Genetic Disease | Phase II/ Phase III Developments | FRANCE

Sanofi

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FRANCE France

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Chinaplas 2020

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Positive topline results demonstrated by olipudase alfa, first and only investigational therapy in late-stage development for acid sphingomy...

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

Details:

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

Lysogene

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FRANCE France

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Chinaplas 2020

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Lysogene Announces Important Updates on AAVance Phase 2/3 Clinical Trial with LYS-SAF302 in MPS IIIA

Lead Product(s): LYS-SAF302

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

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).

Lysogene

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FRANCE France

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Chinaplas 2020

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Lysogene Receives FDA Fast Track Designation for LYS-SAF302 Gene Therapy in MPS IIIA

Lead Product(s): LYS-SAF302

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 February 25, 2020

Details:

The AAVance Phase 2/3 clinical study is designed as an open-label, single-arm, multicenter study of LYS-SAF302 for the treatment of MPS IIIA.

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Sanofi

Positive topline results demonstrated by olipudase alfa, first and only investigational therapy in late-stage development for acid sphingomy...

Lysogene

Lysogene Announces Important Updates on AAVance Phase 2/3 Clinical Trial with LYS-SAF302 in MPS IIIA

Lysogene

Lysogene Receives FDA Fast Track Designation for LYS-SAF302 Gene Therapy in MPS IIIA