Thread by @DrTiffanyL

Telomere length elongation ex-vivo in a patient with #dyskeratosiscongenita? #DC Presentation by Dr. Kasiana Myers #ASH22 1/n

Utilizing ZSCAN4 - a protein that enhances genome stability and regulates telomere elongation in EXG-001 -- a non-integrating temperature-sensitive non-transmissible Sendai virus vector. #ASH22 @KMyersBMT 2/n

@KMyersBMT ZSCAN4 is transiently (but critically) expressed during meiosis and embryonic stem cells. EXG-34217 is the non temp sensitive product we are talking about here. Hope to enroll 12 subjects! No chemo or radiation, an autologous infusion. #teamtelomere #ASH22 3/n

@KMyersBMT Important to include this knowledge (see slide) as you think about this -- especially in these patients with cancer predisposition. This makes it hard to know engraftment of treated vs untreated cells though bc its gone from the body due to temp in a few days. #ASH22 4/n

@KMyersBMT Challenging collection but successful using Plerixafor and GCSF and were able to return just under 1x10^6 treated cells. They have 9 months of follow-up since infusion in pt 1 in Feb 2022. Telomere length normalized! #ASH22 5/n

@KMyersBMT Conclusions here - safety shown in this one patient so far. There was an expected post drop in with elongation at the 6 month mark. They are still enrolling #teamtelomere #ASH22 6/6

@KMyersBMT @KMyersBMT comments on the ability to re-dose (not on this protocol so far but maybe) and there is no exclusion for 'bone marrow failure' on the study but there is exclusion for those that don't mobilize well (not enough collected and this patient had great counts). #ASH22