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Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Acoramidis Hydrochloride","moa":"","graph1":"Cardiology\/Vascular Diseases","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Cardiovascular Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Ribitol","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"BridgeBio Pharma \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Not Applicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Infigratinib","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"BridgeBio Pharma \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Not Applicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Qatar Investment Authority","pharmaFlowCategory":"D","therapeuticArea":"Cardiology\/Vascular Diseases","country":"","productType":"Small molecule","year":"2023","type":"Private Placement","leadProduct":"Acoramidis Hydrochloride","moa":"","graph1":"Cardiology\/Vascular Diseases","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0.25,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Cardiovascular Diseases","amount2New":0.25,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ Qatar Investment Authority","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Qatar Investment Authority"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Resilience","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Collaboration","leadProduct":"BBP-812","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BridgeBio Pharma \/ BridgeBio Pharma","highestDevelopmentStatusID":"7","companyTruncated":"BridgeBio Pharma \/ BridgeBio Pharma"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Oncology","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"BBO-8520","moa":"","graph1":"Oncology","graph2":"Phase I","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Oncology","amount2New":0,"dosageForm":"","sponsorNew":"BridgeBio Pharma \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"BridgeBio Pharma \/ Not Applicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Blue Owl Capital","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Financing","leadProduct":"Acoramidis Hydrochloride","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0.5,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.5,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ Blue Owl Capital","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Blue Owl Capital"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Kyowa Kirin","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Licensing Agreement","leadProduct":"Infigratinib","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BridgeBio Pharma \/ Kyowa Kirin","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Kyowa Kirin"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Public Offering","leadProduct":"Acoramidis Hydrochloride","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0.25,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.25,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ J.P. Morgan","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ J.P. Morgan"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Bayer AG","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Licensing Agreement","leadProduct":"Acoramidis Hydrochloride","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ Bayer AG","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Bayer AG"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Invitae","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2024","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"BridgeBio Pharma \/ BridgeBio Pharma","highestDevelopmentStatusID":"2","companyTruncated":"BridgeBio Pharma \/ BridgeBio Pharma"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Bayer AG","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Acquisition","leadProduct":"Acoramidis Hydrochloride","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ Bayer AG","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Bayer AG"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Infigratinib","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BridgeBio Pharma \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Not Applicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Acoramidis Hydrochloride","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Acoramidis Hydrochloride","moa":"","graph1":"Cardiology\/Vascular Diseases","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Cardiovascular Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Not Applicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Cormorant Asset Management","pharmaFlowCategory":"D","therapeuticArea":"Oncology","country":"","productType":"Small molecule","year":"2024","type":"Private Placement","leadProduct":"BBO-8520","moa":"","graph1":"Oncology","graph2":"Phase I","graph3":"BridgeBio Pharma","amount2":0.20000000000000001,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Oncology","amount2New":0.20000000000000001,"dosageForm":"","sponsorNew":"BridgeBio Pharma \/ Cormorant Asset Management","highestDevelopmentStatusID":"6","companyTruncated":"BridgeBio Pharma \/ Cormorant Asset Management"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Infigratinib","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"BridgeBio Pharma \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Not Applicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Oncology","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"BBO-8520","moa":"","graph1":"Oncology","graph2":"Phase I","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Oncology","amount2New":0,"dosageForm":"","sponsorNew":"BridgeBio Pharma \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"BridgeBio Pharma \/ Not Applicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Ribitol","moa":"","graph1":"Technology","graph2":"Phase I\/II","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Technology","amount2New":0.94999999999999996,"dosageForm":"","sponsorNew":"BridgeBio Pharma \/ Not Applicable","highestDevelopmentStatusID":"14","companyTruncated":"BridgeBio Pharma \/ Not Applicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"BridgeBio Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Demerger","leadProduct":"Undisclosed","moa":"","graph1":"Technology","graph2":"Phase I\/II","graph3":"BridgeBio Pharma","amount2":0.29999999999999999,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Technology","amount2New":0.94999999999999996,"dosageForm":"","sponsorNew":"BridgeBio Pharma \/ BridgeBio Pharma","highestDevelopmentStatusID":"14","companyTruncated":"BridgeBio Pharma \/ BridgeBio Pharma"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"BBP-812","moa":"","graph1":"Technology","graph2":"Phase I\/II","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Technology","amount2New":0.94999999999999996,"dosageForm":"","sponsorNew":"BridgeBio Pharma \/ Not Applicable","highestDevelopmentStatusID":"14","companyTruncated":"BridgeBio Pharma \/ Not Applicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"BBP-631","moa":"","graph1":"Technology","graph2":"Phase I\/II","graph3":"BridgeBio 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Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Infigratinib","moa":"","graph1":"Technology","graph2":"Phase I\/II","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Technology","amount2New":0.94999999999999996,"dosageForm":"","sponsorNew":"BridgeBio Pharma \/ Not Applicable","highestDevelopmentStatusID":"14","companyTruncated":"BridgeBio Pharma \/ Not Applicable"}]

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                          01

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          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).

                          Brand Name : BBP-418

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          September 30, 2024

                          Lead Product(s) : Ribitol

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          02

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          Not Confirmed

                          Details : Truseltiq (infigratinib) is an oral small molecule, ATP-competitive, FGFR1-3 TKI in development for the treatment of achondroplasia in children.

                          Brand Name : Truseltiq

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          September 17, 2024

                          Lead Product(s) : Infigratinib

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          03

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          Not Confirmed

                          Details : BBP-812 is an investigational intravenous adeno-associated virus serotype 9 (AAV9) gene therapy candidate which is currently being evaluated for the treatment of Canavan disease.

                          Brand Name : BBP-812

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          September 10, 2024

                          Lead Product(s) : BBP-812

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          04

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          Not Confirmed

                          Details : BBP-631 is an investigational adeno-associated virus (AAV) 5 gene therapy candidate, which is being evaluated for the treatment of congenital adrenal hyperplasia.

                          Brand Name : BBP-631

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          September 10, 2024

                          Lead Product(s) : BBP-631

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          05

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          Not Confirmed

                          Details : GondolaBio will conduct R&D activities for early-stage clinical or preclinical programs across erythropoietic protoporphyria, alpha-1 antitrypsin deficiency and tuberous sclerosis complex.

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          August 21, 2024

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Preclinical

                          Recipient : GondolaBio

                          Deal Size : $300.0 million

                          Deal Type : Demerger

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                          06

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          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).

                          Brand Name : BBP-418

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          June 18, 2024

                          Lead Product(s) : Ribitol

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          07

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          Not Confirmed

                          Details : BBO-8520 is a first-in-class direct inhibitor of KRASG12C (ON). It is being evaluated for the treatment of KRASG12C mutant non-small cell lung cancer.

                          Brand Name : BBO-8520

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          June 06, 2024

                          Lead Product(s) : BBO-8520,Pembrolizumab

                          Therapeutic Area : Oncology

                          Highest Development Status : Phase I

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          08

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          Not Confirmed

                          Details : Truseltiq (infigratinib) is an oral small molecule, ATP-competitive, FGFR1-3 TKI in development for the treatment of achondroplasia in children.

                          Brand Name : Truseltiq

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          June 04, 2024

                          Lead Product(s) : Infigratinib

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          09

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          Not Confirmed

                          Details : The net proceeds will support the clinical advancement of BBO-8520, which is being evaluated in early-stage clinical trials with pembrolizumab for treating KRASG12C mutated non-small cell lung cancer.

                          Brand Name : BBO-8520

                          Molecule Type : Small molecule

                          Upfront Cash : Undisclosed

                          May 02, 2024

                          Lead Product(s) : BBO-8520,Pembrolizumab

                          Therapeutic Area : Oncology

                          Highest Development Status : Phase I

                          Sponsor : Cormorant Asset Management

                          Deal Size : $200.0 million

                          Deal Type : Private Placement

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                          10

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          Not Confirmed

                          Details : The net proceeds will be used in the development of AG10 (acoramidis HCl). Which is being evaluated in the Phase III clinical trial studies for the treatment of Transthyretin Amyloid Cardiomyopathy.

                          Brand Name : AG10

                          Molecule Type : Small molecule

                          Upfront Cash : Undisclosed

                          March 04, 2024

                          Lead Product(s) : Acoramidis Hydrochloride

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : J.P. Morgan

                          Deal Size : $250.0 million

                          Deal Type : Public Offering

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