Polpharma European CDMO Partner & API Manufacturer since 1951 Polpharma European CDMO Partner & API Manufacturer since 1951

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III","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"AMO Pharma"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Jefferies LLC","pharmaFlowCategory":"D","amount":"$55.0 million","upfrontCash":"Undisclosed","newsHeadline":"Reneo Announces Pricing of Public Offering of Common Stock","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Reneo Pharmaceuticals"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Quoin Pharmaceuticals Announces 50% Enrollment in Open Label Netherton Syndrome Clinical Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Quoin Pharmaceuticals"},{"orgOrder":0,"company":"Minoryx Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Minoryx Gains FDA Approval to Initiate a Phase 3 Clinical Trial in Patients with Cerebral Adrenoleukodystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"SPAIN","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Minoryx Therapeutics"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Mereo BioPharma Group","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx and Mereo BioPharma Announce Positive Data from the Ongoing Phase 2\/3 Orbit Study of Setrusumab (UX143) in Osteogenesis Imperfecta (OI)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Syncona Limited","pharmaFlowCategory":"D","amount":"$120.0 million","upfrontCash":"Undisclosed","newsHeadline":"Beacon Therapeutics Launches with \u00a396 Million ($120 million) to Develop a New Generation of Gene Therapies for Retinal Diseases Resulting in Blindness","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Cell and Gene 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Secures Nearly 95 Million USD in Series C+ Financing for Gene Therapy Clinical Trials","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Neurophth Therapeutics"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Reneo Pharmaceuticals Announces Last Patient Last Visit in the Pivotal STRIDE Study of Mavodelpar in Primary Mitochondrial Myopathies (PMM)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Reneo 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Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Quoin Pharmaceuticals"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Reneo Pharmaceuticals Announces Results from Pivotal STRIDE Study of Mavodelpar in Primary Mitochondrial Myopathies (PMM)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Reneo Pharmaceuticals"},{"orgOrder":0,"company":"Anavex Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not 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Therapeutics"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pharvaris Announces Phase 3 Clinical Study Design for Recently Initiated RAPIDe-3 Study, and Presents Quality-of-Life Improvement and Caregiver Behavior Data at Two Recent HAE Congresses","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Pharvaris"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pharvaris Announces Positive Top-line Phase 2 Data from the CHAPTER-1 Study of Deucrictibant for the Prophylactic Treatment of HAE Attacks","therapeuticArea":"Genetic 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Hoffmann-La Roche","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Disc Reports Topline Results from Phase 2 AURORA Study of Bitopertin in Patients with Erythropoietic Protoporphyria (EPP)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Disc medicine"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AMO Pharma Completes Meeting with U.S. FDA and Outlines Plans to Advance Clinical Development of AMO-02 (tideglusib) in Treatment of Myotonic Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"AMO Pharma"}]

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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

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            WBC 2024

            Not Confirmed

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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

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            WBC 2024

            Not Confirmed

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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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            WBC 2024

            Not Confirmed

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            Details:

            AMO-02 (tideglusib) is an investigational therapy in development for the treatment of DM1 with a dual mechanism disrupting the pathogenic RNA repeat in DM1and inhibiting excess levels of the kinase GSK3β.

            Lead Product(s): Tideglusib

            Therapeutic Area: Genetic Disease Product Name: AMO-02

            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 May 02, 2024

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            DISC-1459 (bitopertin) is an investigational, clinical-stage, orally-administered inhibitor of glycine transporter 1 (GlyT1) that is designed to modulate heme biosynthesis. It is being evaluated for the treatment of erythropoietic protoporphyria.

            Lead Product(s): Bitopertin

            Therapeutic Area: Genetic Disease Product Name: DISC-1459

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

            Partner/Sponsor/Collaborator: F. Hoffmann-La Roche

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 01, 2024

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            PHVS416 (deucrictibant) is a potent, selective, and orally available antagonist of the bradykinin B2 receptor, which is being evaluated for the treatment of hereditary angioedema attacks.

            Lead Product(s): Deucrictibant

            Therapeutic Area: Genetic Disease Product Name: PHVS416