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                          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 : Other Small Molecule

                          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

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                          02

                          Lead Product(s) : Olipudase Alfa

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          April 29, 2025

                          Lead Product(s) : Olipudase Alfa

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          Lead Product(s) : Efanesoctocog Alfa

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase IV

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Protein

                          Upfront Cash : Inapplicable

                          April 24, 2025

                          Lead Product(s) : Efanesoctocog Alfa

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase IV

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          04

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          March 30, 2025

                          Lead Product(s) : ABBV-CLS-628

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Calico Life Sciences

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Abbvie Company Banner

                          05

                          Lead Product(s) : SAR446268

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          February 25, 2025

                          Lead Product(s) : SAR446268

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          06

                          Lead Product(s) : Avalglucosidase Alfa

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase IV

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          October 30, 2024

                          Lead Product(s) : Avalglucosidase Alfa

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase IV

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          07

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          September 19, 2024

                          Lead Product(s) : Fosigotifator

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Abbvie Company Banner

                          08

                          Details : Under the terms of the agreement, Sionna will assume all development for ABBV-2222 (galicaftor), which is being evaluated in combination with navocaftor for the treatment of Cystic Fibrosis.

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Undisclosed

                          July 16, 2024

                          Lead Product(s) : Galicaftor,Navocaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Sionna Therapeutics

                          Deal Size : Undisclosed

                          Deal Type : Licensing Agreement

                          Abbvie Company Banner

                          09

                          Lead Product(s) : Avalglucosidase Alpha

                          Therapeutic Area : Genetic Disease

                          Study Phase : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Nexviazyme (avalglucosidase alfa) is a USFDA approved GAA stimulator enzyme replacement therapy. It is being evaluated for the treatment of treatment-naïve Infantile-onset Pompe disease.

                          Product Name : Nexviazyme

                          Product Type : Other Large Molecule

                          Upfront Cash : Inapplicable

                          May 02, 2024

                          Lead Product(s) : Avalglucosidase Alpha

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          10

                          Lead Product(s) : Eliglustat

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          January 05, 2024

                          Lead Product(s) : Eliglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner