Sagetis Biotech today announced that in light of promising in-vivo results obtained, Duchenne Parent Project España has awarded Dr. Salvador Borrós and his team at Sagetis Biotech a one year extension to the signed 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).
The ongoing experiments with Adeno-associated viruses (AAVs) coated with Sagetis proprietary Poly(beta-amino ester)s (PBAE) polymers have shown an increase in their targeting to the muscle tissue and reduced neutralization by the immune system when administered systemically in comparison to the naked adenoviral vectors.
This one-year expansion will be aimed to achieve an even more stable interaction between the polymeric coating and the viral particles after their intravenous administration to ultimately allow the administration of repeated doses of a recombinant AAV containing the dystrophin gene.
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.
About Sagetis Biotech
Sagetis Biotech is devoted to developing an innovative polymeric technology (Viroshield) to overcome a fundamental engineering challenge in gene therapy: the development of safe and effective delivery vectors. This coating technology is able to modify the behavior of oncolytic viruses, improving their therapeutic effect by different means. The coated oncolytic adenoviruses are protected against pre-existing neutralizing antibodies, by masking their surface antigenic epitopes, while preserving their transduction capability. This technology can be adapted to coat both enveloped and non-enveloped viruses.
Privately funded and based in Barcelona, Sagetis Biotech is also developing SAG-101: a polymer-coated adenovirus for the treatment of pancreatic ductal adenocarcinoma.
For more information, please visit www.sagetis-biotech.com
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.