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Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Kwangdong Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Licensing Agreement","leadProduct":"OCU400","moa":"NR2E3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ocugen","amount2":0.01,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Kwangdong Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"Ocugen \/ Kwangdong Pharmaceutical"}]

Find Drugs for Genetic Disease in Phase III Clinical Development

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                          Top Deals by Deal Size (USD bn)

                          01

                          Lead Product(s) : Rilzabrutinib

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Rilzabrutinib is a Other Small Molecule drug candidate, which is currently being evaluated in Phase III clinical studies for the treatment of Anemia, Sickle Cell.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          May 16, 2025

                          Lead Product(s) : Rilzabrutinib

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          02

                          Lead Product(s) : Fitusiran

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase III

                          Sponsor : Undisclosed

                          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 : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          April 04, 2023

                          Lead Product(s) : Fitusiran

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          03

                          Details : Vertex will have the rights to conduct research using ImmunoGen's ADC technology to discover novel targeted conditioning agents for use with gene editing. CTX001 (exagamglogene autotemcel) is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edite...

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : $15.0 million

                          March 01, 2023

                          Lead Product(s) : Exagamglogene Autotemcel

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Vertex Pharmaceuticals

                          Deal Size : $352.0 million

                          Deal Type : Licensing Agreement

                          Immunogen

                          04

                          Lead Product(s) : Efanesoctocog Alpha

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : BIVV001 (efanesoctocog alfa) is a novel and investigational recombinant factor VIII therapy with the potential to deliver near-normal factor activity levels for most of the week, extending bleed protection in a once-weekly dose for people with haemophili...

                          Product Name : Undisclosed

                          Product Type : Protein

                          Upfront Cash : Inapplicable

                          January 25, 2023

                          Lead Product(s) : Efanesoctocog Alpha

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          05

                          Lead Product(s) : Fitusiran

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Fitusiran is a Oligonucleotide drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Hemophilia A.

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          December 22, 2022

                          Lead Product(s) : Fitusiran

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          06

                          Lead Product(s) : Efanesoctocog Alpha

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : BIVV001 (efanesoctocog alfa) is a novel and investigational recombinant factor VIII therapy that is designed to extend protection from bleeds with once-weekly prophylactic dosing for people with hemophilia A.

                          Product Name : Undisclosed

                          Product Type : Protein

                          Upfront Cash : Inapplicable

                          November 30, 2022

                          Lead Product(s) : Efanesoctocog Alpha

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          07

                          Lead Product(s) : Fitusiran

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase III

                          Sponsor : Undisclosed

                          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 : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          July 10, 2022

                          Lead Product(s) : Fitusiran

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          08

                          Lead Product(s) : Efanesoctocog Alpha

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase III

                          Sponsor : Swedish Orphan Biovitrum AB

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Investigational once-weekly BIVV001 (efanesoctocog alfa) prophylaxis met the primary efficacy endpoint providing clinically meaningful bleed protection for people with severe hemophilia A.

                          Product Name : Undisclosed

                          Product Type : Protein

                          Upfront Cash : Inapplicable

                          July 10, 2022

                          Lead Product(s) : Efanesoctocog Alpha

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Swedish Orphan Biovitrum AB

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          09

                          Lead Product(s) : Efanesoctocog Alpha

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase III

                          Sponsor : Swedish Orphan Biovitrum AB

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : BIVV001 (efanesoctocog alfa) is a novel and investigational factor VIII therapy designed to provide normal to near-normal factor activity levels for the majority of the week in a once-weekly prophylactic treatment regimen.

                          Product Name : Undisclosed

                          Product Type : Protein

                          Upfront Cash : Inapplicable

                          June 01, 2022

                          Lead Product(s) : Efanesoctocog Alpha

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Swedish Orphan Biovitrum AB

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          10

                          Lead Product(s) : Venglustat

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Venglustat is a Other Small Molecule drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Fabry Disease.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          March 15, 2022

                          Lead Product(s) : Venglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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