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Developments Genetic Disease Phase I
Netherlands 
Details:
Positive safety, tolerability and pharmacodynamics data from multiple dose levels in healthy volunteers support evaluation of AZ-3102, a small molecule designed to be a potent and selective inhibitor of two target enzymes involved in glycolipid metabolism in Gangliosidoses.
Lead Product(s): AZ-3102
Therapeutic Area: Genetic Disease Product Name: AZ-3102
Highest Development Status: Phase I Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 14, 2022
Details:
The ODD for AZ-3102, selectively inhibit two enzymes involved in glycolipid metabolism GCS and GbA2 for the treatment ofGM2 gangliosidosis has been granted based on efficacy demonstrated in a Sandhoff mouse model, including a clear effect on animal survival.
Lead Product(s): AZ-3102
Therapeutic Area: Genetic Disease Product Name: AZ-3102
Highest Development Status: Phase I Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 01, 2022
Details:
Phase 1 clinical study investigates safety and tolerability, pharmacokinetics and pharmacodynamic effects of first-in-class azasugar AZ-3102 in healthy subjects. AZ-3102 is being initially developed for the treatment of patients suffering from GM1 and GM2 gangliosidoses.
Lead Product(s): AZ-3102
Therapeutic Area: Genetic Disease Product Name: AZ-3102
Highest Development Status: Phase I Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable April 06, 2021
