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1/ CRISPR Therapeutics $CRSP getting whacked today after releasing a first look at phase 1 clinical results for CTX110, a cell therapy engineered with gene editing tools. A patient died in the study and it was deemed related to treatment.
But some context and nuance is needed.
But some context and nuance is needed.
2/ The patient death occurred in the highest dose cohort (DL4). While it was a small trial, half that dose yielded promising results in the form of complete responses (CR), defined here as no evidence of disease outside of lymph nodes.
3/ This is what phase 1 trials are designed to do: determine the dose level(s) to be studied in mid- and late-stage trials.
It seems unlikely for additional patients to receive DL4. That doesn't mean the same won't happen in DL3, but important to point out.
It seems unlikely for additional patients to receive DL4. That doesn't mean the same won't happen in DL3, but important to point out.
4/ There shouldn't be any unique safety concerns from using CRISPR tools to engineer cell therapies. Safety concerns would arise from the inherent safety issues of the cell types involved. CAR-T has more concerns, NK has fewer concerns, and so on.
5/ That said, one of the goals of using gene editing to engineer cell therapies is to make them safer / trigger fewer immune responses.
Today's news doesn't quite throw cold water on that goal, but it could be in the minds of analysts anyway.
Today's news doesn't quite throw cold water on that goal, but it could be in the minds of analysts anyway.
6/ The overall study results were encouraging, but it was a small study. Today's stock slide has more to do with the uninterrupted rise of $CRSP than a major concern at this time.
This will be something to watch for all first-generation CRISPR companies, however.
This will be something to watch for all first-generation CRISPR companies, however.
7/ The secret / not secret is that first-generation CRISPR tools don't work all that well in vivo (engineering cells in the body), which is why $CRSP entire clinical pipeline right now is ex vivo (engineering cells outside the body).
8/ $EDIT also has a deep pipeline of ex vivo (cell therapy) assets, although its first programs are in vivo. The latter will be more difficult to make work with first-generation CRISPR tools.
9/ Ex vivo assets will reach market first, but they still come with all known safety risks of the cell types and conditioning regimens involved. Keep that in mind as programs (both in vivo and ex vivo) near data readouts.
$CRSP $EDIT $NTLA
$CRSP $EDIT $NTLA
