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[{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Roche Trial of Ionis Huntington\u2019s Disease Drug Halted After Side Effects","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Santhera Completes Enrollment of Phase 3 SIDEROS Study with Puldysa\u00ae (Idebenone) in Duchenne Muscular Dystrophy (DMD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Santhera Pharmaceuticals"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Highbridge Capital Management","pharmaFlowCategory":"D","amount":"$21.1 million","upfrontCash":"Undisclosed","newsHeadline":"Santhera Receives Financing Commitment of up to CHF 20 Million from Fund Managed by Highbridge Capital Management","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Santhera Pharmaceuticals"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"UK\u2019s MHRA renews EAMS Scientific Opinion for Santhera\u2019s Idebenone in Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Santhera Pharmaceuticals"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Santhera to Discontinue Phase 3 SIDEROS Study and Development of Puldysa\u00ae in Duchenne Muscular Dystrophy (DMD) and Focus on Vamorolone","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Santhera Pharmaceuticals"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Santhera to Discontinue Phase 3 SIDEROS Study and Development of\n Puldysa\u00ae in Duchenne Muscular Dystrophy (DMD) and Focus on Vamorolone","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Santhera Pharmaceuticals"},{"orgOrder":0,"company":"F. 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            The financing will be used to fund the upcoming Phase 3 studies of ColiFin (colistimethate sodium), an inhaled antibiotic therapeutic for the management of chronic infections in cystic fibrosis patients.

            Lead Product(s): Colistimethate Sodium

            Therapeutic Area: Genetic Disease Product Name: ColiFin

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: SPRIM Global Investments

            Deal Size: $2.5 million Upfront Cash: Undisclosed

            Deal Type: Financing August 15, 2023

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            r-hIL-18BP (tadekinig alfa) is a recombinant human interleukin-18 binding protein inhibiting IL18. It captures excess free IL-18 mimicking the normal physiological situation, and therefore represents a well differentiated approach for the treatment of IL-18-opathies.

            Lead Product(s): Tadekinig Alfa

            Therapeutic Area: Genetic Disease Product Name: r-hIL-18BP

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 07, 2023

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            This collaboration is expected to provide capitalization through significant clinical milestones in ColiFin®’s Phase 3 development. ColiFin® (colistimethate sodium) is being developed for the treatment of chronic lung infections in cystic fibrosis (CF) patients.

            Lead Product(s): Colistimethate Sodium

            Therapeutic Area: Genetic Disease Product Name: ColiFin

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: SPRIM Global Investments

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Partnership February 08, 2023

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            Under the license agreement between Roche and Chugai, Chugai obtained exclusive marketing right in Japan for delandistrogene moxeparvovec (SRP-9001), an investigational gene therapy for Duchenne muscular dystrophy.

            Lead Product(s): Delandistrogene Moxeparvovec

            Therapeutic Area: Genetic Disease Product Name: SRP-9001

            Highest Development Status: Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Chugai Pharmaceutical

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement December 16, 2021

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            Lucerastat demonstrated a substantial reduction in levels of the Fabry-disease biomarker plasma Gb3 after 6 months of treatment. The potential effect of lucerastat on kidney function over 6 months of treatment was evaluated at the interim analysis of the extension study.

            Lead Product(s): Lucerastat

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 13, 2021

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            The safety, tolerability, pharmacodynamics, and pharmacokinetics of oral lucerastat were evaluated in study in adult patients with Fabry disease. In this single-center, open-label, randomized study, 10 patients with Fabry disease were randomized to lucerastat for 12 weeks.

            Lead Product(s): Lucerastat

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 11, 2021

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            STEER will build upon the Phase 1/2 study which showed that treatment with OAV-101 IT led to significant increases in Hammersmith Functional Motor Scale-Expanded scores and a clinically meaningful response in older patients between ≥2 years and <5 years old with SMA Type 2.

            Lead Product(s): OAV-101

            Therapeutic Area: Genetic Disease Product Name: OAV-101

            Highest Development Status: 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 August 03, 2021

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            New clinical study to evaluate safety and efficacy of Zolgensma in children up to 21 kg, adding to real-world experience and regulatory approvals in Europe and Canada.

            Lead Product(s): Onasemnogene abeparvovec

            Therapeutic Area: Genetic Disease Product Name: Zolgensma

            Highest Development Status: 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 April 23, 2021

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            Tominersen, previously IONIS-HTTRx or RG6042, is an investigational antisense therapy designed to reduce the production of all forms of the huntingtin protein (HTT), including its mutated variant, mHTT.

            Lead Product(s): Tominersen

            Therapeutic Area: Genetic Disease Product Name: RG6042

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Ionis Pharmaceuticals

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 22, 2021

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            Data from an interim analysis conducted by the independent Data and Safety Monitoring Board concluded that the Phase 3 SIDEROS study with Puldysa® (idebenone) was unlikely to meet its primary endpoint.

            Lead Product(s): Idebenone

            Therapeutic Area: Genetic Disease Product Name: Puldysa

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 06, 2020

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