[{"orgOrder":0,"company":"ML Bio Solutions","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Ribitol","moa":"Glycosylation","graph1":"Genetic Disease","graph2":"Phase III","graph3":"ML Bio Solutions","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"ML Bio Solutions \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"ML Bio Solutions \/ Inapplicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Ribitol","moa":"Glycosylation","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"BridgeBio Pharma \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Inapplicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Ribitol","moa":"Glycosylation","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"BridgeBio Pharma \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Inapplicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Ribitol","moa":"Glycosylation","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Sachet","sponsorNew":"BridgeBio Pharma \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Inapplicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Ribitol","moa":"Glycosylation","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Sachet","sponsorNew":"BridgeBio Pharma \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Larkin Community Hospital","sponsor":"Ferrer Internacional | Xlear, Inc","pharmaFlowCategory":"DU","therapeuticArea":"Infections and Infectious Diseases","country":"","productType":"Dietary Supplement","year":"2020","type":"Inapplicable","leadProduct":"Ribitol","moa":"Xylose isomerase; Xylose isomerase; Xylose isomerase; Xylose isomerase; Hyaluronate lyase","graph1":"Infections and Infectious Diseases","graph2":"Phase III","graph3":"Larkin Community Hospital","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Infectious Diseases","amount2New":0,"dosageForm":"Nasal Spray","sponsorNew":"Larkin Community Hospital \/ Ferrer Internacional | Xlear, Inc","highestDevelopmentStatusID":"10","companyTruncated":"Larkin Community Hospital \/ Ferrer Internacional | Xlear, Inc"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Ribitol","moa":"Glycosylation","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Sachet","sponsorNew":"BridgeBio Pharma \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Bridge Biotherapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Ribitol","moa":"Glycosylation","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Bridge Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Sachet","sponsorNew":"Bridge Biotherapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Bridge Biotherapeutics \/ Inapplicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Ribitol","moa":"Glycosylation","graph1":"Genetic Disease","graph2":"Phase I","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BridgeBio Pharma \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"BridgeBio Pharma \/ Inapplicable"}]

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                          01

                          ChemOutsourcing
                          Not Confirmed
                          ChemOutsourcing
                          Not Confirmed

                          Details : BBP-418 (ribitol), an investigational oral substrate supplementation therapy, being investiated in patients with limb-girdle muscular dystrophy type 2I (LGMD2I/R9).

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          September 30, 2024

                          Lead Product(s) : Ribitol

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          ChemOutsourcing
                          Not Confirmed
                          ChemOutsourcing
                          Not Confirmed

                          Details : BBP-418 (ribitol), an investigational oral substrate supplementation therapy, being investiated in patients with limb-girdle muscular dystrophy type 2I (LGMD2I/R9).

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          June 18, 2024

                          Lead Product(s) : Ribitol

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          ChemOutsourcing
                          Not Confirmed
                          ChemOutsourcing
                          Not Confirmed

                          Details : BBP-418, an investigational oral substrate supplementation therapy, upstream of the mutant FKRP enzyme to help drive residual activity of the enzyme to glycosylate αDG in patients with limb-girdle muscular dystrophy type 2I (LGMD2I/R9).

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          July 31, 2023

                          Lead Product(s) : Ribitol

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          ML Bio Solutions

                          Country arrow
                          ChemOutsourcing
                          Not Confirmed

                          ML Bio Solutions

                          Country arrow
                          ChemOutsourcing
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          March 20, 2023

                          Lead Product(s) : Ribitol

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          ChemOutsourcing
                          Not Confirmed
                          ChemOutsourcing
                          Not Confirmed

                          Details : BBP-418 is being investigated to function as a substrate supplementation therapy that is hypothesized to drive glycosylation of αDG and target the root cause of the disease.

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          November 03, 2022

                          Lead Product(s) : Ribitol

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          ChemOutsourcing
                          Not Confirmed
                          ChemOutsourcing
                          Not Confirmed

                          Details : Phase 2 results continue to support the potential for BBP-418 to restore glycosylation of alpha-dystroglycan (αDG), reduce muscle damage, and preserve motor function for patients over time.

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          October 14, 2022

                          Lead Product(s) : Ribitol

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          ChemOutsourcing
                          Not Confirmed
                          ChemOutsourcing
                          Not Confirmed

                          Details : 43% increase in ratio of glycosylated alpha-dystroglycan to total αDG from baseline were measured for BBP-418, across all three dosing cohorts, signifying the oral therapy has potential to address both the root cause of LGMD2i and drive functional impro...

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          March 14, 2022

                          Lead Product(s) : Ribitol

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          ChemOutsourcing
                          Not Confirmed
                          ChemOutsourcing
                          Not Confirmed

                          Details : The Phase 1 clinical trial is designed to assess safety, tolerability, pharmacokinetics and food effect of BBP-418 in healthy volunteers.

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          November 06, 2020

                          Lead Product(s) : Ribitol

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          Larkin Community Hospital

                          Country arrow
                          ChemOutsourcing
                          Not Confirmed

                          Larkin Community Hospital

                          Country arrow
                          ChemOutsourcing
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Dietary Supplement

                          Upfront Cash : Inapplicable

                          November 02, 2020

                          Lead Product(s) : Ribitol

                          Therapeutic Area : Infections and Infectious Diseases

                          Highest Development Status : Phase III

                          Sponsor : Ferrer Internacional | Xlear, Inc

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          Iperboreal Pharma

                          Country arrow
                          ChemOutsourcing
                          Not Confirmed

                          Iperboreal Pharma

                          Country arrow
                          ChemOutsourcing
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Dietary Supplement

                          Upfront Cash : Inapplicable

                          June 27, 2019

                          Lead Product(s) : Ribitol

                          Therapeutic Area : Nephrology

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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