I would like to see a partnership deal on TRegs for these big indications. IMO, the partnership should be structured similar in structure to how HemA was structured. The HemA trial, with Pfizer, pushed forward at a very aggressive pace compared to all other trials going on by SGMO. Why? How can we harness this type of pace on future indications ? Competitive lead (BMRN) I suspect (at the time).
Certainly, Fabry does not measure up (which should have dosed first patient in Oct 2019, if they were operating on same relative timelines as Hema, ~8 months from IND to first dose). If they had dosed on same relative timeline then we would actually have at least one patient with 2 year data (even if lowest dose cohort). Note the IND was roughly 1 year prior to COVID.Fabry's utilization of the same AAV that HemA uses should actually have helped accelerate the Fabry timeline or at a minimum helped it keep a consistent timeline. I'm not saying that SGMO management is not doing what they were guiding too here, I'm saying that their pace of progress pursuing this indication is significantly different then HemA. Why ?
How do we move forward safely and purposely without having to wait for competitors to fire up anew and possibly surpass SGMO with anything in the SGMO pipeline?
Another example is SCD (BC11A enhancer) trial (allong with BT). We started pursuing fetal globin production (BC11A) while Bluebird only had their adult hemoglobin trial in progress. We even had the experience of ex-vivo edits with HIV trials. But, somehow Bluebird pretty much has been able to pretty much fail on their adult hemoglobin trial and have been able to fire up and swtich gears into a fetal globin SCD trial, and it seems is about in the same place we are...
Back to TRegs. I would like to see SGMO get back to a more assertive progress oriented timeline, with a partner and plan similar to how they worked HemA with Pfizer. JMO.