[{"orgOrder":0,"company":"Bausch Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Antibiotic","year":"2021","type":"Inapplicable","leadProduct":"Rifaximin","moa":"Bacterial DNA-directed RNA polymerase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Bausch Health","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bausch Health \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Bausch Health \/ Undisclosed"},{"orgOrder":0,"company":"Bausch Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Antibiotic","year":"2020","type":"Inapplicable","leadProduct":"Rifaximin","moa":"Bacterial DNA-directed RNA polymerase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Bausch Health","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bausch Health \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Bausch Health \/ Undisclosed"},{"orgOrder":0,"company":"Liminal BioSciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Fezagepras","moa":"G-protein coupled receptor 84 | Free fatty acid receptor 1||GPR84","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Liminal BioSciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Liminal BioSciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Liminal BioSciences \/ Undisclosed"},{"orgOrder":0,"company":"Laurent Pharmaceuticals","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"LAU-7b","moa":"Retinoic acid receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Laurent Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Laurent Pharmaceuticals \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"8","companyTruncated":"Laurent Pharmaceuticals \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Laurent Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Fenretinide","moa":"retinol derivatives","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Laurent Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Laurent Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Laurent Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Nova Mentis Life Science","sponsor":"KGK Science","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Psilocybine","moa":"Serotonin 2a (5-HT2a) receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Nova Mentis Life Science","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Nova Mentis Life Science \/ KGK Science","highestDevelopmentStatusID":"8","companyTruncated":"Nova Mentis Life Science \/ KGK Science"},{"orgOrder":0,"company":"Bausch Health","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Methyltyrosine","moa":"Tyrosine 3-hydroxylase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Bausch Health","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Bausch Health \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Bausch Health \/ Undisclosed"},{"orgOrder":0,"company":"Thiogenesis Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"TTI-0102","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Thiogenesis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Thiogenesis Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Thiogenesis Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Qu Biologics Inc","sponsor":"The National Research Council of Canada Industrial Research Assistance Program","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Vaccine","year":"2022","type":"Inapplicable","leadProduct":"Qbkpn SSI","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Qu Biologics Inc","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Qu Biologics Inc \/ The National Research Council of Canada Industrial Research Assistance Program","highestDevelopmentStatusID":"8","companyTruncated":"Qu Biologics Inc \/ The National Research Council of Canada Industrial Research Assistance Program"},{"orgOrder":0,"company":"Qu Biologics Inc","sponsor":"National Research Council of Canada Industrial Research Assistance Program","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Vaccine","year":"2022","type":"Funding","leadProduct":"QBKPN SSI","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Qu Biologics Inc","amount2":0.01,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Injection","sponsorNew":"Qu Biologics Inc \/ National Research Council of Canada Industrial Research Assistance Program","highestDevelopmentStatusID":"8","companyTruncated":"Qu Biologics Inc \/ National Research Council of Canada Industrial Research Assistance Program"},{"orgOrder":0,"company":"Satellos Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"SAT-3247","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Satellos Bioscience","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Satellos Bioscience \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Satellos Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"Satellos Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SAT-3247","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Satellos Bioscience","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Satellos Bioscience \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Satellos Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"Satellos Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SAT-3247","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Satellos Bioscience","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Satellos Bioscience \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Satellos Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"Satellos Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SAT-3247","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Satellos Bioscience","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Satellos Bioscience \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Satellos Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"Satellos Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SAT-3247","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Satellos Bioscience","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Satellos Bioscience \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Satellos Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"Satellos 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II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Satellos Bioscience \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Satellos Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"Satellos Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SAT-3247","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Satellos Bioscience","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Satellos Bioscience \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Satellos Bioscience \/ Undisclosed"},{"orgOrder":0,"company":"Satellos Bioscience","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic 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Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Satellos Bioscience \/ Undisclosed"}]

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                          01

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : SAT-3247 is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Muscular Dystrophy, Duchenne.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          March 10, 2025

                          Lead Product(s) : SAT-3247

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : TTI-0102 is a drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of MELAS Syndrome.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          October 16, 2024

                          Lead Product(s) : TTI-0102

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : Psilocybin is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Fragile X Syndrome.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          April 27, 2023

                          Lead Product(s) : Psilocybine

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : KGK Science

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : XRx-008 (oxypurinol) inhibits the enzyme xanthine oxidase, blocking the conversion of the oxypurines hypoxanthine and xanthine to uric acid, which results in a decrease in the concentrations of uric acid in the blood and urine.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          April 21, 2023

                          Lead Product(s) : Oxypurinol

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : XORTX’s lead program XRx-008 is designed to deliver the xanthine oxidase inhibitor (“XOI”) oxypurinol at concentrations sufficient to substantially inhibit aberrant purine metabolism in the kidneys of individuals with progressing kidney disease due...

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          December 19, 2022

                          Lead Product(s) : Oxypurinol

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : XORTX’s XRx-008 therapeutic development program for ADPKD, is advancing a proprietary composition of xanthine oxidase inhibitor, to manage aberrant purine metabolism and chronically high serum uric acid concentration.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          December 08, 2022

                          Lead Product(s) : Oxypurinol

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : XRx-008 is designed to deliver xanthine oxidase inhibitor oxypurinol at concentrations sufficient to substantially inhibit aberrant purine metabolism in kidney of individuals with progressing kidney disease due to ADPKD inhibit production of uric acid in...

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          November 28, 2022

                          Lead Product(s) : Oxypurinol

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : LAU-7b (oral fenretinide) is a novel candidate that acts on cell membrane lipids to modulate inflammation signaling and protein trafficking. Fenretinide was shown to correct the levels of certain membrane phospholipids and sphingolipids in multiple in vi...

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          November 03, 2022

                          Lead Product(s) : Fenretinide

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Lead Product(s) : QBKPN SSI

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase II

                          Sponsor : National Research Council of Canada Industrial Research Assistance Program

                          Deal Size : $6.6 million

                          Deal Type : Funding

                          Details : QBKPN SSI is designed to reprogram the functional competence of immune cells to fix the immune dysregulation that typically develops with aging, providing broad immune protection, not only against COVID-19.

                          Product Name : QBKPN SSI

                          Product Type : Vaccine

                          Upfront Cash : Undisclosed

                          October 17, 2022

                          Lead Product(s) : QBKPN SSI

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : National Research Council of Canada Industrial Research Assistance Program

                          Deal Size : $6.6 million

                          Deal Type : Funding

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                          10

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : The offering will support to increase the liquidity and raise capital to further develop and advance the pipeline of product candidates, including the conduct of clinical studies and initiation of a registration trial (Phase 3) for lead program XRx-008 h...

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Undisclosed

                          October 04, 2022

                          Lead Product(s) : Oxypurinol

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Alliance Global Partners

                          Deal Size : $5.0 million

                          Deal Type : Public Offering

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