AAVantgarde Bio develops gene therapies utilizing proprietary Adeno-Associated Viral (AAV) vector technology to enable the delivery of large genes for the treatment of inherited retinal disorders. The company addresses the limitations of traditional AAV vectors, which restrict the payload size, thereby expanding the potential for genetic medicine in underserved patient populations.
Funding
$70.4M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
FCFounders
Product
Problem
Traditional Adeno-Associated Viral (AAV) vectors used in gene therapy have limited payload capacity, restricting their application to diseases requiring smaller genes. This limitation hinders the treatment of inherited disorders caused by large genes, leaving a significant portion of patients with unmet medical needs.
Solution
AAVantgarde Bio develops gene therapies for inherited retinal disorders (IRD) by overcoming the payload limitations of standard AAV vectors. The company's proprietary AAV vector technology enables the delivery of large genes, expanding the scope of treatable genetic illnesses. This approach allows for addressing previously inaccessible genetic targets, offering potential therapeutic solutions for underserved patient populations with debilitating diseases. The company's pipeline focuses on IRDs with high unmet needs, with the potential to expand to other therapeutic areas.
Target Audience
The primary target audience includes patients with inherited retinal disorders caused by large genes, as well as the medical community focused on gene therapy and treatment of genetic illnesses.
Features
- Proprietary AAV vector technology enabling delivery of large genes.
- Focus on inherited retinal disorders (IRDs) with high unmet need.
- Pipeline programs targeting specific IRDs.
- Potential to expand technology to other therapeutic areas beyond IRDs.
- LUCE-1 clinical study for retinitis pigmentosa related to USH1B.