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Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Orchard Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Orchard Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Libmeldy","moa":"ARSA","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Orchard Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Orchard Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"RA Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2023","type":"Private Placement","leadProduct":"Libmeldy","moa":"ARSA","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Orchard Therapeutics \/ RA Capital Management","highestDevelopmentStatusID":"15","companyTruncated":"Orchard Therapeutics \/ RA Capital Management"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Erkim","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2024","type":"Partnership","leadProduct":"Atidarsagene Autotemcel","moa":"ARSA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Erkim","highestDevelopmentStatusID":"15","companyTruncated":"Orchard Therapeutics \/ Erkim"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"MEI Pharma","pharmaFlowCategory":"DU","therapeuticArea":"Oncology","country":"JAPAN","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Zandelisib","moa":"PI3K delta","graph1":"Oncology","graph2":"Phase II","graph3":"Kyowa Kirin","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Oncology","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Kyowa Kirin \/ MEI Pharma","highestDevelopmentStatusID":"8","companyTruncated":"Kyowa Kirin \/ MEI Pharma"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Ophthalmology","country":"JAPAN","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Tivozanib","moa":"VEGF1 receptor","graph1":"Ophthalmology","graph2":"Phase II","graph3":"Kyowa Kirin","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Ophthalmology","amount2New":0,"dosageForm":"Ophthalmic Solution","sponsorNew":"Kyowa Kirin \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Kyowa Kirin \/ Inapplicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"OTL-203","moa":"Alpha-L-iduronidase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Orchard Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Orchard Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"OTL-203","moa":"Alpha-L-iduronidase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Orchard Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Orchard Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"San Raffaele-Telethon Institute for Gene Therapy","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"OTL-203","moa":"Alpha-L-iduronidase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Orchard Therapeutics \/ San Raffaele-Telethon Institute for Gene Therapy","highestDevelopmentStatusID":"10","companyTruncated":"Orchard Therapeutics \/ San Raffaele-Telethon Institute for Gene Therapy"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-203","moa":"Alpha-L-iduronidase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Orchard Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Orchard Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-203","moa":"Alpha-L-iduronidase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Orchard Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Orchard Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-203","moa":"Alpha-L-iduronidase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Orchard Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Orchard Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-203","moa":"Alpha-L-iduronidase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Orchard Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Orchard Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-203","moa":"Alpha-L-iduronidase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Orchard Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Orchard Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-203","moa":"Alpha-L-iduronidase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Orchard Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Orchard Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-201","moa":"SGSH","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Orchard Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"OTL-201","moa":"SGSH","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Orchard Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"OTL-201","moa":"SGSH","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Orchard Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-201","moa":"SGSH","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Orchard Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-201","moa":"SGSH","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Orchard Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"JAPAN","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"KW-6356","moa":"||A2AR","graph1":"Neurology","graph2":"Phase II","graph3":"Kyowa Kirin","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Oral","sponsorNew":"Kyowa Kirin \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Kyowa Kirin \/ Inapplicable"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Cerecor","pharmaFlowCategory":"D","therapeuticArea":"Pulmonary\/Respiratory Diseases","country":"JAPAN","productType":"Antibody","year":"2021","type":"Licensing Agreement","leadProduct":"AEVI-002","moa":"LIGHT","graph1":"Pulmonary\/Respiratory Diseases","graph2":"Phase I","graph3":"Kyowa Kirin","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Respiratory Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Kyowa Kirin \/ Cerecor","highestDevelopmentStatusID":"6","companyTruncated":"Kyowa Kirin \/ Cerecor"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Grunenthal","pharmaFlowCategory":"D","therapeuticArea":"Neurology","country":"JAPAN","productType":"Controlled Substance","year":"2022","type":"Collaboration","leadProduct":"Fentanyl","moa":"Mu opioid receptor","graph1":"Neurology","graph2":"Approved FDF","graph3":"Kyowa Kirin","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Sublingual Tablet","sponsorNew":"Kyowa Kirin \/ Grunenthal","highestDevelopmentStatusID":"15","companyTruncated":"Kyowa Kirin \/ Grunenthal"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Kura Oncology","pharmaFlowCategory":"DU","therapeuticArea":"Oncology","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Ziftomenib","moa":"MLL-Menin interaction","graph1":"Oncology","graph2":"Phase I\/ Phase II","graph3":"Kyowa Kirin","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Oncology","amount2New":0,"dosageForm":"Oral","sponsorNew":"Kyowa Kirin \/ Kyowa Kirin","highestDevelopmentStatusID":"7","companyTruncated":"Kyowa Kirin \/ Kyowa Kirin"}]

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                          Top Deals by Deal Size (USD bn)

                          01

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : KO-539 (ziftomenib), a menin-KMT2A/MLL inhibitor, being investigated for the treatment of patients with relapsed/refractory NPM1-mutant acute myeloid leukemia (AML).

                          Product Name : KO-539

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          April 08, 2025

                          Lead Product(s) : Ziftomenib

                          Therapeutic Area : Oncology

                          Highest Development Status : Phase I/ Phase II

                          Recipient : Kura Oncology

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : KHK4083 (rocatinlimab) is an anti-OX40 mAb that inhibits and reduces the number of OX40+ pathogenic T cells responsible for driving systemic and local atopic dermatitis inflammatory responses.

                          Product Name : KHK4083

                          Product Type : Antibody

                          Upfront Cash : Inapplicable

                          September 25, 2024

                          Lead Product(s) : Rocatinlimab

                          Therapeutic Area : Dermatology

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : The partnership covers a promotion and distribution agreement for commercial rights of Kyowa’s established medicines, including Crysvita (burosumab), indicated for X-linked hypophosphatemia.

                          Product Name : Crysvita

                          Product Type : Antibody

                          Upfront Cash : Undisclosed

                          August 02, 2024

                          Lead Product(s) : Burosumab

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : DKSH

                          Deal Size : Undisclosed

                          Deal Type : Partnership

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                          04

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

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

                          Product Name : Libmeldy

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          July 10, 2024

                          Lead Product(s) : Atidarsagene Autotemcel

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Erkim

                          Deal Size : Undisclosed

                          Deal Type : Partnership

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                          05

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : Under the terms of the agreement, NewBridge will commercialise CRYSVITA (burosumab), which is indicated for the treatment of X-Linked Hypophosphataemia.

                          Product Name : Crysvita

                          Product Type : Antibody

                          Upfront Cash : Undisclosed

                          July 08, 2024

                          Lead Product(s) : Burosumab

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : NewBridge Pharmaceuticals

                          Deal Size : Undisclosed

                          Deal Type : Agreement

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                          06

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : KHK4951 (tivozanib) is a small-molecule VEGFR-1, -2, and -3 tyrosine kinase inhibitor, currently evaluated in phase 2 trials for diabetic macular edema & wet age-related macular degeneration.

                          Product Name : KHK4951

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          June 02, 2024

                          Lead Product(s) : Tivozanib

                          Therapeutic Area : Ophthalmology

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          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.

                          Product Name : OTL-203

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          May 02, 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 : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : Under the terms of the agreement, Boehringer Ingelheim will receive exclusive, worldwide rights from Kyowa Kirin to develop novel, first-in-class treatments for fibro-inflammatory diseases.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Undisclosed

                          May 01, 2024

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Immunology

                          Highest Development Status : Undisclosed

                          Sponsor : Boehringer Ingelheim GmbH

                          Deal Size : $441.8 million

                          Deal Type : Licensing Agreement

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                          09

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

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

                          Product Name : Libmeldy

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          March 20, 2024

                          Lead Product(s) : Atidarsagene Autotemcel

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

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

                          Product Name : Libmeldy

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          March 18, 2024

                          Lead Product(s) : Atidarsagene Autotemcel

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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