A Novel Encapsulated Cell Therapy for Sanfilippo Syndrome Type IIIA

• Unité de recherche: INNOSUISSE
• Numéro de projet: 127.375 IP-LS
• Titre du projet anglais: A Novel Encapsulated Cell Therapy for Sanfilippo Syndrome Type IIIA

Mots-clé (Anglais)

Life sciences, Medtech

Résumé des résultats (Abstract) (Anglais)

Release Tx (RTx) develops the Myo-P® platform for intracerebral enzyme therapy in genetic CNS disorders. RTx seeks funding to complete a preclinical proof of concept using its technology to deliver continuous Heparan Sulfamidase (SGSH) in a mouse model of MPS IIIA (Sanfilippo syndrome type IIIA).