
Grade 2 Moderate AE
Grade 3 Severe AE
Grade 4 Life-threatening or disabling AE
Grade 5 Death related to AE
!!!!


so, thats concerning. I mean, these are high risk patients that are going to die regardless. Not sold in its current state, its going to be moved to a front like therapy earlier and earlier in disease course - likely for salvage therapy until more data out showing efficacy with less adverse events. Basically, monitoring risk/benefit. Near 100% chance of death in 3 months? Sure, I'll take the ___% chance the chemo kills me for a 30% chance at longer life. Contrast that to something like, say, sickle cell where average life span is in the 40s. Say youre in your 20s, but with frequent flares. What % chance of dying from gene therapy are you willing to take? If you still have 20+ years left, risk tolerance may vary widely.

It is a really risky drug for the patient. but there will be a percentage of folks that do elect to go for it, and I think the market cap for said drug company should be over 5b.

Somehow they need a treatment pathway that decreased AE's without affecting efficacy. I'm also incredibly leery of companies that suggest their drug's efficacy deepens over time (cough, NKTR, cough). But this company catches my eye.
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