YTD005 / Novartis - LARVOL DELTA

Refined design of a CD117 antibody-drug conjugate safely and effectively conditions non-human primates for autologous transplant-based gene therapy (ASH 2025) - "ADC-Design:To address these issues, we developed a novel CD117-ADC (YTD005), with 4 key design features: 1st,YTD005 comprises an antagonistic anti-CD117 clone, discovered as a high affinity binder by phage displaypanning...We designed a novel CD117-ADC with 4 key features (antagonistic epitope, lack of Fc function, short half-life, and non-cell-permeable payload) to optimize both safety and efficacy. We demonstrate that a singleinfusional treatment with this ADC leads to engraftment of gene-modified cells, with mixed chimerismthat was stable up to >1 year post-infusion."

Gene therapy • Bone Marrow Transplantation • Gene Therapies • Transplantation • CD34 • KIT • THY1