ABOUT

R&D

CARE™, Cell-specific AAV Research Engine

CARE™ explores novel AAV vectors with enhanced target specificity and transduction efficiency, powered by InsightMiner™ which suggests amino acid sequences to be inserted into certain region of AAV cap gene.



CARE™ is also aimed at less targeting certain tissues or cells such as liver, in particular. Pharmaceutical companies developing AAV gene therapy programs are occasionally challenged with safety issues during clinical trials caused by, for instance, liver toxicity in higher dose groups. With their leverage of less transducing liver cells, our candidates for liver-detargeting vectors should be of priceless assistance to the sponsors in clinical study in terms of safety.

  • Conventional libraries from directed evolution & error-prone PCR

    01

  • In-silico / machine learning : InsightMiner™

    02

  • Key features – enhanced tropism, immune evasion,
    and BBB penetration

    03

  • XOB-031 : AAV-9 based rAAV vector with
    CNS tropism & liver de-targeting
    (vector backbone of GXC-303)

    04
AAV gene therapy programs

Genixcure develops therapeutic programs for CNS & genetic rare diseases

  • Vector design by codon optimization & promoter optimization

    01

  • Leveraged by in-house rAAV vectors from CARE™
    with enhanced tropism, immune evasion, and BBB penetration

    02

  • Partnering with sponsors developing AAV gene therapy
    by Genixcure’s rAAV vectors(i.e. GXC-AAVs)
    carrying the sponsors’ GOI(Gene Of Interest)

    03