CSBio CSBio

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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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Therapeutics"}]

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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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            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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            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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            The company intends to use the net proceeds for the clinical development of DNL310 (tividenofusp alfa), a fusion protein composed of IDS fused to Denali’s proprietary Enzyme transport vehicle. It is being evaluated in the treatment of Mucopolysaccharidosis Type II.

            Lead Product(s): Tividenofusp Alfa

            Therapeutic Area: Genetic Disease Product Name: DNL310

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

            Partner/Sponsor/Collaborator: Undisclosed

            Deal Size: $500.0 million Upfront Cash: Undisclosed

            Deal Type: Private Placement February 27, 2024

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            RGX-121 is an investigational, one-time gene therapy designed to deliver the IDS gene that encodes the iduronate-2-sulfatase enzyme (I2S) using the NAV® AAV9 vector. It is under development for the treatment of Hunter syndrome.

            Lead Product(s): RGX-121

            Therapeutic Area: Genetic Disease Product Name: RGX-121

            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 07, 2024

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            PHVS416 (deucrictibant) is a potent, selective, and orally available antagonist of the bradykinin B2 receptor. It is being evaluated in phase 2/3 trials for the prophylactic treatment of HAE Attacks.

            Lead Product(s): Deucrictibant

            Therapeutic Area: Genetic Disease Product Name: PHVS416

            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 22, 2024

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            ANAVEX 2-73 (blarcamesine) is an orally available drug candidate that restores cellular homeostasis by targeting sigma-1 and muscarinic receptors. It is under phase 3 clinical development for the treatment of patients with Rett syndrome (RTT).

            Lead Product(s): Blarcamesine

            Therapeutic Area: Genetic Disease Product Name: Anavex 2-73

            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 02, 2024

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            REN001 (mavodelpar) is a potent and selective peroxisome proliferator-activated receptor delta (PPARδ) agonist which is being evaluated for the treatment of primary mitochondrial myopathies (PMM).

            Lead Product(s): Mavodelpar

            Therapeutic Area: Genetic Disease Product Name: REN001

            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 December 14, 2023

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            QRX003 is a once-daily topical lotion comprised of a broad-spectrum serine protease inhibitor, formulated with the proprietary invisicare® delivery technology. It is being investigated for treating netherton syndrome.

            Lead Product(s): QRX003

            Therapeutic Area: Genetic Disease Product Name: QRX003

            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 October 24, 2023

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            UX143 (setrusumab) is a fully human monoclonal antibody that inhibits sclerostin, a protein that acts on a key bone-signaling pathway that inhibits the maturation and activity of bone-forming cells in Osteogenesis Imperfecta.

            Lead Product(s): Setrusumab

            Therapeutic Area: Genetic Disease Product Name: UX143

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

            Partner/Sponsor/Collaborator: Mereo BioPharma Group

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 14, 2023

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