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Our technology

GeneToBe is pioneering gene therapies including CRISPR Cas9 gene editing and targeted gene silencing — offering a transformative approach to repair the underlying basis of genetically-defined maladies.

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Gene silencing via therapeutic siRNAs

Precision therapy in
defined patient segments

  • GeneToBe leverages a systematic and advanced platform to create liver-selective siRNAs

  • Selection of targets and design of optimized siRNAs to accelerate bench-to-bedside translation

  • Bias targeting: allows intervention against pathogenic variants to preserve normal physiological function of wild-type forms

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Gene editing via miCas9 platform technology

Patented MiCas9 yields improved
gene editing with higher fidelity

  • MiCas9 efficiency is driven by Brex27, which favors recruitment and stabilization of RAD51

  • Enhances homology directed repair efficiency

  • Improves genome editing specificity and precision

  • Platform amenable for use in many diseases/therapeutic areas

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Ma, L., Ruan, J., Song, J. et al. MiCas9 increases large size gene knock-in rates and reduces undesirable on-target and off-target indel edits. Nat Commun 11, 6082 (2020).

GeneToBe’s Animal Models of Rare Genetic Diseases

At GeneToBe, our scientists have developed genetically-modified rabbit models for Cystic Fibrosis and Usher Syndrome to more accurately replicate human disease conditions. These models are important for establishing proof of concept, enhancing translational relevance, and refining therapeutic discovery and development in our current programs. Our scientists have the technology to create novel rabbit models for other rare genetic diseases. 

  • Translational endpoints to enable clinical development

  • Use of matched clinical techniques

  • Detailed structure and function analysis

  • Deep molecular evaluation of biological samples

Preclinical Models of Human Disease​

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