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

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            Investigational data from long-term, follow-up studies showed that olipudase alfa provided sustained improvement in lung function (as measured by DLco) and reduction of spleen and liver volumes over time in patients with ASMD.

            Lead Product(s): Olipudase Alfa

            Therapeutic Area: Genetic Disease Product Name: GZ402665

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 09, 2022

            Sanofi Company Banner

            TMF Summit 2024

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            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.

            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

            Sanofi Company Banner

            TMF Summit 2024

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            Olipudase alfa is the first and only investigational enzyme replacement therapy in late-stage development for the treatment of ASMD.

            Lead Product(s): Olipudase alfa

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 30, 2020

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            TMF Summit 2024

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            LYS-SAF302 (olenasufligene relduparvovec) is a novel gene therapy in Phase 2/3 development for the treatment of MPS IIIA, or Sanfilippo syndrome type A. LYS-SAF302 delivers a functional copy of the human SGSH gene directly to brain cells using the AAV carrier.

            Lead Product(s): Olenasufligene Relduparvovec

            Therapeutic Area: Genetic Disease Product Name: LYS-SAF302

            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 November 18, 2022

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            LYS-SAF302 (olenasufligene relduparvovec) delivers a working copy of the human SGSH gene directly to the brain through intra-cerebral injection. Mutations to the SGSH gene are responsible for the developmental, physical and behavioral symptoms of the disease.

            Lead Product(s): Olenasufligene Relduparvovec

            Therapeutic Area: Genetic Disease Product Name: LYS-SAF302

            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 November 18, 2022

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            Preliminary results evaluating LYS-SAF302 (olenasufligene relduparvovec) in patients with Sanfilippo syndrome type A who have been followed for at least two years demonstrated improvement, stabilization, or slowing down of decline in cognitive-developmental age.

            Lead Product(s): Olenasufligene Relduparvovec

            Therapeutic Area: Genetic Disease Product Name: LYS-SAF302

            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 May 18, 2022

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            AAVance trial evaluates, effectiveness of a one-time intracerebral delivery of a recombinant adeno-associated virus vector rh.10 carrying the N-sulfoglucosamine sulfohydrolase gene LYS-SAF302 (olenasufligene relduparvovec) in children with MPS IIIA.

            Lead Product(s): Olenasufligene Relduparvovec

            Therapeutic Area: Genetic Disease Product Name: LYS-SAF302

            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 February 24, 2022

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            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.

            Lead Product(s): AAVrh10-h.SGSH

            Therapeutic Area: Genetic Disease Product Name: LYS-SAF302

            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

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            Company has announced death of a patient in the AAVance study , a phase 2/3 international clinical study evaluating gene therapy treatment of Mucopolysaccharidosis type IIIA (MPS IIIA, known as Sanfilippo Syndrome type A) .

            Lead Product(s): AAVrh10-h.SGSH

            Therapeutic Area: Genetic Disease Product Name: LYS-SAF302

            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

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            Details:

            Following discussions with the U.S. FDA, a clinical hold was issued for the clinical trial AAVance, a global Phase 2/3 clinical trial of LYS-SAF302, a gene therapy for the treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA, also known as Sanfilippo syndrome type A).

            Lead Product(s): Olenasufligene Relduparvovec

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 June 05, 2020

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