Imagine a tiny biological delivery truck used to carry healthy genes to the liver. Currently, many people's immune systems block these trucks, or the treatment fades away as children grow. This work creates smarter, stealthier trucks that can bypass the body's defenses and stay active even as the liver grows.
Current liver gene therapies are inaccessible to 50% of patients due to immune rejection and are ineffective in children because the treatment dilutes as the liver grows.
A toolkit including 3000+ synthetic AAV capsids, 48 immune-reducing Ide molecules, and self-replicating AAVs for long-term expression.
If you are a gene therapy developer dealing with high patient exclusion rates due to pre-existing immunity — this project developed 48 new Ide molecules that reduce immune responses. This allows a larger patient population to qualify for treatment.
If you are a rare disease biotech dealing with the loss of therapeutic effect in growing children's livers — this project developed self-replicating AAVs. This ensures the treatment lasts longer by preventing the gene from being diluted during cell division.
If you are a genome editing firm dealing with delivery toxicity and inefficiency — this project developed a library of over 3000 AAV capsid variants. These synthetic carriers provide higher potency and lower toxicity for liver-directed delivery.
Based on available project data, specific pricing or cost-per-dose information is not provided.
The project focuses on the design of 3000+ variants and new molecules, but based on available project data, industrial scale-up metrics are not yet detailed.
The project has identified promising Cas9 variants and capsid libraries, but specific licensing terms are not mentioned in the provided data.
Based on available project data, the project runs from 2022 to 2027, indicating a multi-year development cycle for these tools.
The project aims to reduce hepatotoxicity and immune responses, which are primary regulatory concerns for liver-directed gene therapies.
The consortium is well-balanced for translation, consisting of 10 partners across 5 countries. With a 30% industry ratio (3 companies, including 3 SMEs), there is a clear bridge between the 7 academic and research entities and the commercial market, ensuring that the developed AAV tools are designed with industrial applicability in mind.
Contact Fondazione Telethon ETS in Italy
Contact us to explore licensing opportunities for the 3000+ AAV capsid variants.
