Translational Candidate A gene editing therapy for Duchenne muscular dystrophy that permanently removes a hotspot region of patient mutations to restore dystrophin. Area of Impact Duchenne muscular dystrophy (DMD), a…
Translational Candidate We engineered a human embryonic stem cell-secreted signaling protein into a biologic for treatment of skeletal muscle disorders. Area of Impact Skeletal muscle disorders (including DM1 and sarcopenia)…
Translational Candidate Smooth muscle cell progenitors (pSMCs) differentiated from patient iPSCs which is injected into the urethral muscle to regenerate a a weak urethra. Area of Impact Surgery for urinary…
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