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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 LiuSSSherif SharabyVTVictor Tkachev

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Overview

Observational analysis demonstrates safe CD117 antibody-drug conjugate effectively conditions Rhesus macaques for gene therapy, indicating improved transplantation outcomes.

Key Points

  • Engraftment of transgene-modified cells was achieved with a mean vector copy number of 0.082 at Day +30.
  • Pancytopenia was observed in all recipients, with neutrophils reaching a nadir of 100/mL at median day +9.
  • Rhesus macaques underwent hematopoietic stem cell transplant after conditioning with a novel antibody-drug conjugate design.
  • Potential benefits of this approach include enhanced safety and efficacy in gene therapy settings, suggesting broader application.

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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