AavantiBio is using gene transfer and gene editing to develop new therapies to treat genetic diseases. Precision genetic medicine technologies have the potential to treat or cure many serious genetic diseases with significant unmet need. Beginning with Friedreich’s ataxia, AavantiBio is building a platform combining their technology with chemistry and manufacturing to create many therapeutic modalities. Critical to AavantiBio’s approach is transient immunomodulation, to prevent immune responses to adeno-associated viruses that deliver the genetic payload. AavantiBio is using comprehensive preclinical and clinical data to support their immunomodulation method. Currently, AavantiBio’s pipeline is in preclinical stages with promising efficacy and safety in mice, rats and non-human primates.
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