Case study 03
Next Generation AAV Therapeutics
Athebody® DARPin is an ideal modality to drive AAV tropism.
Pubished partnership: RegenXbio
AAV-mediated delivery limited by high liver uptake
- Safe and effective delivery of AAV to extrahepatic tissues has proven challenging due to high liver uptake
- Next generation AAV vectors can be engineered to target extrahepatic tissues and require:
– Reduction of liver uptake
– Redirection of binding to organ-specific receptors
Advantages of targeted AAV vectors
- Systemic delivery with specific targeting to extrahepatic organs, resulting in reduced liver toxicity and improved efficacy
- Local delivery (e.g. hearing loss, ophthalmology) with increased transduction efficiency, enabling dual-AAV for delivering larger cargo
- Lower therapeutic doses required to transduce target organs
- Improved safety profiles for better therapeutic indices
- Improving access to indications incl. cardiovascular, musculoskeletal and neurological tissues
Athebody® DARPins can be rationally designed to any receptor of interest, incorporated into the AAV capsid and enable efficient manufacturing
What is your project?
We are looking forward to hearing your ideas how Athebody® DARPins can enable your projects and how you like to access it.