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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& #39;t mean the same won& #39;t happen in DL3, but important to point out.
It seems unlikely for additional patients to receive DL4. That doesn& #39;t mean the same won& #39;t happen in DL3, but important to point out.
4/ There shouldn& #39;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& #39;s news doesn& #39;t quite throw cold water on that goal, but it could be in the minds of analysts anyway.
Today& #39;s news doesn& #39;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& #39;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& #39;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.
