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KINGDOM","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Eplontersen","moa":"ATTR","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"AstraZeneca \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"AstraZeneca \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"FRANCE","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Fitusiran\u00a0","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"FRANCE","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Fitusiran\u00a0","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"FRANCE","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Fitusiran\u00a0","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"FRANCE","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Fitusiran\u00a0","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Public Offering","leadProduct":"Zilganersen","moa":"GFAP","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Ionis Pharmaceuticals \/ Morgan Stanley","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Morgan Stanley"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Zilganersen","moa":"GFAP","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Ionis Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Nippon Shinyaku","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"JAPAN","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Viltolarsen","moa":"DMD dystrophin pre-mRNA exon 53","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Nippon Shinyaku","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Nippon Shinyaku \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Nippon Shinyaku \/ Inapplicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Eplontersen","moa":"ATTR","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ionis Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Eplontersen","moa":"ATTR","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ionis Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Eplontersen","moa":"ATTR","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ionis Pharmaceuticals \/ AstraZeneca","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ AstraZeneca"},{"orgOrder":0,"company":"Dicerna Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Nedosiran","moa":"LDH","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Dicerna Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Dicerna Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Dicerna Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Dicerna Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Nedosiran","moa":"LDH","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Dicerna Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Dicerna Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Dicerna Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Dicerna Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Nedosiran","moa":"LDH","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Dicerna Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Dicerna Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Dicerna Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Eplontersen","moa":"ATTR","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ionis Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Expanded Collaboration","leadProduct":"Eplontersen","moa":"ATTR","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ionis Pharmaceuticals \/ AstraZeneca","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ AstraZeneca"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Eplontersen","moa":"ATTR","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ionis Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Agreement","leadProduct":"Eplontersen","moa":"ATTR","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ AstraZeneca","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ AstraZeneca"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Agreement","leadProduct":"Eplontersen","moa":"ATTR","graph1":"Rare Diseases and Disorders","graph2":"Phase 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Securities","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"GERMANY","productType":"Antibody","year":"2025","type":"Public Offering","leadProduct":"Vilobelimab","moa":"C5a","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"InflaRx","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.029999999999999999,"dosageForm":"Intravenous Infusion","sponsorNew":"InflaRx \/ Guggenheim Securities","highestDevelopmentStatusID":"10","companyTruncated":"InflaRx \/ Guggenheim Securities"}]

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                          01

                          Lead Product(s) : Fitusiran

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : SAR-439774 (fitusiran) is an investigational, subcutaneously administered small interference RNA therapeutic in development for the prophylactic treatment of people with hemophilia A or B, with or without inhibitors.

                          Product Name : SAR439774

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          April 04, 2023

                          Lead Product(s) : Fitusiran

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          02

                          Lead Product(s) : Fitusiran

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : SAR-439774 (fitusiran) is an investigational, subcutaneously administered small interference RNA therapeutic in development for the prophylactic treatment of people with hemophilia A or B, with or without inhibitors.

                          Product Name : SAR439774

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          July 10, 2022

                          Lead Product(s) : Fitusiran

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          03

                          Lead Product(s) : Fitusiran 

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Data from phase 3 studies achieved primary and secondary endpoints for ALN-AT3SC (Fitusiran) prophylaxis demonstrated significant and clinically meaningful improvements in bleed protection across all study populations.

                          Product Name : ALN-AT3SC

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          December 14, 2021

                          Lead Product(s) : Fitusiran 

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          04

                          Lead Product(s) : Fitusiran 

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Sanofi will resume fitusiran dosing in ongoing U.S. adolescent and adult clinical studies. Fitusiran is an investigational, small interference RNA therapy in development for the treatment of people with hemophilia A or B, with or without inhibitors.

                          Product Name : ALN-AT3SC

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          December 10, 2020

                          Lead Product(s) : Fitusiran 

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          05

                          Lead Product(s) : Fitusiran 

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : The WFH, EHC and NHF have learned of and subsequently confirmed a decision by Sanofi Genzyme to initiate a voluntary sponsor-led global dosing hold on its full clinical development program for fitusiran due to the identification of new adverse events.

                          Product Name : ALN-AT3SC

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          November 05, 2020

                          Lead Product(s) : Fitusiran 

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          06

                          Lead Product(s) : Fitusiran 

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Long-term interim results from the Phase 2 open-label extension (OLE) study reinforce fitusiran’s potential to restore hemostatic balance and to lower annualized bleed rates (ABRs) over a period up to 57 months.

                          Product Name : ALN-AT3SC

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          June 19, 2020

                          Lead Product(s) : Fitusiran 

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          07

                          Biotech Digital Meet
                          Not Confirmed
                          Biotech Digital Meet
                          Not Confirmed

                          Details : The net proceeds from the offering will used to fund the clinical development of Gohibic (vilobelimab). It is being evaluated for the treatment of Pyoderma Gangraenosum.

                          Product Name : Gohibic

                          Product Type : Antibody

                          Upfront Cash : Undisclosed

                          February 13, 2025

                          Lead Product(s) : Ifx-1

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Sponsor : Guggenheim Securities

                          Deal Size : $30.0 million

                          Deal Type : Public Offering

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                          08

                          Biotech Digital Meet
                          Not Confirmed
                          Biotech Digital Meet
                          Not Confirmed

                          Details : Qtorin rapamycin has the potential to be the first approved therapy and standard of care in the U.S. for the treatment of microcystic lymphatic malformations.

                          Product Name : Qtorin Rapamycin

                          Product Type : Antibiotic

                          Upfront Cash : Inapplicable

                          February 10, 2025

                          Lead Product(s) : Sirolimus

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          09

                          Biotech Digital Meet
                          Not Confirmed
                          Biotech Digital Meet
                          Not Confirmed

                          Details : SL1009 is a sodium dichloroacetate, which inhibits PDK to stimulate residual PDC activity and ATP production by mitochondria. It is being investigated for pyruvate dehydrogenase complex deficiency.

                          Product Name : SL1009

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          January 28, 2025

                          Lead Product(s) : Dichloroacetic Acid

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          Biotech Digital Meet
                          Not Confirmed
                          Biotech Digital Meet
                          Not Confirmed

                          Details : Under the licensing agreement, Nippon received an option to acquire exclusive U.S. rights to commercialize r-hIL-18BP (tadekinig alfa) to treat NLRC4-MAS.

                          Product Name : r-hIL-18BP

                          Product Type : Protein

                          Upfront Cash : $6.0 million

                          January 27, 2025

                          Lead Product(s) : Tadekinig Alfa

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Sponsor : Nippon Shinyaku

                          Deal Size : $686.0 million

                          Deal Type : Licensing Agreement

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