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

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              Sanofi

              • Development Update

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              FRANCE France

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              WRIB

              Participation Not Confirmed

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

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              Lead Product(s): Olipudase alfa

              Therapeutic Area: Genetic Disease

              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.

              Zynerba Pharmaceuticals

              • Development Update

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              UNITED STATES United States

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              WRIB

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              Zynerba Pharmaceuticals Announces the Completion of Enrollment in the Pivotal CONNECT-FX Trial of Zygel™ in Fragile X Syndrome

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              Lead Product(s): ZYN002

              Therapeutic Area: Genetic Disease

              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 February 26, 2020

              Details:

              The trial is evaluating the efficacy and safety of Zygel™ CBD Gel as a treatment for behavioral symptoms of Fragile X syndrome (FXS) in 210 children ages three through 17 with full mutation FXS.

              Lysogene

              • Development Update

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              FRANCE France

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              WRIB

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

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              Lead Product(s): LYS-SAF302

              Therapeutic Area: Genetic Disease

              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 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.

              Stealth Biotherapeutics

              • Development Update

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              UNITED STATES United States

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              WRIB

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              Stealth BioTherapeutics Announces Results From Study to Evaluate Efficacy of Elamipretide

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              Lead Product(s): Elamipretide

              Therapeutic Area: Genetic Disease

              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 February 18, 2020

              Details:

              Elamipretide achieves statistical significance on key secondary endpoints

              JCR Pharmaceuticals

              • Development Update

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              JAPAN Japan

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              WRIB

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              JCR Announces Presentations on JR-141 and JR-162 at the 16th Annual WORLDSymposium 2020

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              Lead Product(s): JR-141

              Therapeutic Area: Genetic Disease

              Highest Development Status: Phase II/ Phase III Product Type: Undisclosed

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable February 13, 2020

              Details:

              The presentations highlight two candidates from JCR’s development pipeline for lysosomal storage disorders; JR-141 and JR-162.

              Zynerba Pharmaceuticals

              • Development Update

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              UNITED STATES United States

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              WRIB

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              Zynerba Pharma Achieves Patient Screening Target for Zygel

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              Lead Product(s): Cannabidiol

              Therapeutic Area: Genetic Disease

              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 February 03, 2020

              Details:

              Zynerba Pharma has successfully achieved its patient screening target in its 14-week pivotal CONNECT-FX trial assessing the efficacy and safety of Zygel™ CBD Gel in children and adolescents.

              Zynerba Pharmaceuticals

              • Development Update

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              UNITED STATES United States

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              WRIB

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              Zynerba Pharmaceuticals Announces that Enrollment is Nearing Completion in Pivotal CONNECT-FX Trial in Fragile X Syndrome

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              Lead Product(s): Cannabidiol

              Therapeutic Area: Genetic Disease

              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 January 13, 2020

              Details:

              The trial has enrolled more severely affected population than that enrolled in the previously completed Phase 2 FAB-C trial as measured by baseline behavioral symptoms.

              Orphazyme

              • Development Update

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              DENMARK Denmark

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              WRIB

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              Orphazyme reports positive arimoclomol data from open-label phase 2/3 extension in Niemann-Pick disease Type C

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              Lead Product(s): Arimoclomol

              Therapeutic Area: Genetic Disease

              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 January 03, 2020

              Details:

              Orphazyme expects to receive approval for NPC in H1 2021 in the US through the fast track approval process, with the aim to bring the product to patients shortly thereafter.

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