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Find Drugs for Genetic Disease in Phase II/ Phase III Clinical Development in FRANCE

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            Lead Product(s): Venglustat

            Therapeutic Area: Genetic Disease Product Name: GZ402671

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 01, 2021

            Details:

            The STAGED-PKD study was stopped for futility following an independent analysis of the annualized rate of change in total kidney volume (TKV) in patients receiving venglustat compared to placebo.

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            Lead Product(s): LYS-SAF302

            Therapeutic Area: Genetic Disease Product Name: AAVrh10-h.SGSH

            Highest Development Status: Phase II/ Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 26, 2020

            Details:

            AAVance clinical trial is a global Phase 2/3 gene therapy trial for the treatment of Mucopolysaccharidosis. The company remains committed to the LYS-SAF302 development program and the Sanfilippo patient community.

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            Lead Product(s): LYS-SAF302

            Therapeutic Area: Genetic Disease Product Name: AAVrh10-h.SGSH

            Highest Development Status: Phase II/ Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 15, 2020

            Details:

            Company has announced death of a patient in t