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                          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

                          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

                          04

                          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

                          05

                          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

                          06

                          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

                          07

                          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

                          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 : 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

                          09

                          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

                          Sanofi Company Banner

                          10

                          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 : The study met the primary endpoint, showing a clinically meaningful prevention of bleeds in people with severe hemophilia A receiving weekly prophylaxis with BIVV001 (efanesoctocog alfa) over a period of 52 weeks.

                          Product Name : Undisclosed

                          Product Type : Protein

                          Upfront Cash : Inapplicable

                          March 09, 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