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                          01

                          Iran Expo
                          Not Confirmed
                          Iran Expo
                          Not Confirmed

                          Details : Through the acquisition, Spruce will focus on the clinical development of AX 250 (tralesinidase alfa) enzyme replacement therapy (TA-ERT) for the treatment of Sanfilippo Syndrome Type B.

                          Product Name : AX 250

                          Product Type : Protein

                          Upfront Cash : Undisclosed

                          April 15, 2025

                          Lead Product(s) : Tralesinidase Alfa

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Spruce Biosciences

                          Deal Size : Undisclosed

                          Deal Type : Acquisition

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                          02

                          Iran Expo
                          Not Confirmed
                          Iran Expo
                          Not Confirmed

                          Details : Palynziq (pegvaliase) is a PEG PAL enzyme that converts phenylalanine to ammonia and trans-cinnamic acid. It reduces blood phenylalanine concentrations in patients with PKU.

                          Product Name : Palynziq

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          April 02, 2025

                          Lead Product(s) : Pegvaliase

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          Iran Expo
                          Not Confirmed
                          Iran Expo
                          Not Confirmed

                          Details : Roctavian (valoctocogene roxaparvovec) is a gene therapy approved for severe hemophilia A, that uses an adeno-associated virus 5, codes for human Factor VIII.

                          Product Name : Roctavian

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          May 08, 2024

                          Lead Product(s) : Valoctocogene Roxaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          Iran Expo
                          Not Confirmed
                          Iran Expo
                          Not Confirmed

                          Details : Voxzogo (vosoritide) is a C-type natriuretic peptide (CNP) analog, represents a new class of therapy, which acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.

                          Product Name : Voxzogo

                          Product Type : Peptide

                          Upfront Cash : Inapplicable

                          October 20, 2023

                          Lead Product(s) : Vosoritide

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          Iran Expo
                          Not Confirmed
                          Iran Expo
                          Not Confirmed

                          Details : Voxzogo (vosoritide) is a C-type natriuretic peptide (CNP) analog, represents a new class of therapy, which acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.

                          Product Name : Voxzogo

                          Product Type : Peptide

                          Upfront Cash : Inapplicable

                          September 15, 2023

                          Lead Product(s) : Vosoritide

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          Iran Expo
                          Not Confirmed
                          Iran Expo
                          Not Confirmed

                          Details : Roctavian (valoctocogene roxaparvovec) is a gene therapy that uses an adeno-associated virus 5, codes for human Factor VIII, together with a human liver-specific promoter. It is currently being investigated for adults with severe hemophilia A.

                          Product Name : Roctavian

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          September 01, 2023

                          Lead Product(s) : Valoctocogene Roxaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          Iran Expo
                          Not Confirmed
                          Iran Expo
                          Not Confirmed

                          Details : Voxzogo (vosoritide) is a C-type natriuretic peptide (CNP) analog, represents a new class of therapy, which acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.

                          Product Name : Voxzogo

                          Product Type : Peptide

                          Upfront Cash : Inapplicable

                          July 03, 2023

                          Lead Product(s) : Vosoritide

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          Iran Expo
                          Not Confirmed
                          Iran Expo
                          Not Confirmed

                          Details : Roctavian (valoctocogene roxaparvovec) is a gene therapy that uses an adeno-associated virus 5, codes for human Factor VIII, together with a human liver-specific promoter. It is currently being investigated for adults with severe hemophilia A.

                          Product Name : Roctavian

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          June 29, 2023

                          Lead Product(s) : Valoctocogene Roxaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          Iran Expo
                          Not Confirmed
                          Iran Expo
                          Not Confirmed

                          Details : Roctavian (valoctocogene roxaparvovec) is a gene therapy that uses an adeno-associated virus 5, codes for human Factor VIII, together with a human liver-specific promoter. It is currently being investigated for adults with severe hemophilia A.

                          Product Name : Roctavian

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          June 03, 2023

                          Lead Product(s) : Valoctocogene Roxaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          10

                          Iran Expo
                          Not Confirmed
                          Iran Expo
                          Not Confirmed

                          Details : The GENEr8-1 Phase 3 study for valoctocogene roxaparvovec demonstrated stable and durable bleed control, including a reduction in the mean annualized bleeding rate (ABR) and the mean annualized Factor VIII infusion rate.

                          Product Name : Roctavian

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          December 10, 2022

                          Lead Product(s) : Valoctocogene Roxaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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