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December 8, 2025Blood

Refined design of a CD117 antibody-drug conjugate safely and effectively conditions non-human primates for autologous transplant-based gene therapy

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Authors

YLYi LiuBeijing Technology and Business UniversitySSSherif SharabyUniversity of North Carolina at Chapel HillVTVictor TkachevBoston Children's Hospital

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Overview

Conditioning with a refined CD117 antibody-drug conjugate demonstrates robust engraftment in non-human primates, indicating improved safety and efficacy for gene therapy.

Key Points

  • To design and evaluate a novel CD117 antibody-drug conjugate for safe conditioning in autologous transplant-based gene therapy.
  • Developed YTD005 with four key design features to optimize safety and efficacy.
  • Administered YTD005 in Rhesus Macaques undergoing autologous gene therapy using CD34+ cells.
  • Monitored toxicity and engraftment outcomes up to one year post-transfusion.
  • YTD005 achieved median depletion of 98.2% of Lin- CD34+ hematopoietic stem and progenitor cells.
  • The conditioning regimen was well tolerated with no significant non-hematologic toxicities observed.
  • Engraftment occurred median day +13 post-transfusion with stable results observed for up to one year.

Cite This Study

Liu et al. (2025) studied this question.

synapsesocial.com/papers/69362f744fa91c937236e281https://doi.org/10.1182/blood-2025-5871
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