Genetic Disease | Phase II/ Phase III Developments | FRANCE

Sanofi

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Sanofi Halts Venglustat Clinical Programme in ADPKD

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.

Lysogene

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Lysogene Provides Update on the AAVance Clinical Trial Evaluating LYS-SAF302 in Patients with MPS IIIA

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.