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    [{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Acer Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Sodium Phenylbutyrate","moa":"Mitochondria-mediated apoptosis","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Relief Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Relief Therapeutics \/ Acer Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Relief Therapeutics \/ Acer Therapeutics"},{"orgOrder":0,"company":"Spexis","sponsor":"SPRIM Global Investments","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibiotic","year":"2023","type":"Financing","leadProduct":"Colistimethate Sodium","moa":"Cell membrane","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Spexis","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"Spexis \/ SPRIM Global Investments","highestDevelopmentStatusID":"10","companyTruncated":"Spexis \/ SPRIM Global Investments"},{"orgOrder":0,"company":"Spexis","sponsor":"SPRIM Global Investments","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibiotic","year":"2023","type":"Financing","leadProduct":"Colistimethate Sodium","moa":"Cell membrane","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Spexis","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"Spexis \/ SPRIM Global Investments","highestDevelopmentStatusID":"10","companyTruncated":"Spexis \/ SPRIM Global Investments"},{"orgOrder":0,"company":"Spexis","sponsor":"SPRIM Global Investments","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibiotic","year":"2023","type":"Partnership","leadProduct":"Colistimethate Sodium","moa":"Cell membrane","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Spexis","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"Spexis \/ SPRIM Global Investments","highestDevelopmentStatusID":"10","companyTruncated":"Spexis \/ SPRIM Global Investments"},{"orgOrder":0,"company":"Idorsia Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Lucerastat","moa":"GCS","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Idorsia Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Hard Capsule","sponsorNew":"Idorsia Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Idorsia Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Idorsia Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Lucerastat","moa":"GCS","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Idorsia Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Hard Capsule","sponsorNew":"Idorsia Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Idorsia Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Tominersen","moa":"Huntingtin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"F. Hoffmann-La Roche \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"F. Hoffmann-La Roche \/ Inapplicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Ionis Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Tominersen","moa":"Huntingtin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"F. Hoffmann-La Roche \/ Ionis Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"F. Hoffmann-La Roche \/ Ionis Pharmaceuticals"},{"orgOrder":0,"company":"AB2 Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Tadekinig Alpha","moa":"IL-18 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"AB2 Bio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"AB2 Bio \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"AB2 Bio \/ Inapplicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"Onasemnogene Abeparvovec","moa":"SMN1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Inapplicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Phase III","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"F. Hoffmann-La Roche \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"F. Hoffmann-La Roche \/ Inapplicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Chugai Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell and Gene therapy","year":"2021","type":"Licensing Agreement","leadProduct":"Delandistrogene Moxeparvovec","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Phase III","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"F. Hoffmann-La Roche \/ Chugai Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"F. Hoffmann-La Roche \/ Chugai Pharmaceutical"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Highbridge Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2020","type":"Financing","leadProduct":"Idebenone","moa":"CYP450-2C9","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Santhera Pharmaceuticals \/ Highbridge Capital Management","highestDevelopmentStatusID":"10","companyTruncated":"Santhera Pharmaceuticals \/ Highbridge Capital Management"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Idebenone","moa":"CYP450-2C9","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Santhera Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Santhera Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Idebenone","moa":"CYP450-2C9","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Santhera Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Santhera Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Idebenone","moa":"CYP450-2C9","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Santhera Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Santhera Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Idebenone","moa":"CYP450-2C9","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Santhera Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Santhera Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"OAV-101","moa":"SMN1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Inapplicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"Onasemnogene Abeparvovec","moa":"SMN1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Inapplicable"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Deucrictibant","moa":"Bradykinin B2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharvaris","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Softgel Capsule","sponsorNew":"Pharvaris \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharvaris \/ Inapplicable"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Pharvaris

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                            Lead Product(s) : Deucrictibant

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Pharvaris Announces Orphan Designation Granted to Deucrictibant by the European Commission

                            Details : PHVS416 (deucrictibant) is a novel, potent, oral small-molecule bradykinin B2 receptor antagonist currently in development to prevent the occurrence of bradykinin-mediated angioedema attacks.

                            Product Name : PHVS416

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            April 01, 2025

                            Lead Product(s) : Deucrictibant

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Novartis Pharmaceuticals Corporation

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                            Lead Product(s) : Onasemnogene Abeparvovec

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Novartis Phase III Data Shows Efficacy of Onasemnogene Abeparvovec in SMA

                            Details : Zolgensma (onasemnogene abeparvovec) is USFDA approved, one time gne therapy that acts as SMN1 gene stimulant. It is indicated for the treatment of spinal muscular atrophy.

                            Product Name : Zolgensma

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            March 19, 2025

                            Lead Product(s) : Onasemnogene Abeparvovec

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Novartis Pharmaceuticals Corporation

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                            Lead Product(s) : Onasemnogene Abeparvovec

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Novartis Phase III Study of Onasemnogene Abeparvovec Meets Primary Endpoint in SMA

                            Details : Zolgensma (onasemnogene abeparvovec) is USFDA approved, one time gne therapy that acts as SMN1 gene stimulant. It is indicated for the treatment of spinal muscular atrophy.

                            Product Name : Zolgensma

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            December 30, 2024

                            Lead Product(s) : Onasemnogene Abeparvovec

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Spexis

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                            Spexis

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                            Lead Product(s) : Colistimethate Sodium

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : SPRIM Global Investments

                            Deal Size : $2.5 million

                            Deal Type : Financing

                            DEALS_DEV arrow-down

                            Spexis Announces USD $2.5 Million Capital Commitment to Support the Upcoming Phase 3 ColiFin® Studies

                            Details : 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.

                            Product Name : ColiFin

                            Product Type : Antibiotic

                            Upfront Cash : Undisclosed

                            August 15, 2023

                            Lead Product(s) : Colistimethate Sodium

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : SPRIM Global Investments

                            Deal Size : $2.5 million

                            Deal Type : Financing

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                            05

                            Spexis

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                            Spexis

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                            Lead Product(s) : Colistimethate Sodium

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : SPRIM Global Investments

                            Deal Size : Undisclosed

                            Deal Type : Partnership

                            DEALS_DEV arrow-down

                            Spexis and SPRIM Global Investments (SGI) Announce Plans for Clinical Trial Partnership to Fund up to Half of ...

                            Details : 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 (C...

                            Product Name : ColiFin

                            Product Type : Antibiotic

                            Upfront Cash : Undisclosed

                            August 02, 2023

                            Lead Product(s) : Colistimethate Sodium

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : SPRIM Global Investments

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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                            06

                            AB2 Bio

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                            AB2 Bio

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                            Lead Product(s) : Tadekinig Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            AB2 Bio Completes Enrolment in Pivotal Phase 3 Trial of Tadekinig alfa to Treat Primary Monogenic IL-18 Driven...

                            Details : 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...

                            Product Name : r-hIL-18BP

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            July 03, 2023

                            Lead Product(s) : Tadekinig Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Relief Therapeutics

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                            Relief Therapeutics

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                            Lead Product(s) : Sodium Phenylbutyrate

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Acer Therapeutics

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Relief Therapeutics and Acer Therapeutics Announce that the European Commission Has Granted Orphan Drug Design...

                            Details : ACER-001 (sodium phenylbutyrate) is an immediate-release, polymer coated, multi-particulate formulation of sodium phenylbutyrate for oral administration via suspension, that is designed to improve palatability.

                            Product Name : Olpruva

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            December 08, 2022

                            Lead Product(s) : Sodium Phenylbutyrate

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Acer Therapeutics

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            F. Hoffmann-La Roche

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                            F. Hoffmann-La Roche

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                            Lead Product(s) : Delandistrogene Moxeparvovec

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Chugai Pharmaceutical

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

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                            Chugai In-Licenses Gene Therapy Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy

                            Details : 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.

                            Product Name : SRP-9001

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            December 16, 2021

                            Lead Product(s) : Delandistrogene Moxeparvovec

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Chugai Pharmaceutical

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

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                            09

                            Idorsia Pharmaceuticals

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                            Idorsia Pharmaceuticals

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                            Lead Product(s) : Lucerastat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Idorsia to Further Characterize Lucerastat for The Treatment of Fabry Disease by Continuing the Open-Label Ext...

                            Details : 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...

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            December 13, 2021

                            Lead Product(s) : Lucerastat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Idorsia Pharmaceuticals

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                            Lead Product(s) : Lucerastat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Idorsia Announces the Results of MODIFY, a Phase 3 Study of Lucerastat in Fabry disease

                            Details : 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 lucer...

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            November 10, 2021

                            Lead Product(s) : Lucerastat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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