Barcelona, December 22 2017 – Sagetis Biotech today 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).
Duchenne Parent Project España, a nonprofit organization dedicated to the fight to end Duchenne muscular dystrophy (Duchenne), announced today that it has awarded Dr. Salvador Borros 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.
Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 3,500 to 5,000 live male births. The loss of a key muscle protein called dystrophin causes muscle wasting and weakness, eventually leading to the loss of ambulation, difficulty breathing, and heart failure.
Adeno-associated viruses (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.
About Sagetis Biotech
Sagetis Biotech is devoted to developing an innovative gene delivery technology to overcome a fundamental engineering challenge in gene therapy: the development of safe and effective delivery vectors.
Privately funded and based in Barcelona, Sagetis Biotech is developing SAG-101: a polymer-coated adenovirus for the treatment of pancreatic cancer.
About Duchenne Parent Project España
The Duchenne Parent Project España Association is a non-profit association created and directed by parents of children with Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD).
The aim of this association is to: help, stimulate, promote scientific and medical research so that the therapies that cure the muscular dystrophies of Duchenne and Becker, as well as, the protection of families that need it, are put into action.