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                          01

                          Antibody Engineering
                          Not Confirmed
                          Antibody Engineering
                          Not Confirmed

                          Details : The agreement aims to commercialize Libmeldy (atidarsagene autotemcel), the only approved therapy for eligible children with early-onset metachromatic leukodystrophy (MLD).

                          Brand Name : Libmeldy

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          October 07, 2024

                          Lead Product(s) : Atidarsagene Autotemcel

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Er-Kim

                          Deal Size : Undisclosed

                          Deal Type : Partnership

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                          02

                          Antibody Engineering
                          Not Confirmed
                          Antibody Engineering
                          Not Confirmed

                          Details : Lenmeldy (atidarsagene autotemcel) is a cryopreserved dispersion of CD34+ cells containing hematopoietic stem cells, approved for early-onset metachromatic leukodystrophy treatment.

                          Brand Name : Lenmeldy

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          March 20, 2024

                          Lead Product(s) : Atidarsagene Autotemcel

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          03

                          Antibody Engineering
                          Not Confirmed
                          Antibody Engineering
                          Not Confirmed

                          Details : Lenmeldy (atidarsagene autotemcel) is a cryopreserved dispersion of CD34+ cells containing hematopoietic stem cells, approved for early-onset metachromatic leukodystrophy treatment.

                          Brand Name : Lenmeldy

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          March 18, 2024

                          Lead Product(s) : Atidarsagene Autotemcel

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          04

                          Antibody Engineering
                          Not Confirmed
                          Antibody Engineering
                          Not Confirmed

                          Details : OTL-203 is an investigational hematopoietic stem cell gene therapy using a modified virus to insert a functional IDUA gene copy into patient cells, under Phase 3 for MPS-I Hurler Syndrome.

                          Brand Name : OTL-203

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          February 05, 2024

                          Lead Product(s) : OTL-203

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : San Raffaele-Telethon Institute for Gene Therapy

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          05

                          Antibody Engineering
                          Not Confirmed
                          Antibody Engineering
                          Not Confirmed

                          Details : The agreement aims for enabling reimbursed access to Libmeldy (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, approved for early-onset metachromatic leukodystrophy (MLD).

                          Brand Name : Libmeldy

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          January 25, 2024

                          Lead Product(s) : Atidarsagene Autotemcel

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Beneluxa Initiative

                          Deal Size : Undisclosed

                          Deal Type : Agreement

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                          06

                          Antibody Engineering
                          Not Confirmed
                          Antibody Engineering
                          Not Confirmed

                          Details : Kyowa expands its portfolio to develop candidates with a differentiated platform and supports ongoing and future launches of Libmeldy (atidarsagene autotemcel) for metachromatic leukodystrophy.

                          Brand Name : Libmeldy

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : $387.4 million

                          January 24, 2024

                          Lead Product(s) : Atidarsagene Autotemcel

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : Kyowa Kirin

                          Deal Size : $477.6 million

                          Deal Type : Acquisition

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                          07

                          Antibody Engineering
                          Not Confirmed
                          Antibody Engineering
                          Not Confirmed

                          Details : OTL-200 (libmeldy or atidarsagene autotemcel), is a hematopoietic stem cell (HSC) gene therapy approved by Swissmedic for the treatment of early-onset metachromatic leukodystrophy (MLD).

                          Brand Name : Libmeldy

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          December 11, 2023

                          Lead Product(s) : Atidarsagene Autotemcel

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          08

                          Antibody Engineering
                          Not Confirmed
                          Antibody Engineering
                          Not Confirmed

                          Details : OTL-203 is an IDUA gene Transference cell and gene therapy drug infused intravenously, it is being investigated for the treatment of Hurler subtype of mucopolysaccharidosis type I.

                          Brand Name : OTL-203

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          November 30, 2023

                          Lead Product(s) : OTL-203

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          09

                          Antibody Engineering
                          Not Confirmed
                          Antibody Engineering
                          Not Confirmed

                          Details : Through the acquisition, Kyowa expands its portfolio, enables the development of promising candidates with a clinically differentiated platform, and helps resource the ongoing and future launches of Libmeldy (atidarsagene autotemcel) for metachromatic le...

                          Brand Name : Libmeldy

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : $387.4 million

                          October 05, 2023

                          Lead Product(s) : Atidarsagene Autotemcel

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Kyowa Kirin

                          Deal Size : $387.4 million

                          Deal Type : Acquisition

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                          10

                          Antibody Engineering
                          Not Confirmed
                          Antibody Engineering
                          Not Confirmed

                          Details : Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enz...

                          Brand Name : Libmeldy

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          September 18, 2023

                          Lead Product(s) : Atidarsagene Autotemcel

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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