Type of information: Grant
Company: Sagetis Biotech (Spain)
Investors: Duchenne Parent Project España (Spain)
Funding type: grant
- The grant will be used to evaluate the coating of adeno-associated viruses (AAV) in order to increase their efficacy as a gene therapy strategy for Duchenne muscular dystrophy. AAVs are the most commonly used viral vectors in the development of gene therapy strategies for the treatment of Duchenne muscular dystrophy, since they are persistent in the long term and have demonstrated an excellent safety profile in many clinical trials. However, the AAVs face with several challenges in order to be an efficient gene therapy, as for example that between 30-80% of the human population has antibodies capable of neutralizing their transduction.
- Previous results generated by Sagetis with its lead candidate SAG-101, have shown that by coating a viral particle with cationic polymers, a reduction of its uptake by the liver, an accumulation in the target organ and a neutralization protection are achieved. This project will aim to perform a coating of AAVs with Sagetis polymers to increase their efficacy by means of changing their tropism, decreasing its presence in the liver, increasing their transduction capability in muscle tissue and protecting them from neutralizing antibodies.
- • On December 22, 2017, Sagetis Biotech announced that Duchenne Parent Project España awarded Dr. Salvador Borrós and his team at Sagetis Biotech a grant to evaluate the coating of adeno-associated viruses (AAV) in order to increase their efficacy as a gene therapy strategy for Duchenne muscular dystrophy (DMD).
Therapeutic area: Cancer - Oncology - Rare diseases