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& Gene Therapy","year":"2020","type":"Licensing Agreement","leadProduct":"OTL-103","moa":"Wiskott-Aldrich syndrome protein (WAS)","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Orchard Therapeutics","highestDevelopmentStatusID":"8","companyTruncated":"Orchard Therapeutics \/ Orchard Therapeutics"}]

Find Clinical Drug Pipeline Developments & Deals by Orchard Therapeutics

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          FNCE
                          Not Confirmed
                          FNCE
                          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 & Gene Therapy

                          Upfront Cash : Undisclosed

                          October 07, 2024

                          Lead Product(s) : Atidarsagene Autotemcel

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : Erkim

                          Deal Size : Undisclosed

                          Deal Type : Partnership

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                          02

                          FNCE
                          Not Confirmed
                          FNCE
                          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 & 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 : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          FNCE
                          Not Confirmed
                          FNCE
                          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 & 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 : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          FNCE
                          Not Confirmed
                          FNCE
                          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 : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          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 : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          FNCE
                          Not Confirmed
                          FNCE
                          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).

                          Product Name : Libmeldy

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Undisclosed

                          January 25, 2024

                          Lead Product(s) : Atidarsagene Autotemcel

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : Beneluxa Initiative

                          Deal Size : Undisclosed

                          Deal Type : Agreement

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                          06

                          FNCE
                          Not Confirmed
                          FNCE
                          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.

                          Product Name : Libmeldy

                          Product Type : Cell & 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 FDF

                          Sponsor : Kyowa Kirin

                          Deal Size : $477.6 million

                          Deal Type : Acquisition

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                          07

                          FNCE
                          Not Confirmed
                          FNCE
                          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).

                          Product Name : Libmeldy

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          December 11, 2023

                          Lead Product(s) : Atidarsagene Autotemcel

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          08

                          FNCE
                          Not Confirmed
                          FNCE
                          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.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          November 30, 2023

                          Lead Product(s) : OTL-203

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          FNCE
                          Not Confirmed
                          FNCE
                          Not Confirmed

                          Details : OTL-203 is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Mucopolysaccharidosis I.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          November 29, 2023

                          Lead Product(s) : OTL-203

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          FNCE
                          Not Confirmed
                          FNCE
                          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...

                          Product Name : Libmeldy

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : $387.4 million

                          October 05, 2023

                          Lead Product(s) : Atidarsagene Autotemcel

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : Kyowa Kirin

                          Deal Size : $387.4 million

                          Deal Type : Acquisition

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