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Subject:  Re: Peak Gene Therapy? Date:  6/19/2019  10:34 AM
Author:  2K10 Number:  244 of 252

Rulebook #10. If a company has changed therapeutic focus more than twice and doesnt have a marketed drug and has been public more than 5 years, run.

Fair rule -- but in defense of the timelines, I would just note that with gene therapy we are in new world and approval times may stretch. We are talking about in-vivo gene editing of humans, a genetic change that could be with them for life, not a drug that will clear their system in a short time. FDA allowing testing of 1,2, 5 patients at a time. 5 year rule may not work in this space.

Another risks with the gene editing from investment standpoint, is it seems like many companies going after similar well-understood single gene issues, e.g. hemophilia, sickle cell, beta thalassemia. They may be on track to validate their platform but may not be first to market and if not best in class, may not get to market at all.
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