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    [{"orgOrder":0,"company":"Iperboreal Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Nephrology","country":"ITALY","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Ribitol","moa":"Glycosylation||Xylose isomerase; Xylose isomerase; Xylose isomerase; Xylose isomerase; Hyaluronate lyase","graph1":"Nephrology","graph2":"Phase II","graph3":"Iperboreal Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Nephrology","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Iperboreal Pharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Iperboreal Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Rambam Health Care Campus","sponsor":"Carmel Medical Center","pharmaFlowCategory":"DU","therapeuticArea":"Otolaryngology (Ear, Nose, Throat)","country":"ISRAEL","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Ribitol","moa":"Glycosylation||Xylose isomerase; Xylose isomerase; Xylose isomerase; Xylose isomerase; Hyaluronate lyase","graph1":"Otolaryngology (Ear, Nose, Throat)","graph2":"Undisclosed","graph3":"Rambam Health Care Campus","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Otolaryngology","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Rambam Health Care Campus \/ Carmel Medical Center","highestDevelopmentStatusID":"1","companyTruncated":"Rambam Health Care Campus \/ Carmel Medical Center"},{"orgOrder":0,"company":"Larkin Community Hospital","sponsor":"Ferrer Internacional | Xlear, Inc","pharmaFlowCategory":"DU","therapeuticArea":"Infections and Infectious Diseases","country":"U.S.A","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Ribitol","moa":"Glycosylation||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":"Spray","sponsorNew":"Larkin Community Hospital \/ Ferrer Internacional | Xlear, Inc","highestDevelopmentStatusID":"10","companyTruncated":"Larkin Community Hospital \/ Ferrer Internacional | Xlear, Inc"},{"orgOrder":0,"company":"Case Western Reserve University","sponsor":"Health Resources and Services Administration | University of Washington","pharmaFlowCategory":"DU","therapeuticArea":"Dental and Oral Health","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Ribitol","moa":"Glycosylation||Xylose isomerase; Xylose isomerase; Xylose isomerase; Xylose isomerase; Hyaluronate lyase","graph1":"Dental and Oral Health","graph2":"Undisclosed","graph3":"Case Western Reserve University","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Dental & Oral Health","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Case Western Reserve University \/ Health Resources and Services Administration | University of Washington","highestDevelopmentStatusID":"1","companyTruncated":"Case Western Reserve University \/ Health Resources and Services Administration | University of Washington"},{"orgOrder":0,"company":"Bridge Biotherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Miscellaneous","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 \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Bridge Biotherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"ML Bio Solutions","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","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":"Solution","sponsorNew":"ML Bio Solutions \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"ML Bio Solutions \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","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 \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","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 \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","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 \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","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":"Solution","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","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":"Solution","sponsorNew":"BridgeBio Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","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 \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"BridgeBio Pharma \/ Undisclosed"}]

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    E967

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                            01

                            BridgeBio Pharma

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                            BridgeBio Pharma

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                            Lead Product(s) : Ribitol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            BridgeBio Completes Enrollment in Phase 3 FORTIFY Study of BBP-418 in LGMD2I/R9

                            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 : Miscellaneous

                            Upfront Cash : Inapplicable

                            September 30, 2024

                            Lead Product(s) : Ribitol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            BridgeBio Pharma

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                            BridgeBio Pharma

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                            Lead Product(s) : Ribitol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            BridgeBio Surpasses Enrollment Target and Receives FDA Designation for BBP-418

                            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 : Miscellaneous

                            Upfront Cash : Inapplicable

                            June 18, 2024

                            Lead Product(s) : Ribitol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            BridgeBio Pharma

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                            BridgeBio Pharma

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                            Lead Product(s) : Ribitol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            BridgeBio Pharma Announces Opportunity for Accelerated Approval Pathway in Limb-girdle Muscular Dystrophy Type...

                            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 : Miscellaneous

                            Upfront Cash : Inapplicable

                            July 31, 2023

                            Lead Product(s) : Ribitol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            ML Bio Solutions

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                            ISPE Annual Meeting
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                            ML Bio Solutions

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                            Lead Product(s) : Ribitol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy...

                            Details : Ribitol is a Other Small Molecule drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Muscular Dystrophies, Limb-Girdle.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            March 20, 2023

                            Lead Product(s) : Ribitol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            BridgeBio Pharma

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                            ISPE Annual Meeting
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                            BridgeBio Pharma

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                            Lead Product(s) : Ribitol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            BridgeBio Pharma Presents 12-month Results from Phase 2 Study of BBP-418 in Limb-Girdle Muscular Dystrophy Typ...

                            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 : Miscellaneous

                            Upfront Cash : Inapplicable

                            October 14, 2022

                            Lead Product(s) : Ribitol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Bridge Biotherapeutics

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                            ISPE Annual Meeting
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                            Bridge Biotherapeutics

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                            ISPE Annual Meeting
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                            Lead Product(s) : Ribitol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            BridgeBio Pharma Announces Positive Phase 2 Data for Limb-girdle Muscular Dystrophy Type 2i (LGMD2i)

                            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 : Miscellaneous

                            Upfront Cash : Inapplicable

                            March 14, 2022

                            Lead Product(s) : Ribitol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            BridgeBio Pharma

                            U.S.A arrow
                            ISPE Annual Meeting
                            Not Confirmed

                            BridgeBio Pharma

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                            ISPE Annual Meeting
                            Not Confirmed
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                            Lead Product(s) : Ribitol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            BridgeBio Pharma, Inc. to Host Investor Call to Discuss Phase 2 Data for Limb-girdle Muscular Dystrophy Type 2...

                            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 : Miscellaneous

                            Upfront Cash : Inapplicable

                            March 11, 2022

                            Lead Product(s) : Ribitol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Larkin Community Hospital

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                            ISPE Annual Meeting
                            Not Confirmed

                            Larkin Community Hospital

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                            ISPE Annual Meeting
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                            Lead Product(s) : Ribitol

                            Therapeutic Area : Infections and Infectious Diseases

                            Study Phase : Phase III

                            Sponsor : Ferrer Internacional | Xlear, Inc

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Evaluation of the Efficacy of Xylitol Nasal Spray Against SARS-CoV-2

                            Details : Xylitol is a Dietary Supplement drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of COVID-19.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            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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                            09

                            BridgeBio Pharma

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                            ISPE Annual Meeting
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                            BridgeBio Pharma

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                            Lead Product(s) : Ribitol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            BridgeBio Pharma’s ML Bio Solutions Dosed First Subject in Phase 1 Clinical Trial of BBP-418 For Limb Girdle...

                            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 : Miscellaneous

                            Upfront Cash : Inapplicable

                            June 11, 2020

                            Lead Product(s) : Ribitol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Iperboreal Pharma

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                            ISPE Annual Meeting
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                            Iperboreal Pharma

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                            Lead Product(s) : Ribitol

                            Therapeutic Area : Nephrology

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Efficacy and Safety Assessments of a Peritoneal Dialysis Solution Containing Glucose, Xylitol, and L-Carnitine...

                            Details : Xylitol is a Dietary Supplement drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Kidney Failure, Chronic.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            June 27, 2019

                            Lead Product(s) : Ribitol

                            Therapeutic Area : Nephrology

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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