Musings on Geron's current and future position

[biopearl123]

https://haematologica.org/article/view/8891

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7407874/

First off here are a couple of articles for those who have requested some discussion of the potential "threat" of CRISPR and CAR-t therapies. As you can see, CAR-t requires surface antigen expression on targeted cells which is why is works so well in lymphoid diseases but not so much for MPNs. We have talked about this previously. As to CRISPR I think the emphasis is on hemoglobinopathies for the near future and many pitfalls yet to overcome (discussed in depth in the articles provided). Off the shelf therapies? Not so much. Also vaccines? You can read about the problems associated there too in the articles cited. So to Geron. The recent slide deck is pretty clear. A line in the sand for ET, PV, lower risk MDS, higher risk MDS (read AML), MF. In sum virtually all of the myeloproliferative disorders at some point in their respective clinical natural histories. A pretty broad net backed up by a pretty broad manufacturing and supply plan. Its interesting that the promise of a CRISPR therapy is distant (some read outs in 2026) with tons of speculation and a skyrocketing valuation while Geron is on the cusp of top line PIII data in as little as 18 months which if positive will basically validate the entire platform since there will be further evidence for reversal of fibrosis and alteration of the disease's natural history with all of the end points we have discussed previously. They will be ready to go to market. Current competition: Constellation-currently in studies (combination) for front line--a different patient population, luspatercept--approved for R/S MDS a fraction of the MDS population with a control group that was a little loosey goosey. Rux and cousins? Therapy not likely to alter disease natural history and life expectancy by much. Looks like a line in the sand to me with a long while before other therapies catch up, if ever, due to expense and manufacturing issues (patient specific etc). The prospects of Imetelstat in combination therapy for better or worse has been held back until single agent efficacy proven beyond a shadow of a doubt, thus strengthening Imetelstat as a stand alone therapy and opening the door to much more investigation and potential benefit for the future. Regards to all, better times ahead. bp

[cheng_ho]

Moderna has a large oncology division, working on mRNA vaccines that can be customized against any cell with a mutated antigen.

[biopearl123]

Thanks Bill, so far I see work on melanoma, lymphoma and solid tumors. Did I miss something in reference to MDS/MF? If so please elaborate. Also maybe speculate on a reasonable timeline. Thanks, bp

[cheng_ho]

You'll recall that they made their SARS-CoV-2 vaccine in TWO DAYS. The only delay in getting it out was from FDA forcing them NOT to pay for trial volunteers from high-risk groups.... so they were forced to give vaccines to 35,000 mostly younger white women, thus not getting enough cases. Had they tested in inner-city nursing homes, we'd have had the vaccine in use back in May...

So the issue here is that if the mRNA vaccine works on any cancer, the process for making one for another kind of cancer is very short... literally just a day or two for actually making the product. And they won't be forced to test it on people that don't have cancer.