Apertura Gene Therapy Unveils Novel Engineered AAV Capsids Targeting Human Transferrin Receptor 1 for Neurological Gene Therapy Delivery
Apertura Gene Therapy , a biotechnology company opening opportunities in genetic medicine for treating debilitating diseases with limited options, today unveiled its proprietary engineered Adeno-Associated Virus (AAV) capsids that bind to the human Transferrin Receptor 1 (TfR1).
- Apertura Gene Therapy , a biotechnology company opening opportunities in genetic medicine for treating debilitating diseases with limited options, today unveiled its proprietary engineered Adeno-Associated Virus (AAV) capsids that bind to the human Transferrin Receptor 1 (TfR1).
- This transportation mechanism mediates crossing of the blood-brain barrier (BBB) to enable gene therapy delivery throughout the central nervous system (CNS).
- “Our TfR1 capsids, with their well-characterized mechanism of action, offer the unique advantage of significantly reducing human translation risk – a major historical challenge of engineering novel AAV capsids.
- Apertura is currently advancing two programs utilizing its TfR1 capsids for undisclosed neurologic conditions and is leveraging its platform technologies to engineer novel payloads to regulate genetic expression and develop additional AAV capsids targeting specific human receptors.