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 (TCT-ASTCT-CIBMTR 2026) - "YTD005 incorporates rational design features to optimize safety and efficacy. A single infusional treatment with this ADC leads to engraftment of gene-modified cells, with mixed chimerism stable up to >1-year post-infusion. 1."

Gene therapy • Gene Therapies • Hematological Disorders • Transplantation • CD34 • KIT • THY1

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