TRANSFORMING RARE DISEASE CARE BY TREATING THE CAUSE
Our platform starts with tackling families of rare diseases such as GM2 gangliosidosis and cerebral creatine deficiency syndromes. These rare diseases have little to no available treatment options. With Gencorrex's platform of gene therapies, our approach aims to address the underlying cause of the disease by replacing the missing genes.
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Discover more about the stages of our therapies below! ​
Our Platform
GM2 GANGLIOSIDOSIS
GM2 (GM2 gangliosidosis) are a family of lysosomal storage disorders consisting of Tay-Sachs disease (TSD), Sandhoff disease (SD) and AB-Variant GM2 (ABGM2). We are set to enter early clinical stages for a joint gene therapy treatment for TSD and SD with ABGM2 in the pre-clinical trial application (pre-CTA) enabling phase.
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CEREBRAL CREATINE DEFICIENCY SYNDROMES
CCDS (Cerebral Creatine Deficiency Syndromes) are a group of inborn errors of creatine metabolism consisting of creatine transporter deficiency (CTD), guanidinoacetate methyltransferase deficiency (GAMT-D) and arginine:glycine amidinotransferase deficiency (AGAT-D). Our therapeutics for CTD and GAMT-D are entering CTA-enabling studies in pre-clinical models.
XLID98
X-Linked Intellectual Disability 98 is a rare neurodevelopmental disorder with no available treatments. We are in early pre-clinical investigational stages in cellular and mouse models for validation of a gene therapy treatment. ​