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https://finance.yahoo.com/news/crispr-therapeutics-reports-p...

the highlight:
50% (2/4) complete response (CR) rate at three months in the Dose Level 3 (DL3) cohort; both responders remain in CR

the fine print:
Dose Level 4 (n=1)
One patient received Dose Level 4 of CTX110. On Day 5, the patient experienced Grade 2 CRS which resolved in 5 days. The PET/CT assessment at Day 25 showed the patient had achieved a complete response. The following day, the patient was hospitalized with febrile neutropenia and developed symptoms of short-term memory loss and confusion. The symptoms eventually progressed to significant obtundation that required intubation. He was initially treated for ICANS with steroids, anakinra and intrathecal chemotherapy without improvement. The patient was later found to have reactivation of HHV-6 and HHV-6 encephalitis and treated with antiviral therapy. The decision was made to withdraw supportive care and the patient died 52 days after CTX110 infusion.


zoinks. this is the second biotech in which "off the shelf" CART had a death due to the med. I sense a long drawn out pathway to market, if a pathway exists for the medsin their current state.
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So is it certain that this virus killing the patient was caused by the gene editing?
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No idea.

But CRSPR and vertex had been placed on a clinical hold for sickle cell before.

LogicBio was placed on hold by the FDA (Im unsure of details)

Plus abbvie ended its Editas agreement.

I thought there was another CRISPR system based related death recently, but I cant seem to find it (maybe I'm making this up)...

But there are alot of red flags here; I view CRISPR tech as a whole like "if there is smoke there is fire" ... this isnt like when PARPs were coming out and a bunch of them worked, nor checkpoint inhibitors. Hell, its not even CAR-T where a bunch work but at a big cost / high risk.
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I'm hopeful for SGMO zinc fingers tech for genetic therapies.

As you have noted, BLUE lentiglobin tech is doing well. Last I checked in with them they were doing well on hemophilia and thalassemia trials.

CRISPR seems like it has reputation / too high a risk of off-target modifications.

Allogenic off the shelf therapy is a dream and worth pursuing but seems very far off based on these results.

No position in any of these right now.
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