Spark100 Serotype
Bioengineered clinical AAV capsid derived from AAVrh.74 for hemophilia B gene therapy. Optimized for liver transduction with favorable seroprevalence profile.
Characteristics
Bioengineered from AAVrh.74 template. Optimized for high liver transduction efficiency. Very low pre-existing neutralizing antibody prevalence in humans. Favorable biodistribution profile with liver specificity. Reduced off-target transduction. Strong clinical safety and efficacy data. Used in SPK-9001 hemophilia B program.
Applications
Hemophilia B gene therapy (clinical trials with sustained Factor IX expression). Liver-directed gene therapy in antibody-positive populations. Clinical hemophilia programs. Demonstrates successful AAV bioengineering for clinical translation.
Literature References
- George et al. (2017). Hemophilia B gene therapy with a high-specific-activity factor IX variant. N Engl J Med - George 2017 Spark100
- Majowicz et al. (2019). Successful repeated hepatic gene delivery in mice and non-human primates achieved by sequential administration of AAV5ch and AAV1. Mol Ther Methods Clin Dev - Majowicz 2019 Spark100
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